Europe — EMA
Nitisinone Dipharma
GNH USA supplies Nitisinone Dipharma (Nitisinone), a p‑Hydroxyphenylpyruvate dioxygenase inhibitor for hereditary tyrosinemia type I treatment.
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What is Nitisinone?
Nitisinone, the active ingredient in Nitisinone Dipharma, is a p‑Hydroxyphenylpyruvate dioxygenase (HPPD) inhibitor presented as an oral formulation for oral use. It treats hereditary tyrosinemia type I in patients with this inherited metabolic disorder.
GNH USA supplies Nitisinone Dipharma as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, transplant centres and pharmacies worldwide.
What is Nitisinone used for?
Nitisinone is used to manage disorders of the tyrosine degradation pathway, most notably hereditary tyrosinemia type I.
- Hereditary tyrosinemia type I: reduces accumulation of toxic metabolites and helps preserve liver function. - Preventive management of liver failure in tyrosinemia I: delays the need for liver transplantation. - Metabolic control in affected children: supports normal growth and development by normalising tyrosine metabolism.
What are the side effects of Nitisinone?
Adverse reactions may occur with Nitisinone therapy and should be monitored.
Common Side Effects: - Headache: mild to moderate intensity. - Nausea or vomiting: gastrointestinal discomfort. - Abdominal pain: intermittent cramping. - Fatigue: reduced energy levels.
Serious or Rare Side Effects: - Elevated liver enzymes: indicates hepatic stress. - Neutropenia: reduced white blood cell count. - Peripheral neuropathy: tingling or numbness in extremities.
What precautions apply to Nitisinone?
Before initiating therapy, clinicians should assess baseline organ function and counsel patients on potential risks.
- Liver function monitoring: check transaminases regularly. - Renal function assessment: evaluate serum creatinine and eGFR. - Hypersensitivity avoidance: discontinue if rash or anaphylaxis occurs. - Photosensitivity warning: advise protection from excessive sunlight. - Drug interaction review: consider concomitant medications that affect metabolism. - Store at room temperature below 25°C: keep in the original pack, protect from moisture.
What does Nitisinone interact with?
Nitisinone may interact with other medicines that influence its metabolism or therapeutic effect.
Major Interactions (Avoid): - Other HPPD inhibitors: additive toxicity risk. - Strong CYP3A4 inducers (e.g., rifampicin, carbamazepine): may markedly reduce nitisinone exposure.
Moderate Interactions (Monitor Closely): - Warfarin: potential alteration of INR, requiring closer coagulation monitoring. - NSAIDs: increased risk of hepatic injury. - Oral contraceptives: possible reduced efficacy, advise alternative contraception.
