Nitisinone

Entry

D05177                      Drug

Name

Nitisinone (JAN/USAN/INN);
Orfadin (TN)

Product

NITYR (Cycle Pharmaceuticals), ORFADIN (SWEDISH ORPHAN BIOVITRUM AB (PUBL)), ORFADIN (Swedish Orphan Biovitrum AB (publ))

Generic

NITISINONE (Analog Pharma), NITISINONE (Bryant Ranch Prepack), NITISINONE (Bryant Ranch Prepack), NITISINONE (Bryant Ranch Prepack), NITISINONE (Par Pharmaceutical)

Formula

C14H10F3NO5

Exact mass

329.0511

Mol weight

329.2281

Structure

Remark

Therapeutic category:

3999

ATC code:

A16AX04

Product:

D05177<JP/US>

Efficacy

Antityrosinemia

Disease

Hereditary tyrosinemia type 1 [DS:H00165]

Comment

Treatment of hereditary tyrosinemia

Target

HPD [HSA:3242] [KO:K00457]

Network

N10020

HPD inhibitor to downstream of deficient FAH

Pathway

hsa00130

Ubiquinone and other terpenoid-quinone biosynthesis

hsa00350

Tyrosine metabolism

hsa00360

Phenylalanine metabolism

Interaction

Brite

Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
A ALIMENTARY TRACT AND METABOLISM
  A16 OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
   A16A OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
    A16AX Various alimentary tract and metabolism products
     A16AX04 Nitisinone
      D05177  Nitisinone (JAN/USAN/INN) <JP/US>
USP drug classification [BR:br08302]
Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
  Nitisinone
   D05177  Nitisinone (JAN/USAN/INN)
Therapeutic category of drugs in Japan [BR:br08301]
3  Agents affecting metabolism
  39  Other agents affecting metabolism
   399  Miscellaneous
    3999  Others
     D05177  Nitisinone (JAN/USAN/INN)
Target-based classification of drugs [BR:br08310]
Enzymes
  Oxidoreductases (EC1)
   Dioxygenases
    HPD
     D05177  Nitisinone (JAN/USAN/INN) <JP/US>
New drug approvals in the USA [br08319.html]
New molecular entities and new therapeutic biological products
  D05177
New drug approvals in Europe [br08329.html]
European public assessment reports (EPAR) authorised medicine
  D05177
New drug approvals in Japan [br08318.html]
Drugs with new active ingredients
  D05177
New drug approvals in the USA, Europe and Japan [br08328.html]
Approval dates by FDA, EMA and PMDA
  D05177
Pharmacogenomic biomarkers [br08341.html]
Germline mutations in genetic disorder treatments
  D05177

Other DBs

CAS:	104206-65-7

PubChem:

47206901

ChEBI:

50378

LigandBox:

D05177

NIKKAJI:

J519.110F

KCF data

ATOM        23
            1   C8x C    23.8922  -17.9269
            2   C8x C    22.6973  -18.6148
            3   C8y C    21.5042  -17.9240
            4   C8x C    21.5059  -16.5453
            5   C8y C    22.7007  -15.8574
            6   C8y C    23.8938  -16.5482
            7   N2b N    22.7027  -14.4941 #+
            8   O3a O    23.9220  -13.3092
            9   O3a O    21.5318  -13.3326 #-
            10  C1d C    20.2904  -18.6227
            11  X   F    19.0953  -19.3126
            12  X   F    19.6134  -17.4475
            13  X   F    20.9910  -19.8387
            14  C5a C    25.1009  -15.8530
            15  C1y C    26.3194  -16.5585
            16  O5a O    25.1019  -14.4203
            17  C5x C    26.3176  -17.9900
            18  C1x C    27.5291  -18.6915
            19  C1x C    28.7424  -17.9930
            20  C1x C    28.7442  -16.5616
            21  C5x C    27.5327  -15.8600
            22  O5x O    27.5339  -14.4902
            23  O5x O    25.1061  -18.6874
BOND        24
            1     1   2 2
            2     2   3 1
            3     3   4 2
            4     4   5 1
            5     5   6 2
            6     6   1 1
            7     5   7 1
            8     7   8 2
            9     7   9 1
            10    3  10 1
            11   10  11 1
            12   10  12 1
            13   10  13 1
            14    6  14 1
            15   14  15 1
            16   14  16 2
            17   15  17 1
            18   17  18 1
            19   18  19 1
            20   19  20 1
            21   20  21 1
            22   15  21 1
            23   21  22 2
            24   17  23 2