Orfadin (Nitisinone)

Nitisinone selectively blocks the hepatic enzyme p‑hydroxyphenylpyruvate dioxygenase (HPPD). By inhibiting this step, the conversion of p‑hydroxyphenylpyruvate to downstream toxic compounds is halted, preventing accumulation of harmful metabolites that cause liver damage and systemic complications in hereditary tyrosinemia type I.

The dosage of Orfadin is individualized. A qualified prescriber determines the appropriate amount based on the patient’s age, weight, disease severity, and laboratory monitoring. Dosing regimens are adjusted according to therapeutic response and safety parameters, and patients should follow the specific instructions provided by their healthcare professional.

Safety data for nitisinone during pregnancy and lactation are limited, and the product is classified with an unknown pregnancy category. Use is only recommended when the potential benefit to the mother outweighs any possible risk to the fetus or infant, and decisions should be made in consultation with a specialist.

To request Orfadin, submit an enquiry on the product page at gnhindia.com, specifying the required quantity. GNH India works with hospitals, pharmacies, and procurement teams worldwide, verifies trade or institutional credentials, and then provides pricing, availability, shipping details (including any cold‑chain requirements), and assistance with import documentation.

The primary alternative to nitisinone is liver transplantation, which replaces the defective metabolic pathway but carries surgical risks. Nitisinone offers a non‑surgical option that can control disease progression and delay or avoid transplantation. Compared with other HPPD inhibitors, nitisinone is the only agent approved specifically for hereditary tyrosinemia type I.

Orfadin tablets should be stored at ambient temperature, preferably below 25 °C. Keep them in the original blister pack, protect from moisture and direct sunlight, and store out of reach of children. Do not refrigerate or freeze the tablets, as temperature extremes may affect stability.

Orfadin is taken orally as a tablet, usually with water. The exact timing relative to meals depends on the prescriber’s instructions. Patients should swallow the tablet whole and not split, crush, or chew it unless directed otherwise. Consistent adherence to the prescribed schedule is essential for optimal therapeutic effect.

The most serious adverse events reported with nitisinone include severe hepatic dysfunction, which may manifest as marked elevations in liver enzymes or liver failure, and hypersensitivity reactions such as anaphylaxis or severe skin eruptions. Prompt medical evaluation is required if signs of liver injury or allergic response appear.

Orfadin is contraindicated in patients with known hypersensitivity to nitisinone or any of its excipients. Caution is also advised for individuals with pre‑existing severe liver disease, uncontrolled infections, or those receiving medications that strongly induce HPPD metabolism, as efficacy may be compromised.