Orfadin (Nitisinone)

Save over 90% on our brand and generic Orfadin vs US generic Orfadiin.

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Orfadin is stable at room temperature for 60 days and we ship 60 at a time maximum.  Generic is room stable - see Nityr also for room stable versions of this medicine.

For brand Orfadin, Non-english packaging is often present for European medicines- English label and English insert included. Plan in advance - allow us 7 days to ship this brand name medicine.

According to the European Medicines Agency, brand name Orfadin originating from Europe is manufactured at the following sites:Apotek Produktion & Laboratorier AB Celsiusgatan 43 SE-212 14 Malmö SwedenApotek Produktion & Laboratorier AB Prismavägen 2 SE-141 75 Kungens Kurva Sweden

Information about Orfadin (Nitisinone)

Orfadin (Nitisinone) is a prescription medication primarily used for the treatment of hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder. It works by inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase (HPPD), which is involved in the breakdown of the amino acid tyrosine. By inhibiting this enzyme, Orfadin helps to lower the toxic byproducts associated with HT-1, preventing liver damage, kidney problems, and neurological issues in patients with the condition.

Product Highlights

• Orfadin is primarily used to treat HT-1, a life-threatening metabolic disorder that results from a deficiency of the enzyme fumarylacetoacetate hydrolase (FAH). 
• The disease leads to the accumulation of toxic metabolites (including succinylacetone), which cause severe liver and kidney damage and can affect the nervous system.

Key Ingredient

• Nitisinone

Key Benefits of Orfadin (Nitisinone)

• By inhibiting HPPD, Orfadin reduces the production of toxic metabolites like succinylacetone, thus protecting the liver, kidneys, and nervous system from damage.
• The drug has been shown to improve survival rates and prevent the progression of HT-1 in patients when started early.
• Prevents liver failure and kidney dysfunction, two of the major complications associated with untreated HT-1.
• Prevents or improves neurological symptoms such as developmental delays, movement disorders, and other complications related to HT-1.

Direction of Use

• Orfadin is taken by mouth, typically once or twice daily.
• The starting dose is generally based on body weight (1 mg per kg of body weight, adjusted according to clinical response).
• The dose may be adjusted based on the patient’s response, laboratory tests (such as tyrosine levels), and clinical status.
• For patients who have difficulty swallowing capsules, Orfadin is also available in an oral suspension form.
• It is typically taken with or without food, but it is important to follow the specific dosing instructions provided by the prescribing physician.

Safety Concerns

• Eye-related issues like corneal changes (typically seen with long-term use) and conjunctivitis are frequent. Routine eye exams are advised throughout the course of treatment.
• Some patients may develop a rash or skin irritation.
• Monitoring liver function tests is important to ensure liver health during treatment.
• Orfadin can interact with certain medications, including other drugs that affect liver function. It is essential to notify the healthcare provider of all other medications being used.
• Regular blood tests, including liver function tests and tyrosine levels, are necessary during treatment to ensure optimal dosing and minimize the risk of side effects.

Avoid Orfadin (Nitisinone) If

• If you have a known hypersensitivity or allergic reaction to nitisinone or any other components of the formulation, you should not use Orfadin.
• The safety of Orfadin during pregnancy and breastfeeding has not been established. It is important to discuss potential risks with a healthcare provider before using it during pregnancy or lactation.
• If you have a history of significant eye problems, such as corneal disorders, you may be advised against using Orfadin due to its potential effects on the eyes.
• While Orfadin is used to treat HT-1-related liver disease, caution may be required in patients with other forms of severe liver disease that are not related to HT-1.