GNH USA

Europe — EMA

Nitisinone Dipharma

GNH USA supplies Nitisinone Dipharma (Nitisinone), a p‑Hydroxyphenylpyruvate dioxygenase inhibitor for hereditary tyrosinemia type I and alkaptonuria.

Nitisinone

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What is 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, off‑label, alkaptonuria in affected patients.

GNH USA supplies Nitisinone Dipharma as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, transplant centres, oncology centres, pharmacies and clinics worldwide, with adherence to stringent quality standards.

What is Nitisinone used for?

Nitisinone acts on the tyrosine catabolic pathway and is used for metabolic disorders involving toxic metabolite accumulation.

Hereditary tyrosinemia type I: reduces accumulation of toxic metabolites and helps prevent liver and renal complications. Alkaptonuria (off‑label): lowers homogentisic acid production, potentially reducing ochronosis progression. Metabolic control in tyrosine catabolism disorders: supports normalization of pathway metabolites and mitigates disease‑related complications.

What are the side effects of Nitisinone?

Nitisinone may be associated with a range of adverse reactions, which vary in frequency and severity.

Common Side Effects: - Skin rash or pruritus - Elevated liver enzymes (ALT, AST) - Nausea or vomiting - Diarrhea - Headache - Fatigue

Serious or Rare Side Effects: - Severe liver injury or hepatitis - Hypoglycemia, especially in fasting patients - Peripheral neuropathy - Acute renal failure - Anaphylactic reactions (rare) - Severe photosensitivity

What precautions apply to Nitisinone?

Before initiating therapy, several safety considerations should be observed.

Monitor liver function: obtain baseline and periodic ALT/AST measurements. Avoid use in patients with known hypersensitivity to nitisinone or excipients. Caution in renal impairment: adjust monitoring frequency and assess renal function. Pregnancy: risk unknown; prescribe only if potential benefit outweighs potential risk. Store at below 25°C: keep in the original pack, protect from moisture and direct sunlight.

What does Nitisinone interact with?

Nitisinone can interact with other medicines, requiring attention to avoid reduced efficacy or increased toxicity.

Major Interactions (Avoid): - Concurrent strong CYP2C9 inhibitors: may raise nitisinone plasma concentrations. - Co‑administration with other hepatotoxic drugs: increased risk of liver injury.

Moderate Interactions (Monitor Closely): - Vitamin C supplementation: may diminish therapeutic effect. - Warfarin: monitor INR closely due to potential alteration of anticoagulant effect. - Enzyme‑inducing anticonvulsants (e.g., carbamazepine): may lower nitisinone levels.

Frequently asked questions

What conditions is Nitisinone Dipharma used for?+
Nitisinone Dipharma is indicated for the treatment of hereditary tyrosinemia type I, a rare metabolic disorder that leads to toxic liver and kidney metabolites. It is also used off‑label for alkaptonuria, where it helps reduce the buildup of homogentisic acid that causes tissue discoloration.
How does nitisinone work in the body?+
Nitisinone blocks the enzyme p‑hydroxyphenylpyruvate dioxygenase (HPPD) in the tyrosine degradation pathway. By inhibiting this step, the drug prevents formation of downstream toxic metabolites such as maleylacetoacetate and fumarylacetoacetate, thereby protecting liver and kidney function.
What is the usual dose of Nitisinone Dipharma?+
The specific dose of Nitisinone Dipharma is determined by the prescribing physician based on the patient’s age, weight, disease severity, and laboratory monitoring. Healthcare professionals tailor the regimen to achieve optimal metabolic control while minimizing adverse effects.
Is Nitisinone Dipharma safe during pregnancy or breastfeeding?+
Safety data for nitisinone in pregnancy and lactation are limited, and the pregnancy category is unknown. It should be used only if the potential benefit justifies the possible risk to the fetus, and breastfeeding decisions should be made in consultation with a qualified healthcare provider.
How do I order Nitisinone Dipharma from GNH USA?+
To place an order, submit an enquiry on the product page at gnh-usa.com, specifying the required quantity. GNH USA supplies hospitals, pharmacies and procurement teams worldwide, and will request trade or institutional credentials before providing pricing, availability, shipping details and any necessary import documentation.
How does Nitisinone Dipharma compare with other treatments for hereditary tyrosinemia type I?+
Nitisinone is the primary disease‑modifying therapy for hereditary tyrosinemia type I, directly targeting the metabolic defect. Alternative approaches, such as liver transplantation, address end‑stage disease but do not correct the underlying enzyme deficiency. Nitisinone therefore offers a less invasive, pharmacologic option that can delay or avoid transplantation.
What are the recommended storage conditions for Nitisinone Dipharma?+
Nitisinone Dipharma should be stored at temperatures below 25 °C, in a dry place away from direct sunlight. Keep the product in its original packaging to protect it from moisture and handle it according to the label instructions to maintain stability.
How is Nitisinone Dipharma administered?+
The medication is supplied as an oral formulation and should be taken by mouth with water, usually once daily or as directed by the prescriber. Doses are often adjusted based on regular monitoring of blood and urine metabolites, as well as liver function tests.
What are the most serious risks associated with Nitisinone Dipharma?+
Serious risks include severe liver injury, hypoglycemia, and peripheral neuropathy. Patients should be monitored closely for signs of hepatic dysfunction, unexplained low blood sugar, or neurological symptoms, and any concerning findings should prompt immediate medical evaluation.
Who should not use Nitisinone Dipharma?+
Individuals with known hypersensitivity to nitisinone or any of its excipients should avoid the product. Caution is also advised for patients with pre‑existing severe liver disease, uncontrolled renal impairment, or those taking concomitant medications that pose a high risk of hepatotoxicity.
Information provided on this page is for educational use by procurement and clinical professionals. Always consult a licensed healthcare provider for medical advice and prescribing decisions. GNH USA supplies this product subject to applicable regulatory and licensing requirements.
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Product details

Drug
Nitisinone
Route
Oral Use
Manufacturer
Dipharma Arzneimittel Gmbh
Origin
EU

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