Orfadin (Nitisinone)

Nitisinone, the active ingredient in Orfadin, is a p‑hydroxyphenylpyruvate dioxygenase (HPPD) inhibitor presented as a tablet for oral use. It treats hereditary tyrosinemia type I in patients with this metabolic disorder.

GNH USA supplies Orfadin as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, transplant centres, oncology centres and pharmacies worldwide. The company adheres to stringent quality standards and provides reliable logistics for global distribution. Clients benefit from comprehensive regulatory support and timely delivery across multiple continents. Our network ensures product availability even in remote clinical settings.

Orfadin (nitisinone) is used to manage disorders of the tyrosine degradation pathway, primarily hereditary tyrosinemia type I.

Hereditary tyrosinemia type I (initial therapy): reduces accumulation of toxic metabolites and improves liver function. Hereditary tyrosinemia type I (pre‑transplant management): stabilizes metabolic status to enable safe liver transplantation. Hereditary tyrosinemia type I (long‑term maintenance): maintains metabolic control and delays disease progression.

Nitisinone may cause a range of adverse reactions, which are categorized by frequency and severity.

Common Side Effects: - Elevated liver enzymes: mild to moderate increase in ALT or AST. - Skin rash: pruritic or erythematous eruptions. - Nausea or vomiting: gastrointestinal discomfort. - Headache: mild transient headache.

Serious or Rare Side Effects: - Severe hepatic dysfunction: marked transaminase rise or hepatic failure. - Hypersensitivity reactions: anaphylaxis, angioedema, or severe skin reactions. - Neurological effects: seizures or encephalopathy.

When prescribing nitisinone, clinicians should observe several safety measures and monitoring requirements. - Baseline assessment: obtain liver function tests before initiating therapy. - Monitoring: regularly check hepatic enzymes and renal function during treatment. - Drug interactions: avoid concomitant use with strong CYP inducers without supervision. - Pregnancy and lactation: use only if potential benefit justifies risk, as safety data are limited. - Pediatric use: dose adjustment and growth monitoring are essential. - Store at ambient temperature: keep tablets below 25 °C in the original packaging, protected from moisture.

Nitisinone can interact with other medicines, influencing its efficacy or safety profile.

Major Interactions (Avoid): - Concurrent use with strong CYP3A4 inducers (e.g., rifampicin): may markedly reduce nitisinone plasma levels. - Co‑administration with high‑dose vitamin C: can increase risk of renal stone formation.

Moderate Interactions (Monitor Closely): - Warfarin: nitisinone may potentiate anticoagulant effect; monitor INR closely. - Phenobarbital: may modestly decrease nitisinone concentrations; adjust dose if needed. - Other HPPD inhibitors: combined effect may increase toxicity; avoid simultaneous use.