Last reviewed · How we verify
Orfadin
At a glance
| Generic name | Orfadin |
|---|---|
| Also known as | Nitisinone |
| Sponsor | Cycle Pharmaceuticals Ltd. |
| Target | 4-hydroxyphenylpyruvate dioxygenase, 4-hydroxyphenylpyruvate dioxygenase |
| Modality | Small molecule |
| Therapeutic area | Other |
| Phase | FDA-approved |
Approved indications
- Homogentisate 1,2dioxygenase deficiency
- Tyrosinemia type I
Common side effects
- Elevated tyrosine levels
- Leukopenia
- Thrombocytopenia
- Conjunctivitis
- Corneal opacity
- Keratitis
- Photophobia
- Eye pain
- Blepharitis
- Cataracts
- Granulocytopenia
- Epistaxis
Serious adverse events
- Liver transplantation
- Liver failure
- Malignant hepatic neoplasms
- Benign hepatic neoplasms
- Porphyria
- Death
- Seizure
- Brain tumor
- Encephalopathy
- Gastrointestinal hemorrhage
Key clinical trials
- A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
- Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
- Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules. (PHASE1)
- Long-Term Study of Nitisinone to Treat Alkaptonuria (PHASE2)
- Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria (PHASE2, PHASE3)
- Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine (NA)
- Nitisinone for Type 1B Oculocutaneous Albinism (PHASE1, PHASE2)
- Suitability of Nitisinone in Alkaptonuria 2 (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Orfadin CI brief — competitive landscape report
- Orfadin updates RSS · CI watch RSS
- Cycle Pharmaceuticals Ltd. portfolio CI