Europe — EMA
Nitisinone Dipharma
GNH USA supplies Nitisinone Dipharma (Nitisinone), a p-Hydroxyphenylpyruvate dioxygenase inhibitor for hereditary tyrosinemia type I.
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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 is employed in the management of hereditary tyrosinemia type I and explored off‑label for alkaptonuria, targeting the metabolic pathway that generates toxic tyrosine‑derived compounds.
GNH USA supplies Nitisinone Dipharma as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, transplant centres, oncology centres, pharmacies and clinics worldwide.
What is Nitisinone used for?
Nitisinone acts on the tyrosine degradation pathway and is indicated for specific inherited metabolic disorders. - Hereditary tyrosinemia type I: reduces accumulation of toxic metabolites, improving liver function and survival. - Alkaptonuria (off‑label): may lower homogentisic acid buildup, potentially slowing disease progression.
What are the side effects of Nitisinone?
Nitisinone may cause a range of adverse reactions, from mild to serious.
Common Side Effects: - Nausea: mild to moderate gastrointestinal upset. - Headache: transient, often resolves with continued therapy. - Fatigue: generalized tiredness reported by some patients. - Skin rash: mild erythema or pruritus.
Serious or Rare Side Effects: - Hepatic failure: marked elevation of liver enzymes or liver dysfunction. - Severe neutropenia: significant reduction in white blood cells. - Hypersensitivity reactions: including anaphylaxis or severe skin reactions.
What precautions apply to Nitisinone?
Before initiating Nitisinone therapy, several safety considerations should be observed. - Liver monitoring: regularly assess hepatic enzymes and function. - Renal assessment: evaluate kidney function, especially in patients with pre‑existing impairment. - Hypersensitivity: discontinue if signs of allergic reaction appear. - Pregnancy and lactation: use only if benefits outweigh unknown risks. - Pediatric use: dose adjustment may be required based on weight and age. - Store at below 25°C: keep in the original pack, protect from moisture.
What does Nitisinone interact with?
Nitisinone can interact with other medicines, requiring careful management.
Major Interactions (Avoid): - Concurrent HPPD inhibitors: additive metabolic blockade may increase toxicity. - Strong CYP2C9 inhibitors: may raise plasma levels of Nitisinone. - Warfarin: potential enhancement of anticoagulant effect.
Moderate Interactions (Monitor Closely): - Acetaminophen: monitor liver function due to combined hepatic load. - NSAIDs: increased risk of gastrointestinal irritation. - Vitamin K antagonists: observe INR for possible alterations.
