Product image of Nitisinone Dipharma (Nitisinone) supplied by GNH India

Nitisinone Dipharma

Active Ingredient:
Nitisinone
Origin:
EU

Nitisinone Dipharma (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 India supplies Nitisinone Dipharma as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, transplant centres and pharmacies worldwide.

Manufacturer / TM Owner

Dipharma Arzneimittel Gmbh

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Indications & Clinical Uses

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.

How It Works

  • Nitisinone inhibits the enzyme p‑hydroxyphenylpyruvate dioxygenase (HPPD), a key step in the catabolic pathway of tyrosine. By blocking HPPD, the drug prevents formation of downstream toxic metabolites such as fumarylacetoacetate, thereby reducing hepatic and renal injury associated with hereditary tyrosinemia type I.

Side Effects

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.

Precautions & Warnings

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.

Avoid Interactions

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.

Frequently Asked Questions

Nitisinone Dipharma contains nitisinone, which is prescribed to treat hereditary tyrosinemia type I, a rare genetic disorder that disrupts the normal breakdown of the amino acid tyrosine. By reducing toxic metabolites, it helps protect liver function and supports overall metabolic control.

Nitisinone blocks the enzyme p‑hydroxyphenylpyruvate dioxygenase (HPPD) in the tyrosine catabolic pathway. This inhibition prevents the formation of harmful downstream metabolites such as fumarylacetoacetate, thereby reducing liver and kidney damage associated with hereditary tyrosinemia type I.

The specific dose of Nitisinone Dipharma is determined by the prescribing physician based on the patient’s age, weight, disease severity, and laboratory parameters. Dosage adjustments are made during treatment to achieve optimal metabolic control while minimizing adverse effects.

The safety of nitisinone in pregnancy and lactation has not been established. It is classified as a pregnancy category with unknown risk, and clinicians usually weigh potential benefits against possible fetal or infant exposure before prescribing.

To place an order, 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 necessary import documentation.

The main alternative is liver transplantation, which addresses organ damage but carries surgical risks. Nitisinone offers a pharmacologic approach that can delay or avoid transplantation by controlling metabolite buildup. Compared with dietary restriction alone, nitisinone provides more reliable metabolic control.

Nitisinone Dipharma should be stored at room temperature below 25 °C. Keep the product in its original packaging, protect it from moisture and direct sunlight, and ensure the container is tightly closed to maintain stability.

The medication is taken orally, usually as a tablet or capsule, with or without food as directed by the prescriber. Patients should follow the exact schedule and dosage instructions provided by their healthcare professional.

Serious adverse events include significant liver enzyme elevations, neutropenia, and peripheral neuropathy. Patients should be monitored regularly for hepatic function, blood counts, and neurological symptoms, and any concerning signs should prompt immediate medical evaluation.

Individuals with known hypersensitivity to nitisinone or any component of the formulation should avoid the product. Caution is also advised for patients with severe hepatic impairment, uncontrolled infections, or those taking contraindicated medications that may interact severely.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated:

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