Onasemnogene abeparvovec


Entry
D11559                      Drug                                   
Name
Onasemnogene abeparvovec (USAN/INN);
Onasemnogene abeparvovec-xioi;
Zolgensma (TN)
Product
ZOLGENSMA (Novartis Gene Therapies)
Remark
ATC code: M09AX09
Product: D11559<US>
Efficacy
Spinal muscular atrophy treatment
  Disease
Spinal muscular atrophy (bi-allelic SMN1 mutations) [DS:H00455]
Comment
Gene therapy product
Recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein
Target
SMN1 [HSA:6606] [KO:K13129]
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 M MUSCULO-SKELETAL SYSTEM
  M09 OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
   M09A OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
    M09AX Other drugs for disorders of the musculo-skeletal system
     M09AX09 Onasemnogene abeparvovec
      D11559  Onasemnogene abeparvovec (USAN/INN) <US>
USP drug classification [BR:br08302]
 Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
  Onasemnogene abeparvovec
   D11559  Onasemnogene abeparvovec (USAN/INN)
Target-based classification of drugs [BR:br08310]
 Not elsewhere classified
  Signaling molecules
   Spliceosome
    SMN1
     D11559  Onasemnogene abeparvovec (USAN/INN) <US>
New drug approvals in the USA [br08319.html]
 Cellular and gene therapy products
  D11559
New drug approvals in Europe [br08329.html]
 European public assessment reports (EPAR) authorised medicine
  D11559
New drug approvals in Japan [br08318.html]
 Cellular and gene therapy products
  D11559
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D11559
Pharmacogenomic biomarkers [br08341.html]
 Germline mutations in genetic disorder treatments
  D11559
Other DBs
CAS: 1922968-73-7
PubChem: 384585535