Orfadin (Nitisinone)

Nitisinone blocks the enzyme 4‑hydroxyphenylpyruvate dioxygenase (HPPD) in the tyrosine degradation pathway. This inhibition prevents the formation of harmful downstream metabolites, thereby lowering the risk of liver and kidney damage associated with hereditary tyrosinemia type 1.

The dose of Orfadin is individualized by the prescribing clinician based on the patient’s age, weight, disease severity, and laboratory monitoring results. Healthcare professionals determine the appropriate regimen and adjust it as needed during treatment.

Safety data for nitisinone in pregnancy and lactation are limited. The drug should only be used if the potential benefit to the mother outweighs any possible risk to the fetus or infant, and the decision should be made by a qualified healthcare provider.

To order Orfadin, submit an enquiry on the product page at gnhindia.com, specifying the required quantity. GNH India supplies hospitals, pharmacies and procurement teams worldwide, and will request trade or institutional credentials before providing pricing, availability, shipping details and import documentation.

Orfadin is the primary pharmacologic therapy that directly inhibits toxic metabolite formation, whereas liver transplantation addresses end‑stage disease. Compared with dietary management alone, nitisinone offers a targeted biochemical approach, often reducing the need for transplantation.

Store Orfadin tablets at temperatures below 25 °C in the original packaging, protecting them from moisture and light. Keep the container tightly closed and out of reach of children.

Orfadin is taken orally, usually as a tablet, with or without food as directed by the prescribing clinician. The exact administration schedule is individualized based on the patient’s therapeutic plan.

Serious risks include severe liver injury, acute renal failure, hypersensitivity reactions such as anaphylaxis, and significant elevations in plasma tyrosine that can cause ocular complications. Patients should be monitored regularly for these events.

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 or kidney disease until safety can be confirmed by a healthcare professional.