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Orfadin
Nitisinone
$1145.00
2MG · 60 capsules
BrandBranded medicine· By Apotek Produktion & Laboratorier AB
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2MG
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About Orfadin
Overview
Orfadin capsules contain nitisinone and require a prescription. They are used to treat hereditary tyrosinemia type 1 (HT-1) in adults and children, together with a diet that restricts tyrosine and phenylalanine. Nitisinone blocks an enzyme involved in breaking down tyrosine, helping prevent the buildup of toxic substances that can harm the liver and kidneys.
What it treats
Hereditary tyrosinemia type 1 (HT-1) in adults and children, alongside dietary restriction of tyrosine and phenylalanine.
Safety information
Side effects
Common side effects include raised blood tyrosine levels; low platelet or white blood cell counts; eye inflammation, pain, clouding or cataracts; sensitivity to light; nosebleeds; itching, dry or peeling skin; rash; and hair loss. Contact your doctor if you develop eye symptoms, a sudden change in neurological function, or painful, thickened patches of skin on your palms or soles. Raised tyrosine levels can cause eye problems, intellectual disability and developmental delay, particularly if dietary tyrosine and phenylalanine are not adequately restricted. Severe reductions in platelet counts and low white blood cell counts can also occur. Regular eye examinations and blood tests are needed to monitor these risks.
When to call your doctor
If symptoms are severe or getting worse quickly, seek emergency care rather than waiting.
Before taking this medicine
The label lists no contraindications for Orfadin. Careful restriction of dietary tyrosine and phenylalanine is required. Inadequate restriction can raise blood tyrosine levels, which may cause eye symptoms, intellectual disability, developmental delay or painful, thickened patches of skin on the palms and soles. Do not change the Orfadin dose to lower tyrosine levels. A slit-lamp eye examination is needed before treatment and regularly during treatment. Repeat examination is needed if eye symptoms develop or blood tyrosine exceeds 500 micromol/L. A sudden change in neurological status requires a blood tyrosine check. Low white blood cell counts and severely low platelet counts can occur. These blood counts should be monitored.
Interactions
Orfadin can increase levels of certain other medicines in the body. Tell your doctor if you take celecoxib, tolbutamide, phenytoin or warfarin, as your prescriber may need to reduce their doses. Orfadin can also increase levels of medicines such as adefovir, ganciclovir and methotrexate. Your doctor should monitor for side effects related to these medicines.
Using Orfadin
Dosing
Dosing is set by your prescriber. Each health care professional may prescribe medication differently according to a patient's medical history and condition, so follow the directions on your prescription label and speak to your doctor or pharmacist before changing anything.
Storage
Store below 25°C in a dry place, in the original pack, away from direct light and moisture. Do not keep it in a bathroom. Keep all medicines out of the sight and reach of children.
Clinical information on this page is summarised from the US FDA label ORFADIN (NITISINONE) CAPSULE [SWEDISH ORPHAN BIOVITRUM AB (PUBL)] (DailyMed, retrieved 19 September 2026).
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