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Orfadin
Orfadin (generic name: nitisinone) is a medication used to treat hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder that affects the body's ability to break down the amino acid tyrosine. If left untreated, HT-1 can lead to serious liver, kidney, and neurological problems. Orfadin works by blocking the enzyme 4-hydroxyphenylpyruvate dioxygenase (HPPD), reducing the production of toxic byproducts that accumulate in HT-1 patients. When used in combination with a low-tyrosine and low-phenylalanine diet, Orfadin helps prevent the serious health complications associated with HT-1.
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Frequently Asked Questions
How long do I need to take Orfadin?
Orfadin is typically a lifelong treatment for hereditary tyrosinemia type 1, used to prevent complications associated with the condition. Patients must continue taking it and adhering to their dietary restrictions unless advised otherwise by their doctor.
Can Orfadin cure hereditary tyrosinemia type 1?
No, Orfadin does not cure HT-1, but it helps manage the disease by preventing the buildup of toxic substances that can damage the liver and kidneys.
Do I need regular monitoring while taking Orfadin?
Yes, you will need regular blood tests to monitor your liver function, blood cell counts, and levels of succinylacetone and tyrosine. You may also need eye exams to check for any complications from elevated tyrosine levels.
Can children take Orfadin?
Yes, Orfadin is used in both children and adults with hereditary tyrosinemia type 1. The dosing is typically based on body weight, and the medication is part of a comprehensive treatment plan that includes dietary management.
What should I do if I miss a dose of Orfadin?
If you miss a dose, take it as soon as you remember. If it is close to your next scheduled dose, skip the missed dose and resume your normal dosing schedule. Do not double up on doses.
Comprehensive Drug Guide
How Does Orfadin Work?
Orfadin works by inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase (HPPD), which is involved in the breakdown of the amino acid tyrosine. By blocking this enzyme, Orfadin prevents the formation of harmful substances (such as succinylacetone) that can damage the liver, kidneys, and nervous system in patients with hereditary tyrosinemia type 1 (HT-1). This helps reduce the risk of liver failure, cancer, and other serious complications associated with HT-1.
Common Dosages
- Capsules: 2 mg, 5 mg, 10 mg, and 20 mg
- Oral suspension: 4 mg/mL
The typical starting dose is:
- 1 mg/kg body weight per day, divided into two daily doses, adjusted based on the patient's response and tolerance.
Typical Dosing
- For HT-1 patients: The recommended initial dose of Orfadin is 1 mg/kg/day, divided into two doses per day.
- The dose can be adjusted based on clinical response and the levels of succinylacetone in the blood and urine.
- Patients should maintain a low-tyrosine and low-phenylalanine diet while taking Orfadin.
Typical Dosing
FDA Approved Indications
- Hereditary tyrosinemia type 1 (HT-1), used in combination with a low-tyrosine and low-phenylalanine diet.
Who Shouldn't Take Orfadin?
- Known hypersensitivity to nitisinone or any other ingredients in the formulation.
Advice From The Pharmacist
- Take Orfadin twice daily, preferably at the same times each day. You can take it with or without food, but try to maintain consistency in your routine.
- If you are using the oral suspension, shake the bottle well before each use and use the provided dosing syringe to ensure accurate measurement.
- Store Orfadin capsules at room temperature and the oral suspension in the refrigerator after opening.
- Do not consume foods high in tyrosine or phenylalanine while taking Orfadin, as this can increase the risk of side effects. Stick to your low-tyrosine and low-phenylalanine diet as prescribed by your healthcare provider.
Side Effects of Orfadin
Common Side Effects
- Elevated tyrosine levels, which can lead to eye and skin symptoms such as eye irritation, photophobia (light sensitivity), and painful or red eyes.
- Thrombocytopenia (low platelet count)
- Leukopenia (low white blood cell count)
- Nausea
- Fatigue
Uncommon/Severe Side Effects
- Ocular issues (such as corneal ulcers) due to elevated tyrosine levels.
- Severe hypersensitivity reactions (such as rash, itching, swelling, or difficulty breathing).
- Liver damage, especially in patients who are not adhering to dietary restrictions.
Risks and Warnings of Orfadin
- Ocular Toxicity: High levels of tyrosine, which may occur during treatment with Orfadin, can cause eye problems like photophobia or corneal issues. Regular eye exams are recommended to monitor for these complications.
- Dietary Compliance: To avoid elevated tyrosine levels, patients must adhere to a low-tyrosine and low-phenylalanine diet. Failure to follow this diet can lead to serious side effects.
- Hematologic Effects: Orfadin can lead to low platelet and white blood cell counts. Regular blood tests are needed to monitor for these effects.
- Liver Monitoring: Regular monitoring of liver function is important, as HT-1 is associated with liver damage and Orfadin may affect liver health.
Interactions with Orfadin
Common Drug Interactions
- Vitamin K antagonists (e.g., warfarin): Orfadin may increase the risk of bleeding when taken with blood thinners like warfarin, requiring close monitoring.
- Foods high in tyrosine and phenylalanine: Consuming these foods can increase the risk of elevated tyrosine levels and associated side effects.
Alternatives to Orfadin
- Liver transplantation: In cases where medication and dietary management do not control hereditary tyrosinemia type 1, liver transplantation may be considered as an alternative treatment.
- NTBC (nitisinone): The generic form of Orfadin, used to treat hereditary tyrosinemia type 1, may be prescribed as a cost-effective alternative.
