Europe — EMA
Orfadin (Nitisinone)
GNH USA supplies Orfadin (Nitisinone), a metabolic drug for hereditary tyrosinemia type 1. Request a quote for global supply.
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What is Nitisinone?
Nitisinone, the active ingredient in Orfadin, is a drug class presented as a strength/form for oral use. It treats hereditary tyrosinemia type 1 in patients who require metabolic management. GNH USA supplies Orfadin as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals worldwide.
GNH USA supplies Orfadin as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals worldwide.
What is Nitisinone used for?
Orfadin is used to manage disorders of the tyrosine degradation pathway, specifically hereditary tyrosinemia type 1.
- Hereditary tyrosinemia type 1: Reduces the accumulation of toxic metabolites and helps prevent liver failure. - Acute hepatic crisis: Assists in stabilising liver function during episodes. - Long‑term metabolic control: Supports sustained reduction of disease‑related complications.
What are the side effects of Nitisinone?
Adverse reactions to Orfadin have been reported across a range of severity.
Common Side Effects: - Headache - Nausea - Diarrhoea - Elevated liver enzymes - Skin rash - Fatigue
Serious or Rare Side Effects: - Severe liver injury - Acute renal failure - Hypersensitivity reactions including anaphylaxis - Significant elevation of serum tyrosine leading to ocular complications - Metabolic acidosis - Unexplained fever
What precautions apply to Nitisinone?
When prescribing Orfadin, clinicians should consider several safety measures.
- Liver function monitoring: Perform baseline and periodic liver enzyme tests. - Tyrosine level monitoring: Check plasma tyrosine concentrations regularly. - Dietary management: Advise a low‑tyrosine, low‑phenylalanine diet. - Drug interactions: Review concomitant medications for potential HPPD inhibition. - Pregnancy and lactation: Evaluate risk‑benefit ratio before use. - Store at below 25°C: keep in the original container, protect from moisture and light.
What does Nitisinone interact with?
Orfadin may interact with other medicines, requiring attention to the type of interaction.
Major Interactions (Avoid): - Concurrent use of other HPPD inhibitors may increase toxicity. - Strong CYP450 inducers may reduce nitisinone plasma levels.
Moderate Interactions (Monitor Closely): - Anticoagulants (e.g., warfarin) – monitor INR. - NSAIDs – watch for additive liver enzyme elevation. - Vitamin C supplements – may affect tyrosine metabolism. - Oral contraceptives – potential alteration of hormone levels. - High‑protein meals – may influence absorption.
