Nitisinone
Orphan DrugFDA Approved
Contents
Description
Nitisinone is an orphan drug that inhibits 4-hydroxyphenylpyruvate dioxygenase (HPPD). Originally approved for hereditary tyrosinemia type 1, it has been investigated for oculocutaneous albinism due to its effects on tyrosine metabolism. The drug increases tyrosine levels which may enhance melanin production in certain forms of albinism.
Indications & Therapeutic Use
Hereditary tyrosinemia type 1, investigational use in oculocutaneous albinism
Linked Diseases:
Global Availability (7 countries)
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Buy / Source Nitisinone — GNH India
56 matching products available through GNH India. Select a product to view details and request a quote on the GNH India website.
MDK-NITISINONE
2 MGcapsuleCANADA
Nitisinon Dipharma
EU
Nitisinona Dipharma
EU
Nitisinone
10 mg/1capsuleUSA
Nitisinone MDK
5 MGcapsuleUK
Nitisinone Dipharma
EU
Nitisinone Logixx Pharma
EU
NITISINONE TABLETS
10 MGtabletUNITED KINGDOM
Products and ordering are fulfilled by GNH India. You will be redirected to www.gnhindia.com.
Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Nitisinone
| Generic Name | Nitisinone |
| Brands | 1 brand available |
| Active Ingredient | Nitisinone |
| Drug Class | Hereditary tyrosinemia type 1 |
| Manufacturer | Sobi (Swedish Orphan Biovitrum) |
| Dosage Forms | Oral capsule, 2mg, 5mg, 10mg, 20mg; Oral suspension, 4mg/mL |
| Medical Code | A16AX04 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 21 days |
| Reg. Status | FDA Approved |
| Countries | 7 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations8 Validated Nodes