Orphan Drugs

A to Z Drug Facts

Orphan Drugs

The Orphan Drug Act defines an orphan drug as a drug or biological product for the diagnosis, treatment, or prevention of a rare disease or condition. A rare disease is one that affects up to 200,000 people in the US or one that affects greater than 200,000 people but for which there is no reasonable expectation that the cost of developing the drug and making it available will be recovered from sales of that drug in the US.

The FDA Office of Orphan Products Development (OOPD) provides an information package that includes an overview of the FDA’s orphan drug program, a brief description of the orphan products grant program, and a current list of designated orphan products. OOPD’s information package also contains a directory sheet listing sources of information about the treatment of rare diseases, patient organizations, and availability of orphan drugs. Requests for the Rare Disease Information Directory or the entire orphan drugs information package may be made by contacting OOPD:

Those agents that have been approved for marketing or whose specific indication has been approved for marketing are denoted with the footnote 1.

Orphan Drugs

Drug (Trade name); Proposed use; Sponsor

1,5-(Butylimino)-1,5 dideoxy,D-glucitol; Fabry’s disease; Gaucher’s disease; Oxford GlycoSciences

1-(11-dodecylamino-10-hydroxyundecyl)-3-7-dimethylxanthine hydrogen methanesulfonate; Hormone refractory prostate carcinoma; Cell Therapeutics

111Indium pentetreotide (SomatoTher); Somatostatin receptor positive neuroendocrine tumors; Louisiana State Univ Medical Center Foundation

15AU81 (Remodulin); Pulmonary arterial hypertension; United Therapeutics

166Ho-DOTMP; Multiple myeloma; NeoRx

2’-deoxycytidine; Host-protective agent in acute myelogenous leukemia; Steven Grant, MD

2-0-desulfated heparin (Aeropin); Cystic fibrosis; Kennedy & Hoidal, MDs

24,25 dihydroxy-cholecalciferol; Uremic osteodystrophy; Lemmon

2-chloroethyl-3-sarcosinamide-1-nitrosourea (Sarmustine); Malignant glioma; Pangene Lawrence Panasci, MD

2-methoxyestradiol (Panzem); Multiple myeloma; EntreMed

3-(3,5-dimethyl-1H-2ylmethylene)-1,3-dihydro-indol-2-one; Kaposi’s sarcoma; von Hippel-Lindau disease; Sugen

3,4-diaminopyridine; Lambert-Eaton myasthenic syndrome; Jacobus Pharm

3-(4’aminoisoindoline-1’-one)-1-piperidine-2,6-dione (Revimid [proposed]); Multiple myeloma; Celgene

3,5,3’-triiodothyroacetate; Well-differentiated papillary, follicular, or combined papillary/follicular carcinomas of the thyroid gland; Elliot Danforth Jr, MD

4-aminosalicylic acid (Pamisyl) (Rezipas); Mild to moderate ulcerative colitis in patients intolerant to sulfasalazine; Warren Beeken, MD Parke-Davis Squibb

40SD02; Chronic iron overload resulting from conventional transfusional treatment of β-thalassemia major and sickle cell anemia; Biomedical Frontiers

5,6-dihydro-5-azacytidine; Malignant mesothelioma; ILEX Oncology

5-aza-2’-deoxycytidine; Acute leukemia; SuperGen

506U78; Chronic lymphocytic leukemia; GlaxoWellcome

6-hydroxymethylacylfulvene; Histologically confirmed advanced or metastatic pancreatic cancer; ovarian cancer; renal cellcarcinoma; MGI Pharma

8-Cyclopentyl 1,3-dipropylxanthine; Cystic fibrosis; SciClone Pharm

8-methoxsalen (Uvadex); In conjunction with the UVAR photopheresis system to treat diffuse systemic sclerosis; prevention of acute rejection of cardiac allografts; Therakos

9-cis retinoic acid (Panretin); Treatment of acute promyelocytic leukemia; Ligand Pharm

9-nitro-20-(S)-camptothecin (Camvirex); Pancreatic cancer; pediatric HIV infection/AIDS; Stehlin Foundation for Cancer Research NovoMed

Abetimus; Lupus nephritis; La Jolla Pharm

Acetylcysteine (Acetadote)(Mucomyst/Mucomyst 10 IV); IV treatment for moderate to severe acetaminophen overdose; Cumberland Bristol-Myers Squibb

Aconiazide; Tuberculosis; Lincoln Diagnostics

Adeno-associated viral-based vector cystic fibrosis gene therapy; Cystic fibrosis; Targeted Genetics

Adeno-associated viral vector containing the gene for human coagulation Factor IX (Coagulin-B); Intrahepatic and IM treatment of moderate to severe hemophilia; Avigen

Adenovirus-based vector Factor VIII complementary DNA to somatic cells (MiniAdFVIII); Hemophilia A; GenStar Therapeutics

Adenovirus-mediated herpes simplex virus-thymidine kinase gene; With gancyclovir in the treatment of malignant glioma; Ark Therapeutics Ltd.

Albendazole (Albenza); Hydatid disease (cystic echinococcosis caused by E. granulosus larvae or alveolar echinococcos is caused by E. multilocularis larvae;1 neurocysticercosis caused by Taenia solium as: 1) Chemotherapy of parenchymal, subarachnoidal, and racemose (cysts in spinal fluid) neurocysticercosis in symptomatic cases and 2) prophylaxis of epilepsy and other sequelae in asymptomatic neurocysticercosis1; SmithKline Beecham

Aldesleukin (Proleukin); Metastatic renal cell carcinoma;1 metastatic melanoma;1 primary immunodeficiency disease associated with T-cell defects; acute myelogenous leukemia; non-Hodgkin’s lymphoma; Chiron

Alemtuzumab (Campath); Chronic lymphocytic leukemia1; Millennium and ILEX Partners, LP

Alendronate disodium (Fosamax); Bone manifestations of Gaucher’s disease; Richard J. Wenstrup, MD

Alglucerase injection (Ceredase); Replacement therapy in Gaucher’s disease type I,1 II, and III; Genzyme

Alitretinoin (Panretin); Topical treatment of cutaneous lesions in AIDS-related Kaposi’s sarcoma1; Ligand Pharm

Allogeneic human retinal pigment epithelial cells on gelatin microcarriers (Spheramine); Hoehn and Yahr stage 3 and 4 Parkinson’s disease; Titan Pharm

Allogeneic peripheral blood mononuclear cells (sensitized against patient alloantigens by mixed lymphocyte culture) (CYTOIMPLANT); Pancreatic cancer; Applied Immunotherapeutics, LLC

Allopurinol sodium (Zyloprim for injection); Management of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy that causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy1; Catalytica Pharm

Alpha1-proteinase inhibitor (human); Slowing the progression of emphysema in alpha1-antitrypsin deficient patients; Aventis Behring LLC

(Prolastin); Replacement therapy in the alpha1-proteinase inhibitor congenital deficiency state1; Bayer

Alpha-galactosidase A (Fabrase)(CC-Galactosidase)(Replagal); Long-term enzyme replacement therapy for treatment of alpha-galactosidase A deficiency (Fabry’s disease); Robert J. Desnick, MDOrphan MedicalTranskaryotic Therapies

Alpha-melanocyte stimulatinghormone; Prevention and treatment of intrinsic acute renal failure caused by ischemia; National Institute of Diabetes, and Digestive and Kidney Diseases

Alronidase (Aldurazyme); Mucopolysaccharidosis I; BioMarin Pharm

Altretamine (Hexalen); Advanced ovarian adenocarcinoma1; Medimmune Oncology

Amifostine (Ethyol); Reduction of the incidence of moderate to severe xerostomia in postoperative radiation treatment for head and neck cancer;1 reduction of the incidence and severity of toxicities associated with cisplatin administration; myelodysplastic syndromes; chemoprotective agent for the following: cisplatin in metastatic melanoma, cisplatin in advanced ovarian carcinoma,1 cyclophosphamide in advanced ovarian carcinoma; MedImmune Oncology

Aminocaproic acid (Caprogel); Topical treatment of traumatic hyphema of the eye; Eastern Virginia Medical School/Dept. of Ophthalmology

Aminosalicylic acid (Paser Granules); Tuberculosis infections1; Jacobus Pharm Aminosidine; (Gabbromicina); Tuberculosis; Mycobacterium avium complex; Thomas P. Kanyok, PharmD

(Paromomycin); Visceral leishmaniasis (kala-azar); Thomas P. Kanyok, PharmD

Amiodarone (Amio-Aqueous); Incessant ventricular tachycardia; Wyeth Ayerst Research Labs

Amiodarone HCl (Cordarone); Acute treatment and prophylaxis of life-threatening ventricular tachycardia or ventricularfibrillation1; Wyeth-Ayerst

Ammonium tetrathiomolybdate; Wilson’s disease; George J. Brewer, MD

Amphotericin B lipid complex (Abelcet); Invasive fungal infections1; Liposome Co

Anagrelide (Agrylin); Polycythemia vera; essential thrombocythemia;1 thrombocytosis in chronic myelogenousleukemia; Roberts Pharm

Ananain, comosain (Vianain); For enzymatic debridement of severe burns; Genzyme

Ancestim (Stemgen); With filgrastim to decrease the number of phereses required to collect peripheral blood progenitor cells capable of providing rapid multi-lineage hematopoietic reconstitution following myelosuppressive or myeloablative therapy; Amgen

Ancrod (Viprinex); To establish and maintain anticoagulation in heparin-intolerant patients undergoing cardiopulmonary bypass; Knoll Pharm

Angiotensin 1-7 (MARstem); Neutropenia associated with autologous bone marrow transplantation; myelodysplastic syndrome; Maret Pharm

Antiepilepsirine; Drug-resistant generalized tonic-clonic epilepsy in children and adults; Children’s Hospital, Columbus, OH

Antihemophilic factor (human) (Alphanate); von Willebrand’s disease; Alpha Therapeutic

Antihemophilic factor (recombinant) (ReFacto); Control/Prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A1; Genetics Institute

Antihemophilic factor (recombinant) (Kogenate); Prophylaxis/Treatment of bleeding in hemophilia A1 and for prophylaxis when surgery is required in these patients1; Bayer

Antihemophilic factor/von Willebrand factor complex (human), dried,pasteurized (Humate-P); Treatment/Prevention of bleeding in hemophilia A (classical hemophilia) in adult patients and treatment of spontaneous and trauma-induced bleeding episodes in severe von Willebrand disease, and in mild to moderate von Willebrand disease where use of desmopressin is known or suspected to be inadequate in adult and pediatric patients1; Aventis Behring LLC

Antithrombin III (human)

(ATnativ); Hereditary antithrombin III deficiency in connection with surgical or obstetrical procedures or thromboembolism1; Pharmacia & Upjohn AB

(Thrombate III); Replacement therapy in congenital deficiency of AT-III to prevent and treat thrombosis and pulmonary emboli1; Bayer

(Antithrombin III human); To prevent/arrest episodes of thrombosis in patients with congenital AT-III deficiency or to prevent the occurrence of thrombosis in patients with AT-III deficiency who have undergone trauma or who are about to undergo surgery or parturition; American National Red Cross

Antithrombin III (human) concentrate IV (Kybernin P); Prophylaxis/Treatment of thromboembolic episodes in genetic AT-III deficiency; Aventis Behring LLC

Anti-thymocyte globulin (Rabbit) (Thymoglobulin); Myelodysplastic syndrome (MDS); SangStat Medical

Anti-thymocyte serum (Nashville Rabbit Anti-thymocyteSerum); Allograft rejection, including solid organ (kidney, liver, heart, lung, pancreas) and bone marrow transplantation; Applied Medical Research

Antivenin crotalidae polyvalent immune Fab (ovine)(CroFab); Treatment of envenomations inflicted by North American crotalid snakes1; Protherics

Antivenom (crotalidae) purified(avian); Treatment of envenomation by poisonous snakes belonging to the Crotalidae family; Ophidian

AP1903; Acute graft-vs-host disease in patients undergoing bone marrow transplantation; Ariad Gene Therapeutics

APL 400-020 V-Beta DNA vaccine; Cutaneous T-cell lymphoma; Wyeth-Lederle Vaccines & Pediatrics

Apomorphine; Treatment of the on-off fluctuations associated with late-stage Parkinson’s disease; Pentech Pharm

Apomorphine HCl; Treatment of the on-off fluctuations associated with late-stage Parkinson’s disease; Mylan

Aprotinin (Trasylol); Prophylaxis to reduce perioperative blood loss and the homologous blood transfusion requirement in patients undergoing cardiopulmonary bypass surgery in the course of repeat coronary artery bypass graft (CABG) surgery, and in selected cases of primary CABG surgery when the risk of bleeding is especially high (impaired hemostasis), or where transfusion is unavailable or unacceptable1; Bayer

Arcitumomab (99m Tc-labeled CEA-Scan); Diagnosis and localization of primary, residual, recurrent, and metastatic medullary thyroidcarcinoma; Immunomedics

Arginine butyrate; Beta-hemoglobinopathies and beta-thalassemia; Susan P. Perrine, MD

Arsenic trioxide (Trisenox); Acute promyelocytic leukemia;1 chronic myeloid leukemia; multiple myeloma; myelodysplastic syndrome;1 acute myelocytic leukemia subtypes M0, M1, M2, M4, M5, M6, and M7; Cell Therapeutics

Artesunate; Malaria; World Health Organization

Atovaquone (Mepron); AIDS-associated Pneumocystis carinii pneumonia (PCP);1 prevention of PCP in high-risk, HIV-infected patients (defined by ³ 1 episode of PCP or a peripheral CD4+ (T4 helper/inducer) lymphocyte count £ 200/mm3);1 treatment/suppression of Toxoplasma gondii encephalitis; primary prophylaxis of HIV-infected people at high risk for developing T. gondii encephalitis; GlaxoWellcome GlaxoWellcome Res & Devel

Augmerosen (Genasense); Multiple myeloma; acute myelocytic leukemia; chronic lymphocytic leukemia; Genta

Autologous DNP-conjugated tumor vaccine (M-Vax); Adjuvant therapy in melanoma patients with surgically resectable lymph node metastasis (Stage III and limited Stage IV disease); Avax Technologies

Autolymphocyte therapy; Renal cell carcinoma; Cytogen

Azacitadine; Myelodysplastic syndromes; Pharmion

Azathioprine (Imuran); Oral manifestations of graft-vs-host disease; Oral Solutions

B lymphocyte stimulator (BLyS); Common variable immunodeficiency (CVID); Human Genome Sciences

Bacitracin (Altracin); Antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile; AL Labs

Baclofen (Lioresal Intrathecal); Intractable spasticity caused by multiple sclerosis or spinal cord injury; intractable spasticity caused by spinal cord injury, multiple sclerosis, and other spinal diseases (eg, spinal ischemia, spinal tumor, transverse myelitis, cervical spondylosis, degenerative myelopathy);1 spasticity associated with cerebral palsy; Infusaid Medtronic

Basiliximab (Simulect); Prophylaxis of solid organ rejection1; Novartis

Beclomethasone 17,21-dipropionate; To prevent GI graft-vs-host disease; Enteron Pharm

Beclomethasone dipropionate; Oral administration for intestinal graft-vs-host disease; George B. McDonald, MD

Benzoate and phenylacetate (Ucephan); Adjunctive therapy to prevent/treat hyperammonemia in patients with urea cycle enzymopathy caused bycarbamylphosphate synthetase, ornithine, transcarbamylase, or argininosuccinate synthetase deficiency1; B Braun Medical

Benzoate/Phenylacetate (Ammonul); Acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle; Medicis Pharm

Benzophenone-3, octylmethyoxycinna-mate, avobenzone, titanium dioxide, zinc oxide (Total Block VL SPF 75); Prevent visible light-induced skin photosensitivity as a result of porfimer sodium photodynamic therapy; Fallien Cosmeceuticals Ltd.

Benzydamine HCl (Tantum); Prophylactic treatment of oral mucositis resulting from radiation therapy for head andneck cancer; Angelini Pharm

Benzylpenicillin, benzylpenicilloic, benzylpenilloic acid (Pre-Pen/MDM); To assess risk of penicillin administration when it is the preferred drug in adults who have a history of clinical penicillin sensitivity; Hollister-Stier Labs LLC

Beractant (Survanta Intratracheal Suspension); Prevention/Treatment of neonatal respiratory distress syndrome;1 full-term newborns with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis; Ross

Beraprost; Pulmonary arterial hypertension associated with any New York Heart Association classification; United Therapeutics

Beta alethine (Betathine); Multiple myeloma; metastatic melanoma; Dovetail Tech

Betaine (Cystadane); Homocystinuria1; Orphan Medical

Bexarotene (Targretin); Cutaneous manifestations of cutaneous T-cell lymphoma in patients who are refractory to ³ 1 prior systemic therapy1; Ligand Pharm

Bindarit; Lupus nephritis; Angelini Pharm

Bis(4-fluorophenyl)phenylacetamide; Sickle cell disease; ICAgen

Bispecific antibody 520c9x22; In vivo serotherapy for ovarian cancer; Medarex

Bleomycin (Blenoxane); Pancreatic cancer; Genetronics

Bleomycin sulfate (Blenoxane); Malignant pleural effusion1; Bristol-Myers SquibbPharm Res Inst

Bosentan (Tracleer); Pulmonary arterial hypertension1; Actelion Life Sciences

Botulinum toxin type A; Synkinetic closure of the eyelid associated with VII cranial nerve aberrant regeneration; Botulinim Toxin Res Assoc

(Botox); Blepharospasm and strabismus associated with dystonia in adults (³ 12 years of age);1 cervical dystonia;1 dynamic muscle contracture in pediatric cerebral palsy; Allergan

(Dysport); Cervical dystonia; dynamic muscle contractures in pediatric cerebral palsy; essential blepharospasm; Ipsen Limited Porton International

Botulinum toxin type B (NeuroBloc); Cervical dystonia1; Elan Pharm

Botulinum toxin type F (Dysport); Cervical dystonia; essential blepharospasm; Ipsen Limited

Botulism immune globulin; Infant botulism; CA Dept Health Services

Bovine colostrum; AIDS-related diarrhea; Donald Hastings, DVM

Bovine immunoglobulin concentrate, Cryptosporidium parvum (Sporidin-G); Treatment/Symptomatic relief of Cryptosporidium parvum infection of the GI tract in immunocompromised patients; GalaGen

Bovine whey protein concentrate (Immuno-C); Cryptosporidiosis caused by Cryptosporidium parvum in the GI tract of patients who are immunodeficient/immunocompromised or immunocompetent; Biomune Systems

Branched chain amino acids; Amyotrophic lateral sclerosis; Mount Sinai Medical Center

Brimonidine (Alphagan); Anterior ischemic optic neuropathy; Allergan

Bromhexine (Bisolvon); Mild to moderate keratoconjunctivitis sicca in Sjogren’s syndrome; Boehringer Ingelheim

Broxuridine (Broxine/Neomark); Radiation sensitizer in the treatment of primary brain tumors; NeoPharm

Bryostatin-1; With paclitaxel in the treatment of esophageal cancer; GPC Biotech

Buffered intrathecal electrolyte/dextrose injection (Elliotts B Solution); Diluent in intrathecal administration of methotrexate and cytarabine for prevention/treatment of meningeal leukemia or lymphocytic lymphoma1; Orphan Medical

Buprenorphine HCl (Subutex); Opiate addictions in opiate users; Reckitt & Colman

Buprenorphine in combination with naloxone (Suboxone); Opiate addiction in opiate users; Reckitt & Colman

Busulfan

(Busulfex); Preparative therapy in the treatment of malignancies with bone marrow transplantation1; Orphan Medical

(Spartaject); Preparative therapy for malignancies treated with bone marrow transplantation; primary brain malignancies; Sparta SuperGen

(Busulfan-Spartaject); Intrathecal therapy for neoplastic meningitis; SuperGen

Butyrylcholinesterase; Reduction and clearance of toxic blood levels of cocaine in overdose; postsurgical apnea; Shire Labs

BW 12C; Sickle cell disease; Burroughs Wellcome

C1 esterase inhibitor (human); Prevention/Treatment of angioedema caused by C1-esterase inhibitor deficiency; Alpha Therapeutic

C1-esterase inhibitor, human,pasteurized (Berinert P); Prevention/Treatment of acute attacks of hereditary angioedema; Aventis Behring LLC

C1-inhibitor (C1-Inhibitor [human] Vapor Heated, Immuno); Treatment of acute attacks of angioedema; prevention of acute attacks of angioedema, including short-term prophylaxis for patients requiring dental or other surgical procedures; Baxter Healthcare

Caffeine (Cafcit); Apnea of prematurity1; OPR Development, LP

Calcitonin-human for injection (Cibacalcin); Symptomatic Paget’s disease (osteitis deformans)1; Novartis

Calcium acetate (Phos-Lo); Hyperphosphatemia in end-stage renal failure1/disease; Braintree Pharmedic

Calcium carbonate (R & D Calcium Carbonate/600); Hyperphosphatemia in end-stage renal disease; R & D Labs

Calcium gluconate (Calgonate); A wash for hydrofluoric acid spills on human skin; Calgonate

Calcium gluconate gel (H-F Gel); Emergency topical treatment of hydrogen fluoride (hydrofluoric acid) burns; LTR Pharm Paddock Labs

Calfactant (Infasurf); Acute respiratory distress syndrome (ARDS); ONY

Carbamic acid, [[4-[[3-[[4-[1-(4-hydroxyphenyl)-1-methyl-ethyl]phenoxyl]methyl]phenyl]methoxy]-phenyl]iminomethyl]-,ethyl ester; Management of cystic fibrosis; Boeringer Ingelheim

Carbamylglutamic acid; N-acetylglutamate synthetase deficiency; Orphan Europe

Carmustine; Intracranial malignancies; Direct Therapeutics

Cascara sagrada fluid extract; For oral drug overdosage to speed lower bowel evacuation; Intramed

CDP571; Crohn’s disease; Celltech Chiroscience Limited

Centruroides immune F(ab)2 (Alacramyn); Scorpion envenomations requiring medical attention; Silanes Labs SA de CV

Ceramide trihexosidase/alpha-galactosidase A; Fabry’s disease; Genzyme

Cetuximab; Squamous cell cancer of the head and neck in patients who express epidermal growth factor receptor; ImClone Systems

Chenodiol (Chenix); Radiolucent stones in well-opacifying gallbladders, where elective surgery would be undertaken except for presence of increased surgical risk caused bysystemic disease or age1; Solvay

Chimeric (human-murine) G250 IgG monoclonal antibody; Renal cell carcinoma; Wilex Biotechnology GmbH

Chimeric, humanized monoclonal antibody to staphylococcus; Prophylaxis of S. epidermidis sepsis in low birth weight (£ 1500 g) infants; Biosynexus

Chlorhexidine gluconate mouth rinse (Peridex); Amelioration of oral mucositis associated with cytoreductive therapy used in conditioning patients for bone marrow transplantation therapy; Procter & Gamble

Choline chloride (Intrachol); Choline deficiency, specifically the choline deficiency, hepatic steatosis, and cholestasis associated with long-term parenteral nutrition; Orphan Medical

Chondrocyte-alginate gel suspension; To correct vesicoureteral reflux in the pediatric population; Curis

Ciliary neurotrophic factor; Amyotrophic lateral sclerosis; Regeneron Pharm

Cisplatin/Epinephrine (IntraDose); Metastatic malignant melanoma; squamous cell carcinoma of the head and neck; Matrix Pharm

Citric acid, glucono-delta-lactone, and magnesium carbonate (Renacidin Irrigation); Renal and bladder calculi of the apatite or struvite variety1; United-Guardian

Cladribine

(Leustatin Injection); Hairy-cell leukemia;1 chronic lymphocytic leukemia; non-Hodgkin’s lymphoma; acute myeloid leukemia; RW Johnson Pharm Res Inst

(Mylinax); Chronic progressive multiple sclerosis; RW Johnson Pharm Res Inst

Clindamycin (Cleocin); Treatment/Prevention of Pneumocystis carinii pneumonia associated with AIDS; Pharmacia & Upjohn

Clofazimine (Lamprene); Lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum1; Novartis

Clonazepam (Klonopin); Hyperexplexia (startle disease); Hoffmann-La Roche

Clonidine (Duraclon); For continuous epidural administration as adjunctive therapy with intraspinal opiates for pain in cancer patients tolerant or unresponsive to intraspinal opiates1; Roxane Labs

Clostridial collagenase; Advanced (involutional or residual stage) Dupuytren’s disease; L. Hurst, MD and M. Badalamente, PhD

Clotrimazole; Sickle cell disease; Carlo Brugnara, MD

Coagulation factor VIIa(recombinant) (NovoSeven); Bleeding episodes in hemophilia A or B with inhibitors to Factor VIII or Factor IX1; Novo Nordisk Pharm

Coagulation factor IX (Mononine); Replacement treatment for/prophylaxis of hemorrhagic complications of hemophilia B1; Armour Pharm

Coagulation factor IX (human) (AlphaNine); Replacement therapy in hemophilia B for prevention and control of bleeding episodes and during surgery to correct defective hemostasis1; Alpha Therapeutic

Coagulation factor IX (BeneFix); Hemophilia B1; Genetics Institute

Coenzyme Q10; Huntington’s disease; Vitaline

Colfosceril palmitate, cetyl alcohol, tyloxapol

(Exosurf Neonatal for Intratracheal Suspension); Prevention of hyaline membrane disease (respiratory distress syndrome) in infants born at£ 32 weeks gestation;1 to treat established hyaline membrane disease at all gestational ages1; GlaxoWellcome

(Exosurf); Adult respiratory distress syndrome (ARDS); GlaxoWellcome Res & Devel

Collagenase (lyophilized) for injection (Plaquase); Peyronie’s disease; Advance Biofactures

Conjugate of human transferrin and a mutant diphtheria toxin (CRM 107); Malignant tumors of the CNS; INTELLIgene Expressions

Corticorelin ovine triflutate (Acthrel); To differentiate between pituitary and ectopic production of ACTH in ACTH-dependent Cushing’s syndrome1; Ferring Labs

Corticotropin-releasing factor, human (Xerecept); Peritumoral brain edema; Neurobiological Technologies

Coumarin (Onkolox); Renal cell carcinoma; Drossapharm LTD

Cromolyn sodium (Gastrocrom); Mastocytosis1; Fisons

Cromolyn sodium 4% ophthalmic solution (Opticrom 4% Ophthalmic Solution); Vernal keratoconjunctivitis1; Fisons

Cryptosporidium hyperimmune bovine colostrum IgG concentrate; Diarrhea in AIDS patients caused by infection with Cryptosporidium parvum; ImmuCell

CT-2584 mesylate; Adult soft tissue sarcoma; malignant mesothelioma; Cell Therapeutics

CY-1503 (Cylexin); Postischemic pulmonary reperfusion edema following surgical treatment for chronic thromboembolic pulmonary hypertension; for neonates and infants undergoing cardiopulmonary bypass during surgical repair of congenital heart lesions; Cytel

CY-1899; Chronic active hepatitis B infection in HLA-A2 positive patients; Cytel

Cyclosporine ophthalmic (Optimmune); Severe keratoconjunctivitis sicca associated with Sjogren’s syndrome; University of Georgia

Cyclosporine 2% ophthalmic ointment; Treatment of patients at high risk of graft rejection following penetrating keratoplasty; corneal melting syndromes of known or presumed immunologic etiopathogenesis, including Mooren’s ulcer; Allergan

Cyclosporine in combination with omega-3 polyunsaturated fatty acids; Prevention of solid organ graft rejection; RTP Pharma

Cysteamine (Cystagon); Nephropathic cystinosis1; Jess G. Thoene, MD Mylan

Cysteamine HCl; Corneal cystine crystal accumulation in cystinosis patients; Sigma-Tau Pharmaceuticals

Cystic fibrosis gene therapy; Cystic fibrosis; Genzyme

Cystic fibrosis transmembrane conductance regulator gene; Cystic fibrosis; Genetic Therapy

Cystic fibrosis Tr gene therapy(recombinant adenovirus) (AdGVCFTR.10); Cystic fibrosis; GenVec

Cytarabine liposomal (DepoCyt); Neoplastic meningitis1; DepoTech

Cytomegalovirus immune globulin (human) (CytoGam); Prevention or attenuation of primary cytomegalovirus (CMV) disease in immunosuppressed recipients of organ transplants1; MA Public Health Bio Labs

Cytomegalovirus immune globulin IV (human); With ganciclovir sodium for the treatment of CMV pneumonia in bone marrow transplant patients; Bayer

Daclizumab (Zenapax); Prevention of acute renal allograft rejection1; Hoffmann-LaRoche

Dapsone; Prophylaxis of toxoplasmosis in severely immunocompromised patients with CD4 counts < 100; Jacobus Pharm

Dapsone, USP (Dapsone); Prophylaxis of Pneumocystis carinii pneumonia (PCP); with trimethoprim for treatment of PCP; Jacobus Pharm

Daunorubicin citrate liposomeinjection (DaunoXome); Treatment of patients with advanced HIV-associated Kaposi’s sarcoma1; NeXstar

Decitabine; Myelodysplastic syndromes; chronic myelogenous leukemia; SuperGen

Deferiprone (Ferriprox); Iron overload in patients with hematologic disorders requiring chronic transfusion therapy; Apotex Research

Defibrotide; Thrombotic thrombocytopenic purpura; Crinos

Dehydroepiandrosterone; Systemic lupus erythematosus (SLE) and reduction of steroid use in steroid-dependent SLE; Genelabs Tech

Dehydroepiandrosterone sulfate sodium; Treatment of serious burns requiring hospitalization; to accelerate re-epithelialization of donor sites in autologous skin grafting; Pharmadigm

Denileukin diftitox (Ontak); Persistent or recurrent cutaneous T-cell lymphoma whose malignant cells express the CD25 component of the IL-2 receptor; Seragen

Deoxyribose, phosphorothioate; Advanced malignant melanoma (Stages II, III, and IV); Genta

Deslorelin (Somagard); Central precocious puberty; Roberts Pharm

Desmopressin acetate; Mild hemophilia A and von Willebrand’s disease1; Aventis Behring, LLC

Dexamethasone; Used in posterior segment drug delivery system in idiopathic intermediate uveitis treatment; Oculex Pharm

Dexrazoxane (Zinecard); Prevention of cardiomyopathy associated with doxorubicin administration1; Pharmacia & Upjohn

Dextran 70 (Dehydrex); Recurrent corneal erosion unresponsive to conventional therapy; Holles Labs

Dextran and deferoxamine (Bio-Rescue); Acute iron poisoning; Biomedical Frontiers

Dextran sulfate (inhaled, aerosolized) (Uendex); Adjunct to the treatment of cystic fibrosis; Kennedy & Hoidal, MDs

Dextran sulfate sodium; AIDS; Ueno Fine Chemicals

DHA-paclitaxel (Taxoprexin); Pancreatic cancer; Protarga

Dianeal peritoneal dialysis solution with 1.1% amino acids (Nutrineal [Peritoneal Dialysis Solution with 1.1% Amino Acid]); Nutritional supplement for malnourishment in patients undergoing continuous ambulatoryperitoneal dialysis; Baxter Healthcare

Diazepam viscous solution for rectal administration; For the management of selected, refractory patients with epilepsy on stable regimens of antiepileptic drugs (AEDs), who require intermittent use of diazepam to control bouts of increasedseizure activity1; Athena Neurosciences

Diethyldithiocarba-mate (Imuthiol); AIDS; Connaught

Digitoxin; Soft tissue sarcomas; ovarian cancer; PrimeCyte

Digoxin immune FAB (ovine)

(Digibind); Treatment of potentially life-threatening digitalis intoxication in patients refractory to management by conventional therapy1; GlaxoWellcome

(Digidote); Life-threatening acute cardiac glycoside intoxication manifested by conduction disorders, ectopic ventricular activity, and sometimes hyperkalemia; Boehringer Mannheim

Dihydrotestosterone (Androgel-DHT); Weight loss in AIDS with HIV-associated wasting; Unimed

Dimethyl sulfoxide; Increased intracranial pressure in patients with severe, closed-head injury (traumatic brain coma) for whom no other effective treatment is available; Pharma 21

Dimethylsulfoxide; Topical treatment for the prevention of soft tissue injury following extravasation of cytotoxic drugs; palmar-plantar-erythrodysesthesia syndrome; Cancer Technologies

Dipalmitoylphospha-tidylcholine/Phosphatidylglycerol (ALEC); Prevention/Treatment of neonatal respiratory distress syndrome; Forum Products

Disaccharide tripeptide glyceroldipalmitoyl (Immther); Pulmonary and hepatic metastases in colorectal adenocarcinoma; ImmunoTherapeutics

Disodium clodronate; Hypercalcemia of malignancy; Discovery Experimental &Development

Disodium clodronate tetrahydrate (Bonefos); Increased bone resorption caused by malignancy; Anthra Pharm

DMP 777; Therapeutic management of lung disease attributable to cystic fibrosis; DuPont Pharm

DNA-lipid complex (DMRIE/DOPE)/plasmid vector (VCL-1102, Vical) expressing human interleukin-2 (Leuvectin); Renal cell carcinoma; Vical

DNP-Modified autologous tumor vaccine (O-Vax); Adjuvant therapy for the treatment of ovarian cancer; AVAX Technologies

Docosahexanoic acid-paclitaxel (Taxoprexin); Hormone-refractory prostate cancer; Protarga

Dornase alfa (Pulmozyme); Reduces mucous viscosity and enables the clearance of airway secretions in cystic fibrosis1; Genentech

Doxorubicin liposome (Doxil); Ovarian cancer1; Alza

Dronabinol (Marinol); For the stimulation of appetite and prevention of weight loss in patients with a confirmeddiagnosis of AIDS1; Unimed Pharm

Duramycin; Cystic fibrosis; MoliChem Medicines

Dynamine; Lambert-Eaton myasthenic syndrome; hereditary motor and sensory neuropathy type I(Charcot-Marie-Tooth disease); Mayo Foundation

Eflornithine HCl (Ornidyl); Trypanosoma brucei gambiense infection (sleeping sickness)1; Hoechst Marion Roussel

Elcatonin; Intrathecal treatment of intractable pain; Innapharma

Enadoline HCl; Severe head injury; Warner-Lambert

Encapsulated porcine islet preparation (BetaRx); Type I diabetic patients already on immunosuppression; VivoRx

Epidermal growth factor (human); Acceleration of corneal epithelial regeneration and healing of stromal tissue in nonhealing corneal defects; Chiron Vision

Epirubicin (Ellence); Breast cancer1; Pharmacia & Upjohn

Epoetin alfa; Myelodysplastic syndrome; RW Johnson Pharm Res Inst

(Epogen); Anemia associated with end-stage renal disease1 or HIV infection or treatment1; Amgen

(Procrit); Anemia associated with end-stage renal disease; anemia of prematurity in preterm infants; HIV-associated anemia related to HIV infection or treatment; RW Johnson Pharm Res Inst

Epoetin beta (Marogen); Anemia associated with end-stage renal disease; Chugai-USA

Epoprostenol (Flolan); Primary pulmonary hypertension;1 secondary pulmonary hypertension caused byintrinsic precapillary pulmonary vascular disease1; GlaxoWellcome

Erwinia L-asparaginase (Erwinase); Acute lymphocytic leukemia; Porton International

Erythropoietin (recombinant human); Anemia associated with end-stage renal disease; McDonnell Douglas

Etanercept (Enbrel); Reduction in signs and symptoms of moderately to severely active polyarticular-course juvenile rheumatoid arthritis where there has been inadequate response to ³ 1 disease-modifying anti-rheumatic drug;1 Wegener’s granulomatosis; Immunex

Ethanolamine oleate (Ethamolin); Esophageal varices that have recently bled, to prevent rebleeding1; Block Drug

Ethyl eicosapentaenoate; Huntington’s disease; Laxdale Ltd

Etidronate disodium (Didronel); Hypercalcemia of malignancy inadequately managed by dietary modification or oral hydration1; MGI Pharma

Etiocholanedione; Aplastic anemia; Prader-Willi syndrome; SuperGen

Exemestane (Aromasin); Advanced breast cancer in postmenopausal women whose disease have progressed following tamoxifen therapy1; Pharmacia & Upjohn

Exisulind; Suppression and control of colonic adenomatous polyps in the inherited disease adenomatous polyposis coli; Cell Pathways

Factor XIII concentrate (human) pasteurized (Fibrogammin P); Congenital Factor XIII deficiency; Aventis Behring LLC

Fampridine (Neurelan); Relief of symptoms of multiple sclerosis; chronic, incomplete spinal cord injury; Acorda Therapeutics

Felbamate (Felbatol); Lennox-Gastaut syndrome1; Wallace

FIAU; Adjunctive treatment of chronic active hepatitis B; Oclassen

Fibrinogen (human); Control of bleeding and prophylactic treatment of patients deficient in fibrinogen; Alpha Therapeutic

Fibronectin (human plasma derived); Nonhealing corneal ulcers or epithelial defects unresponsive to conventional therapy(underlying cause has been eliminated); Melville Biologics

Filgrastim (Neupogen); Severe chronic neutropenia (absolute neutrophil count < 500/mm3);1 neutropenia associated with bone marrow transplants;1 AIDS patients with CMV retinitis being treated with ganciclovir; mobilization of peripheral blood progenitor cells for collection in patients who will receive myeloablative or myelosuppressive chemotherapy;1 to reduce duration of neutropenia, fever, antibiotic use, and hospitalization following induction and consolidation treatment for acute myeloid leukemia1; Amgen

Flucinolone; Uveitis involving the posterior segment of the eye; Bausch & Lomb

Fludarabine phosphate (Fludara); Treatment/Management of non-Hodgkin’s lymphoma; chronic lymphocytic leukemia (CLL) including refractory CLL1; Berlex

Flunarizine (Sibelium); Alternating hemiplegia; Janssen Research Foundation

Fluorouracil; With interferon alpha-2a, recombinant, for advanced colorectal carcinoma and esophageal carcinoma; glioblastoma multiforme; Hoffmann-La Roche Ethypharm SA

(Adrucil); With leucovorin for metastatic adenocarcinoma of the colon and rectum; Lederle

Fluoxetine (Prozac); Autism; Eric Hollander, MD

Follitropin alfa, recombinant (Gonal-F); Induction of spermatogenesis in men with primary and secondary hypogonadotrophic hypogonadism in whom the cause of infertility is not caused byprimary testicular failure1; Serono Labs

Fomepizole (Antizole); Methanol or ethylene glycol poisoning1; Orphan Medical

Fosphenytoin (Cerebyx); Acute treatment of patients with status epilepticus of the grand mal type1; Warner-Lambert

Fructose-1,6-diphosphate (Cordox); For painful vaso-occlusive episodes associated with sickle cell disease; Questcor Pharm

Gabapentin (Neurontin); Amyotrophic lateral sclerosis; Warner-Lambert

Gallium nitrate injection (Ganite); Hypercalcemia of malignancy1; Solopak Pharm

Gamma hydroxybutyrate; Narcolepsy and auxiliary symptoms of cataplexy, sleep paralysis, hypnagogic hallucinations, and automatic behavior; Biocraft Labs

Gammalinolenic acid; Juvenile rheumatoid arthritis; Robert B. Zurier, MD

Ganaxolone; Infantile spasms; Purdue Pharma LP

Ganciclovir intravitreal implant (Vitrasert Implant); Cytomegalovirus retinitis1; Bausch & Lomb Surgical, Chiron Vision Products

Ganciclovir sodium (Cytovene); Cytomegalovirus retinitis in immunocompromised patients with AIDS1; Syntex (USA)

Gavilimomab; Acute graft-vs-host disease (aGVHD); Abgenix

Gemtuzumab ozogamicin (Mylotarg); CD33-positive acute myeloid leukemia1; Wyeth-Ayerst Labs

Gene plasmid hVEGF165 driven by human cytomegalovirus, and [2,3-bis(oleoyl)propyl]-trimethyl ammonium and dioleoyl phosphatidyl ethanolamine (Trinam); Prevention of complications caused by neointimal hyperplasia disease in certain vascular anastomoses; Ark Therapeutics

Gentamicin impregnated PMMA beads on surgical wire (Septopal); Chronic osteomyelitis of post-traumatic, postoperative, or hematogenous origin; Lipha Pharm

Gentamicin liposome injection (Maitec); Disseminated Mycobacterium avium-intracellulare infection; Liposome Co

Glatiramer acetate (Copaxone); Multiple sclerosis1; Teva Pharm USA

Glatiramer acetate for injection (Copaxone); Primary-progressive multiple sclerosis; Teva Pharm USA

Glutamine; With human growth hormone in treatment of short bowel syndrome (nutrient malabsorption from the GI tract resulting from an inadequate absorptive surface); Nutritional Restart

Glyceryl trioleate and glyceryl trierucate; Adrenoleukodystrophy; Hugo W. Moser, MD

Gonadorelin acetate (Lutrepulse); Ovulation induction in women with hypothalamic amenorrhea caused by a deficiency or absence in quantity or pulse pattern of endogenous GnRH secretion1; Ferring Labs

Gossypol; Cancer of the adrenal cortex; Marcus M. Reidenberg, MD

Gp100 adenoviral gene therapy; Metastatic melanoma; Genzyme

Growth hormone releasing factor; Long-term treatment of children who have growth failure caused by a lack of adequate endogenous growth hormone secretion; ICN Pharm

Guanfacine (Tenex); Fragile X syndrome; Watson Labs

h5G1.1-mAb; Dermatomyositis; idiopathic membranous glomerular nephropathy; Alexion Pharm

Halofantrine (Halfan); Mild to moderate acute malaria caused by susceptible strains of Plasmodium falciparum and P. vivax1; SmithKline Beecham

Halofuginone (Stenorol); Systemic sclerosis; Collgard Biopharmaceuticals Ltd

Heme arginate (Normosang); Symptomatic stage of acute porphyria; myelodysplastic syndromes; Berlex

Hemin (Panhematin); Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms that occur in other patients with AIP, porphyria variegata, and hereditary coproporphyria1; Abbott

Hemin and zinc mesoporphyrin (Hemex); Acute porphyric syndromes; Herbert L. Bonkovsky, MD

Hepatitis B immune globulin IV (human) (Nabi-HB); Prophylaxis against hepatitis B virus reinfection in liver transplant patients; NABI

Herpes simplex virus gene; Primary and metastatic brain tumors; Genetic Therapy

Histamine (Maxamine); Adjunct to cytokine therapy in acute myeloid leukemia treatment and malignant melanoma; Maxim Pharm

Histrelin; Treatment of acute intermittent porphyria, hereditary coproporphyria, and variegate porphyria; Karl E. Anderson, MD

Histrelin acetate (Supprelin Injection); Central precocious puberty1; Roberts Pharm

HLA-B7/Beta2M DNA Lipid (DMRIE/DOPE) Complex (Allovectin-7); Invasive and metastatic melanoma (Stages II, III, IV); Vical Incorporated

Hsp E7; Recurrent respiratory papillomatosis (RRP); StressGen Biotechnologies

Hu1D10, humanized, monoclonal antibody (Remitogen); 1D10+ B cell non-Hodgkin’s lymphoma; Protein Design Labs

Human acid precursor alpha-glucosidase, recombinant; Glycogen storage disease type II; Pharming/Genzyme LLC

Human anti-transforming growth factor beta 1 monoclonal antibody; Systemic sclerosis; Genzyme

Human gammaglobulin; Juvenile rheumatoid arthritis; Protein Therapeutics

Human T-lymphotropic virus type III Gp 160 antigens (Vaxsyn HIV-1); AIDS; MicroGeneSys

Humanized anti-CD2 monoclonal antibody; Graft-vs-host disease; MedImmune

Humanized anti-human CD2 MAb (MEDI-507); For induction of donor-specific immunologic unresponsiveness resulting in prophylaxis of organ rejection without the need for chronic immunosuppressive therapy in allogeneic renaltransplants; Biotransplant

Humanized anti-tac (Zenapax); Prevention of acute graft-vs-host disease following bone marrow transplantation; Hoffmann-La Roche

Humanized MAb (IDEC-131) to CD40L; Systemic lupus erythematosus; Idec Pharm

Humanized monoclonal antibody against Shiga-like toxin II; To prevent the development of or to decrease the incidence and severity of hemolytic uremic syndrome and associated sequelae of Shiga-like toxin-producing E. coli; Teijin America

Hydroxycobalamin (CYANOKIT); Acute cyanide poisoning; Orphan Medical

Hydroxyurea (Droxia); Treatment of patients with sickle cell anemia as shown by the presence of hemoglobin S1; Bristol-Myers Squibb Pharm Res Inst

Hypericin; Glioblastoma multiforme; cutaneous T-cell lymphoma; Nexell Therapeutics

Ibuprofen IV solution (Salprofen); Prevention/Treatment of patent ductus arteriosus; Farmacon-IL-LLC

Icodextrin 7.5% with electrolytesperitoneal dialysis solution (Extraneal [with 7.5% Icodextrin]Peritoneal Dialysis Solution); End-stage renal disease requiring peritoneal dialysis treatment; Baxter Healthcare

Idarubicin HCl for injection (Idamycin); Acute myelogenous leukemia (acute nonlymphocytic leukemia);1 acute lymphoblastic leukemia in pediatric patients; Adria Pharmacia & Upjohn

Idoxuridine; Nonparenchymatous sarcomas; NeoPharm

Iduronate-2-sulfatase; Long-term enzyme replacement therapy for MPS II (Hunter syndrome); Transkaryotic Therapies

Ifosfamide (Ifex); Testicular cancer;1 bone sarcomas; soft tissue sarcomas; Bristol-Myers Squibb Pharm Res Inst

IL-4 pseudomonas toxin fusion protein (IL-4(38-37)-PE38KDEL); Astrocytic glioma; Neurocrine Biosciences

IL-13-PE38QQR; Malignant glioma; NeoPharm

Imatinib mesylate (Gleevec); Chronic myelogenous leukemia;1 GI stromal tumors1; Novartis

Imciromab pentetate (Myoscint); Detecting early necrosis as an indication of rejection of orthotopic cardiac transplants; Centocor

Imexon; Multiple myeloma; metastatic malignant melanoma; AmpliMed

Imiglucerase (Cerezyme); Replacement therapy in patients with types I, II, and III Gaucher’s disease1; Genzyme

Immune globulin IV, human (Gamimune N); Infection prophylaxis in pediatric patients with HIV1; Bayer

Imported fire ant venom, allergenic extract; Skin testing for victims of fire ant stings to confirm fire ant sensitivity and, if positive, as immunotherapy for prevention of IgE-mediated anaphylactic reactions; ALK Labs

Indium-In-111 murine MAb(2B8-MX-DTPA) and Yttrium-Y90 murine MAb(2B8-MX-DTPA) (Melimmune); B-cell non-Hodgkin’s lymphoma; IDEC Pharm

Indium In 111 murine monoclonal antibody FAB to myosin (Myoscint); Aid in diagnosis of myocarditis; Centocor

Infliximab (Remicade); Moderately to severely active Crohn’s disease for the reduction of the signs and symptoms in inadequate response to conventional therapy and fistulizing Crohn’s disease for the reduction in the number of draining enterocutaneous fistula(s)1; Centocor

INH-A00021; Reduction (prevention) of nosocomial bacteria caused by staphylococci in very low birth weight infants; Inhibitex

Inosine pranobex (Isoprinosine); Subacute sclerosing panencephalitis; Newport Pharm

Interferon alfa-1b; Multiple myeloma; Ernest C. Borden

Interferon alfa-2a (Roferon A); Chronic myelogenous leukemia1; Hoffmann-La Roche

Interferon alfa-2a (recombinant) (Roferon-A); AIDS-related Kaposi’s sarcoma;1 renal-cell carcinoma; with fluorouracil for esophageal carcinoma or advanced colorectal cancer; with teceleukin for metastatic renal cell carcinoma or metastatic malignant melanoma; Hoffmann-La Roche

Interferon alfa-2b (recombinant) (Intron A); AIDS-related Kaposi’s sarcoma1; Schering

Interferon beta-1a (recombinant human) (Avonex); Primary brain tumors; juvenile rheumatoid arthritis; multiple sclerosis;1 pulmonary fibrosis; acute non-A, non-B hepatitis; Biogen

Interferon beta-1a (recombinant) (Rebif); Symptomatic patients with AIDS (including CD4 T-cell counts < 200 cells/mm3); secondary progressive multiple sclerosis; Serono

Interferon beta-1b (Betaseron); Multiple sclerosis1; Berlex Labs and Chiron

Interferon gamma-1b (Actimmune); Chronic granulomatous disease;1 renal cell carcinoma; delaying time to disease progression in patients with severe, malignant osteopetrosis1; Genentech InterMune Pharm

Interleukin-1 receptor antagonist (human recombinant) (Antril); Juvenile rheumatoid arthritis; prevention/treatment of graft-vs-host disease in transplantrecipients; Amgen

Interleukin-2

(Teceleukin); Alone or with interferon alfa-2a for metastatic renal-cell carcinoma; with interferon alfa-2a for metastatic malignant melanoma; Hoffmann-La Roche

(Teleleukin); Metastatic malignant melanoma; Hoffmann-La Roche

Intraoral fluoride releasing system (IFRS); Prevent dental caries caused by radiation-induced xerostomia in head and neck cancer; Digestive Care

Iobenguane sulfate I 131; Diagnostic adjunct in patients with pheochromocytoma1; University of Michigan

Iodine I123 murine monoclonal antibody to alpha-fetoprotein; Detects hepatocellular carcinoma and hepatoblastoma and alpha-fetoprotein-producinggerm-cell tumors; Immunomedics

Iodine I123 murine monoclonal antibody to hCG; Detection of hCG-producing tumors (eg, germ-cell, trophoblastic-cell tumors); Immunomedics

Iodine I131 6B-iodomethyl-19-nor-cholesterol; Adrenal cortical imaging; David E. Kuhl, MD

Iodine I131 bis(indium-diethylenetriamine-pentaacetic acid)tyrosyllysine/hMN-14x m734 F(ab')2 bispecific monoclonal antibody (Pentacea); Small-cell lung cancer; IBC Pharm

Iodine I131 murine monoclonal antibody IgG2a to B cell (Immurait, L1-2-I-131); B-cell leukemia and B-cell lymphoma; Immunomedics

Iodine I131 murine monoclonal antibody to alpha-fetoprotein; Hepatocellular carcinoma and hepatoblastoma; alpha-fetoprotein-producing germ-cell tumors; Immunomedics

Iodine I131 murine monoclonal antibody to hCG; hCG-producing tumors (eg, germ-cell, trophoblastic-cell tumors); Immunomedics

Iodine I-131 radiolabeled chimeric MAb tumor necrosis treatment(TNT-1B) (131IchTNT-1); Glioblastoma multiforme and anaplastic astrocytoma; Peregrine Pharm

Isobutyramide; Sickle cell disease and beta-thalassemia; Alpha Therapeutic

(Isobutyramide Oral Solution); Beta-hemoglobinopathies and beta-thalassemia syndromes; Susan P. Perrine, MD

Japanese encephalitis vaccine (live, attenuated); Prevention of Japanese encephalitis; Boran Pharm

L-2-oxothiazolidine-4-carboxylic acid (Procysteine); Adult respiratory distress syndrome (ARDS); amyotrophic lateral sclerosis; Transcend Therapeutics

L-5-hydroxytryptophan; Tetrahydrobiopterin deficiency; Watson Labs

L-baclofen (Neuralgon); Trigeminal neuralgia; intractable spasticity from spinal cord injury or multiple sclerosis; intractable spasticity in children with cerebral palsy; Gerhard Fromm, MD Pharmascience

L-cycloserine; Gaucher’s disease; Meir Lev, MD

L-cysteine; Prevention and lessening of photosensitivity in erythropoietic protoporphyria; Orphan Pharm USA

L-glutamine; Sickle cell disease; Orphan Drugs International, LLC

L-glutamyl-L-tryptophan; AIDS-related Kaposi’s sarcoma; Cytran

L-leucovorin (Isovorin); With high-dose methotrexate in the treatment of osteosarcoma; in combination chemotherapy with the approved agent 5-fluorouracil in the palliative treatment of metastatic adenocarcinoma of the colon and rectum; Lederle

L-threonine (Threostat); Amyotrophic lateral sclerosis; Tyson & Assoc

Lactic acid (Aphthaid); Severe aphthous stomatitis in severely, terminally immunocompromised patients; Frontier Pharm

Lactic acid bacteria (Lactobacilli, Bifidobacteria, and Streptococcus species); Chronic active pouchitis; prevention of disease relapse in patients with chronic pouchitis; VSL Pharm

Lamotrigine (Lamictal); Lennox-Gastaut syndrome1; GlaxoWellcome Res & Devel

Lanreotide, Somatostatin (Ipstyl); Acromegaly; IPSEN

Latrodectus immune F(ab)2 (Aracmyn); Black widow spider envenomations; Rare Disease Therapeutics

Leflunomide; Prevention of acute and chronic rejection in patients with solid organ transplants; James W. Williams, MD

Lepirudin (Refluden); Heparin-associated thrombocytopenia type II1; Hoechst Marion Roussel

Leucovorin (Leucovorin calcium); With 5-fluorouracil for metastatic colorectal cancer;1 rescue use after high-dose methotrexate therapy in the treatment of osteosarcoma1; Immunex

Leupeptin; As an adjunct to microsurgical peripheral nerve repair; Neuromuscular Adjuncts

Leuprolide acetate (Lupron Injection); Central precocious puberty1; Tap Pharm

Levocarnitine (Carnitor); Genetic carnitine deficiency;1 primary and secondary carnitine deficiency of genetic origin;1 prevention/treatment of secondary carnitine deficiency in valproic acid toxicity; pediatric cardiomyopathy; treatment of zidovudine-induced mitochondrial myopathy; manifestations of carnitine deficiency in patients with end-stage renal disease who require dialysis1; Sigma-Tau Pharm

Levodopa and carbidopa (Duodopa); Late-stage Parkinson’s disease; Nouvel Pharma

Levomethadyl acetate HCl (Orlaam); Heroin addiction suitable for maintenance on opiate agonists1; Biodevelopment

Lidocaine patch 5% (Lidoderm Patch); For relief of allodynia (painful hypersensitivity) and chronic pain in postherpetic neuralgia1; Teikoku Pharma USA

Liothyronine sodium injection (Triostat); Myxedema coma/precoma1; SmithKline Beecham

Lipase, amylase, and protease (TheraCLEC-Total); Pancreatic insufficiency; Altus Biologics

Lipid/DNA human cystic fibrosis gene; Cystic fibrosis; Genzyme

Liposomal amphotericin B (AmBisome); Cryptococcal meningitis;1 visceral leishmaniasis;1 histoplasmosis; Fujisawa USA

Liposomal-cis-bis-neodecanoato-trans-R,R-1,2-diaminocyclohexane-Pt (II); Malignant mesothelioma; Antigenics

Liposomal cyclosporin A (Cyclospire); For aerosolized administration in the prevention and treatment of lung allograft rejection and pulmonary rejection events associated with bone marrow transplantation; Vernon Knight, MD

Liposomal N-Acetylglucosminyl-N-Acetylmuramyl-L-Ala-D-isoGln-L-Ala-glycerolidpalmitoyl (ImmTher); Osteosarcoma; Ewing’s sarcoma; Endorex

Liposomal nystatin (Nyotran); Invasive fungal infections; Aronex Pharm

Liposome encapsulated recombinant interleukin-2; Brain and CNS tumors; kidney and renal pelvis cancers; Biomira USA

Lisofylline; Treatment for patients undergoing induction therapy for acute myeloid leukemia; Cell Therapeutics

Lodoxamide tromethamine (Alomide Ophthalmic Solution); Vernal keratoconjunctivitis1; Alcon

Lucinactant (Surfaxin); Meconium aspiration syndrome in newborn infants; respiratory distress syndrome in premature infants; acute respiratory distress in adults; Discovery Labs

Mafenide acetate solution (Sulfamylon solution); Adjunctive topical antimicrobial agent to control bacterial infection when used under moist dressings over meshed autografts on excised burn wounds1; Mylan

Marijuana; HIV-associated wasting syndrome; Multidisciplinary Assoc for Psychedelic Studies

MART-1 adenoviral gene therapy for malignant melanoma; Metastatic melanoma; Genzyme

Matrix metalloproteinase inhibitor (Galardin); Corneal ulcers; Glycomed

Mazindol (Sanorex); Duchenne muscular dystrophy; Platon J. Collipp, MD

Mecamylamine (Inversine); Tourette’s syndrome; Layton Bioscience

Mecasermin (Myotrophin); Growth hormone insufficency syndrome; amyotrophic lateral sclerosis; Genentech Cephalon

Medroxyprogesterone acetate (Hematrol); Immune thrombocytopenic purpura; InKine Pharm

Mefloquine HCl

(Lariam); Acute malaria caused by Plasmodium falciparum and P. vivax;1 prophylaxis of P. falciparum malaria resistant to other available drugs1; Hoffman-La Roche

(Mephaquin); To prevent/treat chloroquine-resistant Falciparum malaria; Mepha AG

Megestrol acetate (Megace); Anorexia, cachexia, or significant weight loss (³ 10% of baseline body weight) with confirmed diagnosis of AIDS1; Bristol-Myers Squibb Pharm Res Inst

Melanoma cell vaccine; Invasive melanoma; Donald L. Morton, MD

Melanoma vaccine (Melacine); Stage III-IV melanoma; Ribi ImmunoChem Res

Melatonin; Circadian rhythm sleep disorders in blind people with no light perception; Robert Sack, MD

Meropenem (Merrem IV); Management of acute pulmonary exacerbations in cystic fibrosis patients caused byrespiratory tract infections with susceptible organisms; Zeneca

Mesna (Mesnex); Prophylactic to reduce the incidence of ifosfamide-induced hemorrhagic cystitis;1 inhibition of the urotoxic effects induced by oxazaphosphorine compounds (eg, cyclophosphamide); Asta Medica Degussa

Methionine/ L-methionine; AIDS myelopathy; Alessandro Di Rocco, MD

Methotrexate sodium (Methotrexate); Osteogenic sarcoma1; Lederle

Methotrexate with laurocapram (Methotrexate/Azone); Topical treatment of Mycosis fungoides; Durham Pharm

Methoxsalen (Uvadex); Used in conjunction with the UVAR photopheresis system to treat graft-vs-host disease; Therakos

Metreleptin; Metabolic disorders secondary to lipodystrophy; leptin deficiency secondary to generalized lipodystrophy and partial familial lipodystrophy; Amgen

Metronidazole (Metrogel); Perioral dermatitis; Galderma

Metronidazole (topical) (Metrogel); Acne rosacea1; Galderma

Microbubble contrast agent (Filmix Neurosonographic Contrast Agent); Intraoperative aid in the identification and localization of intracranial tumors; Cav-Con

Midodrine HCl (Amatine); Symptomatic orthostatic hypotension1; Schier Ridgewood FKA

Mitoguazone (apep); Diffuse Non-Hodgkin’s lymphoma including AIDS-related diffuse Non-Hodgkin’s lymphoma; ILEX Oncology

Mitolactol; Adjuvant therapy in the treatment of primary brain tumors; recurrent invasive or metastatic squamous carcinoma of the cervix; Biopharmaceutics

Mitomycin-C; Refractory glaucoma as an adjunct to ab externo glaucoma surgery; IOP

Mitoxantrone (Novantrone); Hormone refractory prostate cancer;1 progressive-relapsing multiple sclerosis;1 secondary-progressive multiple sclerosis1; Immunex

Mitoxantrone HCl (Novantrone); Acute myelogenous leukemia (acute nonlymphocytic leukemia)1; Lederle

MN14 monoclonal antibody to carcinoembryonic antigen (Cea-Cide); Small-cell lung cancer; pancreatic cancer; Immunomedics

Modafinil (Provigil); Excessive daytime sleepiness in narcolepsy1; Cephalon

Monoclonal antibodies PM-81 and AML-2-23; Exogenous depletion of CD14 and CD15 positive acute myeloid leukemic bone marrow cells from patients undergoing bone marrow transplantation; Medarex

Monoclonal antibody for immunization against lupus nephritis; Lupus nephritis; VivoRx Autoimmune

Monoclonal antibody PM-81; Adjunctive treatment of acute myelogenous leukemia; Medarex

Monoclonal antibody-B43.13 (Ovarex MAb-B43.13); Epithelial ovarian cancer; AltaRex US

Monolaurin (Glylorin); Congenital primary ichthyosis; GlaxoWellcome

Monooctanoin (Moctanin); Dissolution of cholesterol gallstones retained in the common bile duct1; Ethitek Pharm

Morphine sulfate concentrate (preservative free) (Infumorph); For use in microinfusion devices for intraspinal administration for intractable chronic pain1; Elkins-Sinn

MTC-DOX for injection; Hepatocellular carcinoma; FeRx

Multi-vitamin infusion (neonatalformula); To establish/maintain total parenteral nutrition in very low birth weight infants; Astra Pharm

Muramyltripeptide, phosphatidyl-ethanolamine encased in multi-lamellar liposomes; Children and adolescent osteosarcoma; Jenner Biotherapeutics

Murine MAb (Lym-1) and Iodine 131-I radiolabeled murine MAb (Lym-1) to human B-cell lymphoma (Oncolym); B-cell non-Hodgkin’s lymphoma; Berlex Labs

Murine MAb to polymorphic epithelial mucin, human milk fat globule 1 (Theragyn); Adjuvant treatment of ovarian cancer; Antisoma

Mycobacterium avium sensitin RS-10; For use in diagnosis of invasive Mycobacterium avium disease in immunocompetentindividuals; Statens Seruminstitut

N-acetylcysteinate (Nacystelyn Dry Powder Inhaler); Management of cystic fibrosis; Galephar Pharm Res

N-acetyl-procainamide; To prevent life-threatening ventricular arrhythmias in documented procainamide-induced lupus; NAPA of the Bahamas

N-acetylgalactosamine-4-sulfatase, recombinant human; Mucopolysaccharidosis Type VI (Maroteaux-Lamy syndrome); BioMarin Pharm

Nafarelin acetate (Synarel Nasal Solution); Central precocious puberty; Syntex (USA)

Naltrexone HCl (Trexan); Blockade pharmacological effects of exogenous opioids as an adjunct to maintain opioid-free state in detoxified, formerly opioid-dependent individuals1; DuPont Pharm

Natural human lymphoblastoid interferon-alpha; Papillomavirus warts in the oral cavity of HIV positive patients; Behcet’s disease; Amarillo Biosciences

NDROGE; Postanoxic intention myoclonus; Watson Labs

Nebacumab (Centoxin); Gram-negative bacteremia that has progressed to endotoxin shock; Centocor

Neurotrophin-1; Motor neuron disease/amyotrophic lateral sclerosis; Arthur Dale Ericsson, MD

NG-29 (Somatrel); Diagnostic measure of the capacity of the pituitary gland to release growth hormone; Ferring Labs

Nifedipine; Interstitial cystitis; Jonathan Fleischmann, MD

Nitazoxanide (Cryptaz); Cryptosporidiosis; intestinal amebiasis; Romark Labs

Nitisinone (Orfadin); Tyrosinemia type 1;1 alkaptonuria; Swedish Orphan AB

Nitric oxide (INOmax); Persistent pulmonary hypertension in the newborn;1 acute respiratory distress syndrome in adults; INO Therapeutics

Nitroprusside; To treat/prevent cerebral vasospasm following subarachnoid hemorrhage; Jeffrey Evan Thomas, MD

Nolatrexed (THYMITAQ); Hepatocellular carcinoma; Zarix

Novel acting thrombolytic (NAT); Peripheral arterial occlusion (PAO); Amgen

NZ-1002; Enzyme replacement therapy in patients with all subtypes of mucopolysaccharidosis I; Novazyme

Octreotide (Sandostatin LAR); Acromegaly;1 severe diarrhea and flushing associated with malignant carcinoid tumors;1 diarrhea associated with vasoactive intestinal peptide tumors (VIPoma)1; Novartis

Ofloxacin (Ocuflox Ophthalmic Solution); Bacterial corneal ulcers1; Allergan

Oglufanide disodium; Ovarian cancer; Cytran

OM 401 (Drepanol); Prophylactic treatment of sickle cell disease; Omex International

Omega-3 (n-3) polyunsaturatedfatty acid (with all double bonds in the cis configuration); Prevention of organ graft rejection; Research Triangle

Omega-3 (n-3) polyunsaturatedfatty acids (Omacor); IgA nephropathy; Pronova Biocare, AS

Oncorad Ov103; Ovarian cancer; Cytogen

Oprelvekin (Neumega); Prevention of severe chemotherapy-induced thrombocytopenia1; Genetics Institute

Oxaliplatin; Ovarian cancer; Debio Pharm SA

Oxandrolone (Oxandrin); Treatment of patients with Duchenne’s and Becker’s muscular dystrophy; adjunctive therapy for AIDS patients with HIV-wasting syndrome; Bio-Technology General

Oxybate (Xyrem); Narcolepsy; Orphan Medical

Oxymorphone (Numorphan H.P.); Relief of severe intractable pain in narcotic-tolerant patients; DuPont Merck

Oxypurinol; Hyperuricemia in patients intolerant to allopurinol; ILEX Oncology

p1-(uridine 5’-)-p4-(2’-deoxycytidine 5’-) tetraphosphate, tetrasodium salt; Cystic fibrosis; Inspire Pharm

Paclitaxel (Taxol)(Paxene); AIDS-related Kaposi’s sarcoma1; Bristol-Myers Squibb Pharm Res Inst Baker Norton Pharm

Papain, trypsin, and chymotrypsin (Wobe-Mugos); Multiple myeloma; Marlyn Nutraceuticals

Parvovirus B19 (recombinant VP1 and VP2; S. frugiperda cells) vaccine (MEDI-491); Prevention of transient aplastic crisis in patients with sickle cell anemia; MedImmune

Patul-end; Patulous eustachian tube; Ear Foundation

Pegademase bovine (Adagen); Enzyme replacement for ADA deficiency in patients with severe combined immunodeficiency1; Enzon

Pegaspargase (Oncaspar); Acute lymphocytic leukemia1; Enzon

PEG-glucocerebrosidase (Lysodase); Chronic enzyme replacement therapy in Gaucher’s disease patients who are deficient inglucocerebrosidase; National Institute ofMental Health, NIH

Peginterferon alfa-2a (PEGASYS); Renal cell carcinoma; chronic myelogenous leukemia; Hoffmann-La Roche

PEG-interleukin-2; Primary immunodeficiencies associated with T-cell defects; Chiron

Pegvisomant (Somavert); Acromegaly; Sensus

Pegylated arginine deiminase (Hepacid)(Melanocid); Hepatocellular carcinoma; invasive malignant melanoma; Phoenix Pharmacologics

Pegylated recombinant human megakaryocyte growth and development factor (MEGAGEN); To reduce the period of thrombocytopenia in patients undergoing hematopoietic stem celltransplantation; Amgen

Peldesine; Cutaneous T-cell lymphoma; BioCryst Pharm

Pemetrexed disodium (Alimta); Malignant pleural mesothelioma; Eli Lilly

Pentamidine isethionate; Treatment of Pneumocystis carinii pneumonia (PCP); Aventis Behring L.L.C.

(Nebupent); PCP prevention in high-risk patients1; Fujisawa USA

(Pentam 300); Treatment of PCP1; Fujisawa USA

Pentamidine isethionate (inhalation) (Pneumopent); Pneumocystis carinii pneumonia prevention in high-risk patients; Fisons

Pentastarch (Pentaspan); Adjunct in leukapheresis to improve the harvesting and increase the yield of leukocytes bycentrifugal means1; DuPont

Pentosan polysulphate sodium (Elmiron); Interstitial cystitis1; Alza

Pentostatin (Nipent); Chronic lymphocytic leukemia; cutaneous T-cell lymphoma; peripheral T-cell lymphomas; SuperGen

Pentostatin for injection (Nipent); Hairy-cell leukemia1; SuperGen

Perflubron (LiquiVent); Acute respiratory distress disease (ARDS) in adults; Alliance Pharm

Pergolide (Permax); Tourette’s syndrome; Floyd R. Sallee, MD, PhD

Phenylacetate; Adjunct to surgery, radiation therapy, and chemotherapy for the treatment of patients withprimary or recurrent malignant glioma; Elan Pharm Res

Phenylalanine ammonia-lyase (Phenylase); Hyperphenylalaninemia; Ibex Technologies

Phenylbutyrate; Acute promyelocytic leukemia; Elan

Phosphocysteamine; Cystinosis; Medea Research

Physostigmine salicylate (Antilirium); Friedreich’s and other inherited ataxias; Forest Pharm

Pilocarpine HCl (Salagen); Xerostomia induced by radiation therapy for head and neck cancer;1 xerostomia and keratoconjunctivitis sicca in Sjogren’s syndrome1; MGI Pharma

Piracetam (Nootropil); Myoclonus; UCB Pharma

Polifeprosan 20 with carmustine (Gliadel); Malignant glioma1; Guilford Pharm

Poloxamer 188 (Flocor); Vasospasm in subarachnoid hemorrhage patients following surgical repair of a rupturedcerebral aneurysm; sickle cell crisis; severe burns requiring hospitalization; CytRx

Poloxamer 331 (Protox); Initial therapy of toxoplasmosis in AIDS patients; CytRx

Poly I: poly C12U (Ampligen); AIDS; renal cell carcinoma; chronic fatigue syndrome; invasive metastatic melanoma(Stage IIB, III, IV); Hemispherx Biopharma

Poly-ICLC; Primary brain tumors; Andres M. Salazar, MD and Hilton B. Levy, PhD

Polyethylene glycol (PEG)-uricase; To control the clinical consequences of hyperuricemia in patients with severe gout in whom conventional therapy is contraindicated or has been ineffective; Bio-Technology General

Polyethylene glycol-modified uricase (Zurase); Tumor lysis syndrome in cancer patients undergoing chemotherapy; prophylaxis of hyperuricemia in cancer patients prone to develop tumor lysis syndrome during chemotherapy; Phoenix Pharmacologics

Polymeric oxygen; Sickle cell anemia; Capmed USA

Porcine fetal neural dopaminergic cells or precursors aseptically prepared and coated with anti-MHC-1 Ab for intracerebral implantation (NeuroCell-PD); Hoehn and Yahr stage 4 and 5 Parkinson’s disease; Diacrin

Porcine fetal neural dopaminergic cells or precursors aseptically prepared for intracerebral implantation (NeuroCell-PD); Hoehn and Yahr stage 4 and 5 Parkinson’s disease; Diacrin

Porcine fetal neural gabaergic cells or precursors aseptically prepared and coated with anti-MHC-1 Ab for intracerebral implantation (NeuroCell-HD); Huntington’s disease; Diacrin

Porcine fetal neural gabaergic cells or precursors aseptically prepared for intracerebral implantation for Huntington’s disease (NeuroCell-HD); Huntington’s disease; Diacrin

Porcine Sertoli cells (aseptically prepared for intracerebral co-implantation with fetal neural tissue) (N-Graft); Hoehn and Yahr stage 4 and 5 Parkinson’s disease; Titan Pharm

Porfimer (Photofrin); Ablation of high-grade dysplasia in Barrett’s esophagus in patients who are not considered to be candidates for esophagectomy; Axcan Scandipharm

Porfimer sodium (Photofrin); Photodynamic therapy of patients with primary or recurrent obstructing (partially or completely) esophageal carcinoma;1 photodynamic therapy of patients with transitional cell carcinoma in situ of the urinary bladder; QLT Phototherapeutics

Porfiromycin (Promycin); Head, neck, and cervical cancer; Boehringer Ingelheim

Potassium citrate (Urocit-K); Prevention of uric acid nephrolithiasis;1 prevention of calcium renal stones in patients with hypocitraturia;1 avoidance of the complication of calcium stone formation in uric lithiasis1; Univ of Texas Health Science Center at Dallas

Primaquine phosphate; With clindamycin HCl in the treatment of Pneumocystis carinii pneumonia associated with AIDS; Sanofi Winthrop

Progesterone; Establishment and maintenance of pregnancy in women undergoing in vitro fertilization or embryo transfer procedures; Watson Labs

Prostaglandin E1 enol ester (AS-013); Fontaine Stage IV chronic, critical limb ischemia; Welfide International

Protein C concentrate

(Protein C Concentrate [human] Vapor Heated, Immuno); Replacement therapy in patients with congenital or acquired protein C deficiency for the prevention/treatment of warfarin-induced skin necrosis during oral anticoagulation; Immuno Clinical Research

(Protein C Concentrate [human] Vapor Heated, Immuno); Replacement therapy in congenital protein C deficiency for the prevention/treatment of the following: thrombosis, pulmonary emboli, and purpura fulminans; Baxter Healthcare

Pulmonary surfactant replacement, porcine (Curosurf); Treatment/Prevention of respiratory distress syndrome in premature infants; Dey Labs

Purified extract of Pseudomonas aeruginosa (ImmuDyn); Immune thrombocytopenic purpura where it is required to increase platelet counts; DynaGen

Purified type II collagen (Colloral); Juvenile rheumatoid arthritis; AutoImmune

pVGI.1(VEGF2); Thromboangiitis obliterans; Vascular Genetics

Pyruvate; Interstitial lung disease; Cellular Sciences

RII Retinamide; Myelodysplastic syndromes; Sparta Pharm

(R)-N-[2-(6-chloro-5-methoxy-1H-indol-3-yl)propyl]acetamide; Circadian rhythm sleep disorders in blind people with no light perception; Phase 2 Discovery

Radiolabeled monoclonal antibody to CD22 antigen on B-cells (LymphoCIDE); Non-Hodgkin’s lymphoma; Immunomedics

Recombinant bactericidal/permeability-increasing protein (Neuprex); Severe meningococcal disease; Xoma

Recombinant glycine2-human glucagon-like peptide-2; Short bowel syndrome; NPS Allelix

Recombinant human acid alpha-glucosidase; Glycogen storage disease type II; Genzyme

Recombinant human alpha-1 antitrypsin (rAAT); Cystic fibrosis; to delay progression of chronic obstructive pulmonary disease resulting from AAT deficiency-mediated emphysema and bronchiectasis; Arriva Pharm Baxter Healthcare PPL Therapeutics (Scotland) Ltd.

Recombinant human alpha-fetoprotein (rhAFP); Myasthenia gravis; Atlantic Biopharmaceuticals

Recombinant human antithrombin III; Antithrombin III dependent heparin resistance requiring anticoagulation; AT III LLC

Recombinant human C1-esterase inhibitor; Prophylactic treatment of angioedema caused by hereditary or acquired C1-esterase inhibitor deficiency; treatment of acute attacks of angioedema caused by hereditary or acquired C1-esterase inhibitor deficiency; Pharming NV

Recombinant human CD4 immunoglobulin G; AIDS resulting from HIV-1 infection; Genentech

Recombinant human Clara Cell 10kDa protein; Prevention of neonatal bronchopulmonary dysplasia in premature neonates with respiratory distress syndrome; Claragen

Recombinant human endostatin protein; Neuroendocrine tumors; EntreMed

Recombinant human gelsolin; Respiratory symptoms of cystic fibrosis; acute and chronic respiratory symptoms of bronchiectasis; Biogen

Recombinant human highly phosphorylated acid alpha-glucosidase; For enzyme replacement therapy in patients with all subtypes of glycogen storage disease type II (GSDII, Pompe disease); Novazyme Pharm

Recombinant human insulin-like growth factor-I; Postpoliomyelitis syndrome; Cephalon

Recombinant human insulin-like growth factor-I (PV802); Short-bowel syndrome as a result of resection of the small bowel or as a result of congenital dysfunction of the intestines; GroPep Pty Ltd

Recombinant human insulin-like growth factor-I/insulin-like growth factor binding protein-3; Major burns that require hospitalization; Celtrix Pharm

Recombinant human interleukin-12; Renal cell carcinoma; Genetics Institute

Recombinant human keratinocyte growth factor; To reduce the incidence and severity of radiation-induced xerostomia; Amgen

Recombinant human luteinizinghormone (LHadi); With recombinant human follicle stimulating hormone for women with chronic anovulation caused by hypogonadotropic hypogonadism; Serono Labs

Recombinant human nerve growth factor; HIV-associated sensory neuropathy; Genentech

Recombinant human relaxin; Progressive systemic sclerosis; Connetics

Recombinant human superoxidedismutase (Oxsodrol); Prevention of bronchopulmonary dysplasia in premature neonates weighing < 1500 g; Bio-Technology General

Recombinant human thrombopoietin; Used for accelerating platelet recovery in patients undergoing hematopoietic stem-celltransplantation; Genentech

Recombinant humanized MAb 5c8; Prevention and treatment of Factor VIII/Factor IX inhibitors in hemophilia A or B; prevention of rejection of solid organ transplants; prevention of rejection of pancreatic islet cell transplants; immune thrombocytopenic purpura; systemic lupus erythematosus; Biogen

Recombinant methionyl brain-derived neurotrophic factor; Amyotrophic lateral sclerosis; Amgen

Recombinant methionyl human stem cell factor; Primary bone marrow failure; Amgen

Recombinant retroviral vector -glucocerebrosidase; Enzyme replacement therapy for types I, II, or III Gaucher disease; Genetic Therapy

Recombinant secretory leucocyte protease inhibitor; Congenital alpha-1 antitrypsin deficiency; cystic fibrosis; Amgen

Recombinant soluble human CD4 (rCD4); AIDS in patients infected with HIV virus; Genentech

Recombinant urate oxidase; Treatment/Prophylaxis of chemotherapy-induced hyperuricemia; treatment of malignancy-associated hyperuricemia; Sanofi-Synthelabo Research

Recombinant vaccinia (humanpapillomavirus) (TA-HPV); Cervical cancer; Cantab Pharm Res Limited

Reduced L-glutathione (Cachexon); AIDS-associated cachexia; Telluride Pharm

Remacemide (Ecovia); Huntington’s disease; AstraZeneca LP

Respiratory syncytial virus (RSV) immune globulin (human)

(Hypermune RSV); Treatment of RSV lower respiratory tract infections in hospitalized infants and young children; MedImmune

(Respigam); Prophylaxis of RSV lower respiratory tract infections in infants and young children at high risk of RSV disease1; MedImmune and MA Public Health Biologics

Retroviral vector, R-GC and GC gene 1750; Gaucher disease; Genzyme

Reviparin sodium (Clivarine); Deep vein thrombosis that may lead to pulmonary embolism in pediatric patients; long-term treatment of acute deep vein thrombosis with or without pulmonary embolism in pregnant patients; Knoll AG

RGG0853, E1A lipid complex; Ovarian cancer; Targeted Genetics

Rho (D) immune globulin IV (human) (WinRho SD); Immune thrombocytopenic purpura1; Rh Pharmaceuticals

Ribavirin (Virazole); Hemorrhagic fever with renal syndrome; ICN Pharm

Ricin (blocked) conjugated murine MCA (anti-B4); B-cell leukemia and B-cell lymphoma; ex vivo purging of leukemic cells from the bone marrow of non-T-cell acute lymphocytic leukemia patients who are in complete remission; ImmunoGen

Ricin (blocked) conjugated murine MCA (anti-my9); Myeloid leukemia, including acute myelogenous leukemia, and blast crisis of chronic myeloid leukemia; ex vivo treatment of autologous bone marrow and subsequent reinfusion in acute myelogenous leukemia; ImmunoGen

Ricin (blocked) conjugated murine MCA (n901); Small-cell lung cancer; ImmunoGen

Ricin (blocked) conjugated murine monoclonal antibody (CD6); Cutaneous T-cell lymphomas, acute T-cell leukemia-lymphoma, and related mature T-cellmalignancies; ImmunoGen

Rifabutin; Treatment of disseminated Mycobacterium avium complex (MAC) disease; Pharmacia & Upjohn

(Mycobutin); Prevention of disseminated MAC disease in advanced HIV infection1; Adria

Rifalazil; Pulmonary tuberculosis; PathoGenesis

Rifampin (Rifadin IV); Antituberculosis treatment when oral doseform is not feasible1; Hoechst Marion Roussel

Rifampin, isoniazid, pyrazinamide (Rifater); Short-course treatment of tuberculosis1; Hoechst Marion Roussel

Rifapentine (Priftin); Pulmonary tuberculosis;1 treatment of Mycobacterium avium (MAC) complex in patients with AIDS; prophylaxis of MAC in patients with AIDS and a CD4+ count £ 75/mm3; Hoechst Marion Roussel

Rifaximin (Normix); Hepatic encephalopathy; Salix Pharm

Riluzole (Rilutek); Amyotrophic lateral sclerosis;1 Huntington’s disease; Rhone-Poulenc Rorer

Rituximab (Rituxan); Non-Hodgkin’s B-cell lymphoma1; IDEC Pharm

r-SP-C lung surfactant (Venticute); Adult respiratory distress syndrome; Byk Gulden Pharm

Sacrosidase (Sucraid); Congenital sucrase-isomaltase deficiency1; Orphan Medical

S-adenosylmethionine; AIDS-myelopathy; Alessandro Di Rocco, MD

Sargramostim (Leukine); Neutropenia associated with bone marrow transplant, graft failure, and delay of engraftment and for promotion of early engraftment;1 reduce neutropenia and leukopenia and decrease the incidence of death caused byinfection in patients with acute myelogenous leukemia1; Immunex

Satumomab pendetide (OncoScint CR/OV); Detection of ovarian carcinoma1; Cytogen

SB-408075; Pancreatic cancer; SmithKline Beecham

SCH 58500; Primary ovarian cancer; Schering

Secalciferol (Osteo-D); Familial hypophosphatemic rickets; Teva Pharm USA

Secretory leukocyte proteaseinhibitor; Bronchopulmonary dysplasia; Synergen

Selegiline HCl (Eldepryl); Adjuvant to levodopa/carbidopa in idiopathic Parkinson’s disease (paralysis agitans), postencephalitic Parkinsonism and symptomatic Parkinsonism1; Somerset

Sermorelin acetate (Geref); Idiopathic or organic growth hormone deficiency in children with growth failure;1 adjunct to gonadotropin in ovulation induction in anovulatory or oligo-ovulatory infertility after failure of clomiphene citrate or gonadotropin alone; AIDS-associated catabolism/weight loss; Serono

Serratia marcescens extract(polyribosomes) (Imuvert); Primary brain malignancies; Cell Technology

Short chain fatty acid enema (Colomed); Chronic radiation proctitis; Richard I. Breuer, MD

Short chain fatty acid solution (Colomed); Active phase of ulcerative colitis with involvement restricted to the left side of the colon; Richard I. Breuer, MD

Silver sulfadiazine and cerium nitrate (Flammacerium); Prevention of mortality in severly burned patients; Synthes (USA)

Sodium 1,3-propanedisulfonate; Secondary amyloidosis; Neurochem

Sodium dichloroacetate; Congenital lactic acidosis; lactic acidosis in severe malaria; homozygous familial hyper-cholesterolemia; Peter W. Stacpoole, PhD, MD Questcor Pharm

(Ceresine); Severe head injury; Questcor Pharm

Sodium monomercaptoundecahydro-closo-dodecaborate (Borocell); For use in boron neutron capture therapy in glioblastoma multiforme; Neutron Technology & Neutron R & D Partner

Sodium phenylbutyrate; Sickling disorders including S-S, S-C, and S-thalassemia hemiglobinopathy; adjunct to surgery, radiation therapy, and chemotherapy for primary or recurrent malignant glioma; Medicis Pharm Elan Pharm Res

(Buphenyl); The following urea cycle disorders: Carbamylphosphate synthetase deficiency, ornithine transcarbamylase deficiency, arginiosuccinic acid synthetase deficiency1; Medicis

Sodium tetradecyl sulfate (Sotradecol); Bleeding esophageal varices; Elkins-Sinn

Soluble complement receptor type 1; Prevention of postcardiopulmonary bypass syndrome in children undergoing cardiopulmonary bypass; Avant Immunotherapeu-tics

Soluble recombinant human complement receptor type 1; Prevention/Reduction of adult respiratory distress syndrome; T Cell Sciences

Somatostatin; Bleeding esophageal varices; UCB Pharm

(Zecnil); Adjunct to nonoperative management of secreting cutaneous fistulas of the stomach, duodenum, small intestine (jejunum and ileum), or pancreas; Ferring Labs

Somatrem for injection (Protropin); Long-term treatment of children with growth failure caused bylack of adequate endogenous growth hormone secretion;1 short stature associated with Turner’s syndrome; Genentech

Somatropin

(Genotropin); Adults with growth hormone deficiency1; Pharmacia & Upjohn

(Humatrope); Short stature associated with Turner’s syndrome;1 long-term treatment of children with growth failure caused by inadequate secretion of normal endogenous growth hormone1; Eli Lilly

(Nutropin); Long-term treatment of children with growth failure caused by lack of adequate endogenous growth hormone secretion;1 short stature in Turner’s syndrome;1 growth retardation in chronic renal failure;1 replacement therapy for growth hormone deficiency in adults after epiphyseal closure1; Genentech

(Serostim); Catabolism/weight loss in AIDS1; Serono

Somatropin (rDNA)

(Genotropin); Growth failure in children who were born small for gestational age;1 short stature in patients with Prader-Willi syndrome1; Pharmacia & Upjohn

(Neutropin Depot); Long-term treatment of children who have growth failure caused by a lack of adequate endogenous growth hormone secretion; Genentech

(Norditropin); Growth failure in children caused by inadequate growth hormone secretion; Novo Nordisk

(Saizen); Enhancement of nitrogen retention in hospitalized patients suffering from severe burns; idiopathic or organic growth hormone deficiency in children with growth failure; Serono

(Serostim); Alone or with glutamine in short bowel syndrome; treatment of children with AIDS-associated failure-to-thrive, including AIDS-associated wasting; Serono

Sotalol HCl (Betapace); Treatment of life-threatening ventricular tachyarrhythmias1; Berlex

Squalamine lactate; Ovarian cancer refractory or resistant to standard chemotherapy; Genaera

SS1-PE38; Mesothelioma; NeoPharm

SU101; Malignant glioma; ovarian cancer; Sugen

Succimer

(Chemet); Prevention of cystine kidney stones in patients with homozygous cystinuria who are prone to stone development; mercury intoxication; Sanofi Winthrop

(Chemet capsules); Lead poisoning in children1; Bock Pharmacal

Sucralfate suspension; Oral complications of chemotherapy in bone marrow transplants; oral ulcerations and dysphagia in epidermolysis bullosa; Darby Pharm

Sulfadiazine; Used with pyrimethamine for Toxoplasma gondii encephalitis in patients with and without AIDS1; Eon Labs

Sulfapyridine; Dermatitis herpetiformis; Jacobus Pharm

Superoxide dismutase (human); Protection of donor organ tissue from damage or injury mediated by oxygen-derived free radicals that are generated during the necessary periods of ischemia (hypoxia, anoxia) and especially reperfusion associated with the operative procedure; Pharmacia-Chiron Partnership

Suramin (Metaret); Hormone-refractory prostate cancer; Warner-Lambert

Surface active extract of saline lavage of bovine lungs (Infasurf); To prevent/treat respiratory failure caused by pulmonary surfactant deficiency in preterm infants; ONY

Synsorb Pk; Verocytotoxogenic E. coli infections; Synsorb Biotech

Synthetic human parathyroid hormone 1-34; Hypoparathyroidism; Orphan Pharm US

Synthetic human secretin; Used in the evaluation of exocrine pancreas function; used in obtaining desquamated pancreatic cells for cytopathologic examination in pancreatic carcinoma; diagnosis of gastrinoma associated with Zollinger-Ellison syndrome; with diagnostic procedures for pancreatic disorders to increase pancreatic fluid secretion; ChiRhoClin

Synthetic porcine secretin; Used in the evaluation of exocrine pancreas function; used in obtaining desquamated pancreatic cells for cytopathologic examination in pancreatic carcinoma; diagnosis of gastrinoma associated with Zollinger-Ellison syndrome; with diagnostic procedures for pancreatic disorders to increase pancreatic fluid secretion; ChiRhoClin

T-cell depleted stem cell enriched cellular product from peripheral blood stem cells; Chronic granulomatous disease; Nexell Therapeutics

T4 endonuclease V, liposomeencapsulated; To prevent cutaneous neoplasms and other skin abnormalities in xeroderma pigmentosum; AGI Dermatics

Tacrolimus (Prograf); Prophylaxis of graft-vs-host disease; Fujisawa USA

Talc, sterile (Steritalc); Malignant pleural effusion; pneumothorax; Novatech SA

Talc powder, sterile (Sclerosol Intrapleural Aerosol); Malignant pleural effusion1; Bryan

Technetium Tc99m murine monoclonal antibody (IgG2a) to B cell (LymphoScan); Diagnostic imaging in evaluating the extent of disease in patients with histologically confirmed diagnosis of non-Hodgkin’s B-cell lymphoma, acute B-cell lymphoblastic leukemia (in children and adults), and chronic B-cell lymphocytic leukemia; Immunomedics

Technetium Tc99m murine monoclonal antibody to hCG (Immuraid, hCG-Tc-99m); Detection of hCG-producing tumors such as germ-cell and trophoblastic cell tumors; Immunomedics

Technetium Tc99m murine monoclonal antibody to human AFP (AFP-Scan, Immuraid); Detection of alpha-fetoprotein producing germ-cell tumors; detection of hepatocellularcarcinoma and hepatoblastoma; Immunomedics

Technetium Tc99m pterotetramide; Identification of ovarian carcinomas; Endocyte

Technetium Tc99m rh-Annexin V (Apomate); Diagnosis or assessment of rejection status in heart, heart-lung, single lung, or bilateral lung transplants; Theseus Imaging

Temoporfin (Foscan); Palliative treatment of recurrent, refractory, or second primary squamous cell carcinomas of the head and neck considered to be incurable with surgery or radiotherapy; Scotia Pharm

Temozolomide

(Temodar); Recurrent malignant glioma1; Schering-Plough Res Inst

(Temodal); Advanced metastatic melanoma; Schering-Plough Res Inst

Teniposide (Vumon for Injection); Refractory childhood acute lymphocytic leukemia1; Bristol-Myers Squibb Pharm Res Inst

Teriparatide

(Parathar); Diagnostic agent for patients with clinical and laboratory evidence of hypocalcemia caused byhypoparathyroidism or pseudohypoparathyroidism1; Rhone-Poulenc Rorer

(Parathar); Idiopathic osteoporosis; Henri Beaufour Institute USA

Terlipressin (Glypressin); Bleeding esophageal varices; Ferring Labs

Testosterone

(Androgel); Weight loss in AIDS patients with HIV-associated wasting; Unimed

(TheraDerm Testosterone Transdermal System); Physiologic testosterone replacement in androgen deficient HIV-positive patients with anassociated weight loss; Watson Labs

Testosterone propionateointment 2%; Vulvar dystrophies; Star Pharm

Tetrabenazine; Moderate/Severe tardive dyskinesia; Huntington’s disease; Lifehealth Limited

Tetraiodothyroacetic acid; Suppression of thyroid stimulating hormone in patients with well-differentiated cancer of the thyroid gland; Elliot Danforth Jr., MD

Thalidomide; To prevent/treat graft-vs-host disease in bone marrow transplantation; treatment/maintenance of reactional lepromatous leprosy; to prevent/treat graft-vs-host disease; clinical manifestations of mycobacterial infection caused by Mycobacterium tuberculosis and nontuberculous mycobacteria; severe recurrent aphthous stomatitis and treatment and prevention of recurrent aphthous ulcers in severely, terminally immunocompromised patients; primary brain malignancies; Kaposi’s sarcoma; Andrulis Research Celgene Pediatric Pharm

(Synovir); HIV-associated wasting syndrome; Celgene

(Thalomid); Erythema nodosum leprosum;1 multiple myeloma; Crohn’s disease; Celgene

Thymalfasin (Zadaxin); Chronic active hepatitis B; DiGeorge anomaly with immune defects; hepatocellular carcinoma; SciClone Pharm

Thyrotropin alfa (Thyrogen); Well-differentiated papillary, follicular, or combined papillary/follicular carcinomas of the thyroid; adjunct in the diagnosis of thyroid cancer1; Genzyme

Tiazofurin (2-Beta-D-ribofuranosyl-4-thiazolecarboxamide); Chronic myelogenous leukemia (CML); ICN Pharm

Tiopronin (Thiola); Prevention of cystine nephrolithiasis in patients with homozygous cystinuria1; Charles Y. C. Pak, MD

Tiratricol (Triacana); With levothyroxine to suppress thyroid stimulating hormone in patients with well-differentiated thyroid cancer who are intolerant of adequate doses of levothyroxine alone; Laphal Lab

Tizanidine HCl (Zanaflex); Spasticity associated with multiple sclerosis and spinal cord injury; Athena Neurosciences

Tobramycin (Tobi); Bronchiectasis patients infected with Pseudomonas aeruginosa; Chiron

Tobramycin for inhalation (TOBI); Bronchopulmonary infections of P. aeruginosa in cystic fibrosis patients1; Pathogenesis

Topiramate (Topamax); Lennox-Gastaut syndrome1; RW Johnson Pharm Res Inst

Toremifene (Fareston); Hormonal therapy of metastatic breast carcinoma;1 desmoid tumors; Orion

Tositumomab and iodine I 131tositumomab (Bexxar); Non-Hodgkin’s B-cell lymphoma; Coulter Pharm

Tranexamic acid (Cyklokapron); Patients with congenital coagulopathies who are undergoing surgical procedures (eg, dental extractions)1; Pharmacia

Transforming growth factor-beta 2; Full-thickness macular holes; Celtrix Pharm

Transgenic human alpha 1 antitrypsin; Emphysema secondary to alpha 1 antitrypsin deficiency; PPL Therapeutics (Scotland) Limited

Trastuzumab (Herceptin); To treat patients with pancreatic cancer that overexpresses p185HER2; Genentech

Treosulfan (Ovastat); Ovarian cancer; Medac GmbH

Tretinoin; Squamous metaplasia of the ocular surface epithelia (conjunctiva or cornea) with mucousdeficiency and keratinization; Hannan Ophthalmic Marketing Service

(Atragen); Acute and chronic leukemia; Aronex Pharm

(Vesanoid); Acute promyelocytic leukemia1; Hoffmann-La Roche

Tri-antennary glycotripeptide derivative of 5-fluorodeoxyuridine monophosphate; Hepatocellular carcinoma; Cell Works

Trientine HCl (Syprine); Wilson’s disease intolerant or inadequately responsive to penicillamine1; Merck Sharp & Dohme Research

Trimetrexate glucuronate (Neutrexin); Metastatic carcinoma of head and neck (buccal cavity, pharynx, larynx); metastatic colorectal adenocarcinoma; pancreatic adenocarcinoma; Pneumocytosis carinii pneumonia in AIDS patients;1 advanced non-small-cell carcinoma of the lung; metastatic osteogenic sarcoma; Medimmune Oncology

Trisaccharides A and B (Biosynject); Moderate to severe clinical forms of hemolytic disease in newborns arising from placental transfer of antibodies against blood groups A and B; ABO-incompatible solid organ transplantation including kidney, heart, liver, and pancreas; prevent ABO hemolytic reactions arising from ABO-incompatible bone marrow transplantation; Chembiomed

Trisodium citrate concentration (Hemocitrate); For use in leukapheresis procedures; Hemotec Medical

Troleandomycin; Severe steroid-requiring asthma; Stanley M. Szefler, MD

Tumor necrosis factor-binding protein I and II; Symptomatic AIDS patients with CD4 counts or CD4 T-cell counts < 200 cells/mm3; Serono Labs

Tyloxapol (Supervent); Cystic fibrosis; Kennedy & Hoidal, MDs

Ubiquinone (Ubiqgel); Mitochondrial cytopathies; Gel-Tec, Division ofTishcon

Unconjugated Chimeric (human-murine) G250 IgG monoclonal antibody; Renal cell carcinoma; Wilex Biotechnology GmbH

Urofollitropin

(Metrodin); Ovulation induction in patients with polycystic ovarian disease who have an elevated LH/FSH ratio and who have failed to respond to adequate clomiphene citrate therapy1; Serono

(Fertinex); For the initiation and reinitiation of spermatogenesis in adult males with reproductive failure caused byhypothalamic or pituitary dysfunction; hypogonadotropic hypogonadism; Serono

Urogastrone; Acceleration of corneal epithelial regeneration and healing of stromal incisions from corneal transplant surgery; Chiron Vision

Ursodiol

(URSO); Treatment of primary biliary cirrhosis1; Axcan Pharma

Valine, isoleucine, and leucine (VIL); Hyperphenylalaninemia; Leas Research Products

Valrubicin (Valstar); Carcinoma in situ of the urinary bladder1; Anthra Pharm

Vapreotide (Octastatin); GI and pancreatic fistulas; prevention of early postoperative complications following pancreatic resection; esophageal variceal hemorrhage patients with portal hypertension; Debiopharm S.A.

Vasoactive intestinal peptide; Acute respiratory distress syndrome; Sami I. Said, MD

Vasoactive intestinal polypeptide; Acute esophageal food impaction; Research Triangle

Vigabatrin (Sabril); Infantile spasms; Aventis Pharm

Virulizin (Virulizin); Pancreatic cancer; Lorus Therapeutics

Xenogeneic hepatocytes (HepatAssist Liver Assist System); Severe liver failure; Circe Biomedical

Yttrium-90 radiolabeled humanized monoclonal anti-carcinoembryonic antigen IgG antibody (Cea-Cide); Ovarian carcinoma; Immunomedics

Zalcitabine (Hivid); AIDS1; National Cancer Inst, DCT Hoffman-La Roche

Zidovudine (Retrovir); AIDS1 and AIDS-related complex1; GlaxoWellcome

Zinc acetate (Galzin); Wilson’s disease1; Lemmon

Zoledronate (Zometa, Zabel); Tumor-induced hypercalcemia1; Novartis

1 Approved for marketing.

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Copyright © 2003 Facts and Comparisons
David S. Tatro
A to Z Drug Facts