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 4-hydroxyphenylpyruvate dioxygenase (HPPD) inhibitor presented as an oral tablet for oral use. It treats hereditary tyrosinemia type I and is being investigated for alkaptonuria in affected patients.

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 and improves liver function.
  • Alkaptonuria (investigational): may lower homogentisic acid levels, under clinical investigation.

How It Works

  • Nitisinone inhibits the enzyme 4-hydroxyphenylpyruvate dioxygenase (HPPD), a key step in the catabolism of tyrosine. By blocking this step, the drug prevents formation of downstream toxic metabolites such as maleylacetoacetate and fumarylacetoacetate, thereby reducing hepatic and renal toxicity associated with hereditary tyrosinemia type I.

Side Effects

Adverse reactions may vary in frequency and severity.

Common Side Effects

  • Nausea
  • Vomiting
  • Abdominal pain
  • Headache
  • Fatigue
  • Skin rash

Serious or Rare Side Effects

  • Hepatotoxicity (elevated liver enzymes, liver failure)
  • Severe hypoglycemia
  • Peripheral neuropathy
  • Severe skin reactions (Stevens‑Johnson syndrome, toxic epidermal necrolysis)
  • Anaphylactic reactions
  • Acute liver failure

Precautions & Warnings

  • Before initiating therapy, clinicians should consider several safety measures.
  • Monitor liver function: perform baseline and periodic liver enzyme tests.
  • Assess tyrosine levels: avoid excessive dietary tyrosine intake.
  • Avoid in patients with known hypersensitivity to nitisinone.
  • Use caution during pregnancy and breastfeeding: safety data are limited.
  • Adjust dosing in renal impairment: monitor renal function.
  • Store at below 25°C: keep in the original pack, protect from moisture.

Avoid Interactions

Nitisinone may interact with other medicines, requiring attention to potential effects.

Major Interactions (Avoid)

  • Co‑administration with other HPPD inhibitors: risk of additive toxicity.
  • High‑protein supplements or amino‑acid formulas that increase tyrosine levels.

Moderate Interactions (Monitor Closely)

  • Warfarin: possible alteration of INR, monitor coagulation.
  • Phenobarbital or other enzyme‑inducing agents: may affect nitisinone metabolism.
  • Oral contraceptives: monitor for hormonal changes or reduced efficacy.
  • Antacids containing aluminum or magnesium: may affect absorption, separate administration by at least 2 hours.

Frequently Asked Questions

Nitisinone Dipharma is used to treat hereditary tyrosinemia type I, a rare inherited disorder that affects the liver and kidneys. It is also being studied as a potential therapy for alkaptonuria, another metabolic condition.

Nitisinone blocks the enzyme 4‑hydroxyphenylpyruvate dioxygenase (HPPD) in the tyrosine degradation pathway. This inhibition prevents the formation of toxic downstream metabolites that cause organ damage in hereditary tyrosinemia type I.

The prescribed dose of Nitisinone Dipharma is determined by the treating physician based on the patient’s age, weight, disease severity, and laboratory results. Dosage adjustments are made according to clinical response and safety monitoring.

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

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 and any necessary import documentation.

Nitisinone is the primary disease‑modifying therapy for hereditary tyrosinemia type I, directly targeting the metabolic defect. Alternative options such as liver transplantation address organ failure but do not correct the underlying metabolic pathway. Nitisinone therefore offers a less invasive, targeted approach.

Nitisinone Dipharma tablets should be stored at temperatures below 25 °C, in their original packaging, and protected from moisture and direct sunlight. Keep the product out of reach of children.

The medication is supplied as oral tablets and is taken by mouth with water. It is usually administered once daily, but the exact schedule is set by the prescribing clinician based on individual patient needs.

Serious risks include hepatotoxicity, severe hypoglycemia, peripheral neuropathy, and rare but life‑threatening skin reactions such as Stevens‑Johnson syndrome. Patients should be monitored regularly for liver function and any signs of severe adverse reactions.

Patients with known hypersensitivity to nitisinone or any of its excipients should avoid the product. Caution is advised in individuals with severe liver disease, uncontrolled diabetes, or those taking interacting medications listed under major interactions.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated:

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