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

Manufacturer / TM Owner

Dipharma Arzneimittel Gmbh

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Indications & Clinical Uses

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.

How It Works

  • Nitisinone inhibits the enzyme p‑hydroxyphenylpyruvate dioxygenase (HPPD), a key catalyst in the catabolism of tyrosine. By blocking this step, the drug prevents formation of downstream toxic metabolites such as fumarylacetoacetate, thereby reducing hepatic and systemic toxicity associated with hereditary tyrosinemia type I and related disorders.

Side Effects

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.

Precautions & Warnings

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.

Avoid Interactions

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.

Frequently Asked Questions

Nitisinone Dipharma is primarily used to treat hereditary tyrosinemia type I, a rare metabolic disorder that leads to toxic liver and kidney damage. It is also explored off‑label for alkaptonuria, where it may help reduce the buildup of harmful pigments.

Nitisinone blocks the enzyme p‑hydroxyphenylpyruvate dioxygenase (HPPD) in the tyrosine degradation pathway. By inhibiting this step, it prevents the formation of downstream toxic metabolites such as fumarylacetoacetate, thereby protecting liver and kidney tissues from damage.

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 according to clinical response and safety monitoring.

The safety of Nitisinone in pregnancy and lactation has not been established. It should only be used if the potential benefit justifies any possible risk to the fetus or infant, and the decision must be made by a qualified healthcare professional.

To 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, and necessary import documentation.

Nitisinone is the only disease‑modifying therapy that directly inhibits the HPPD enzyme, addressing the metabolic cause of hereditary tyrosinemia type I. Alternative approaches, such as liver transplantation, manage complications but do not correct the underlying metabolic defect.

Store the product at temperatures below 25 °C, in its original packaging, and protect it from moisture and direct sunlight. Do not refrigerate or freeze unless the label specifically indicates otherwise.

Nitisinone Dipharma is taken orally, usually as a tablet or capsule, with or without food as directed by the prescribing clinician. The exact administration schedule is individualized for each patient.

Serious risks include hepatic failure, severe neutropenia, and hypersensitivity reactions such as anaphylaxis. Patients should be monitored regularly for liver function, blood counts, and any signs of allergic response.

Patients with known hypersensitivity to Nitisinone or any component of the formulation should avoid the drug. Caution is also advised for individuals with severe liver or kidney impairment, and the medication should be used only under specialist supervision in such cases.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated:

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