Orfadin

(Nitisinone)

Hereditary tyrosinemia type 1 (HT-1) can only be treated, in combination with dietary restriction of tyrosine and phenylalanine, with the brand-name medication Orfadin. It is used orally, usually once or twice a day, and is available as a capsule and suspension. Patients with HT-1 and AKU lack enzymes to break down the amino acid tyrosine properly, and, as a result, it is converted to harmful substances.

Patients with HT-1 are also at increased risk of developing liver failure, liver cancer, and renal (kidney) disease. These complications of HT-1 were observed in patients treated with nitisinone.

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Generic Versions
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NitisinonePrescription Required

Strength
2mg
Country
India
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Marketed as Lasinone in India

NitisinonePrescription Required

Strength
5mg
Country
India
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Marketed as Lasinone in India

NitisinonePrescription Required

Strength
10mg
Country
India
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Marketed as Lasinone in India

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Important information about Orfadin

How does Orfadin work?

The active ingredient in Orfadin is nitisinone, which is a 4-hydroxyphenylpyruvate dioxygenase (HPPD) inhibitor. HPPD is an enzyme in your body that aids in the breakdown of tyrosine from food. The mechanism of action involves blocking this enzyme to prevent tyrosine from being broken down early in the process and turning into harmful toxins. When used as an adjunct to dietary restriction of tyrosine and phenylalanine, it can be effective in the treatment of hereditary tyrosinemia type 1 (HT-1).

How do you use Orfadin?

Follow all directions given to you by your doctor and read all documents provided, including the full Prescribing Information, FDA-Approved Product Labeling, Instructions for Use, and Medication Guide, before using Orfadin. However, the following is a synopsis of the general guidelines for the use of Orfadin:

  • Orfadin oral suspension can be taken with or without food. Orfadin capsules should be taken at least 1 hour before or 2 hours after a meal.
  • If you want your treatment to work, you have to avoid specific foods. Adhere to the diet plan that your physician or nutritionist has developed for you.
  • Tyrosine and phenylalanine (including artificial sweeteners like aspartame, Equal, or Nutrasweet) are found in diets high in protein. Special foods have been created to substitute high-protein foods since newborns and kids require protein to grow and thrive.
  • Before measuring a dose, shake the liquid oral suspension for at least five seconds. Use a medication dose-measuring tool like an oral syringe (not a kitchen spoon) or the supplied dosing syringe.
  • Open the capsule and mix the medication into a teaspoon of water, infant formula, or applesauce if you or your child is unable to swallow it whole. Without chewing, immediately swallow the concoction. Don't put it away until later.
  • Monitor plasma and/or urine succinylacetone concentrations, liver function parameters, and alpha-fetoprotein levels.

What are the side effects of Orfadin?

The most common side effects of Orfadin include:

  • Elevated tyrosine levels, which can lead to toxicity to your eyes, skin, and nervous system.
  • Blepharitis.
  • Cataracts.
  • Keratitis, inflammation (redness, irritation) of the cornea.
  • Exfoliative dermatitis may occur uncommonly.
  • Changes to your vision or blurred vision.
  • Granulocytopenia (low levels of granulocytes, a type of white blood cell).
  • Eye pain.
  • Conjunctivitis (pink eye).
  • Corneal opacity (clouding of the cornea, the transparent layer that covers your pupil).
  • Photophobia (sensitivity to light).
  • Thrombocytopenia (reduced levels of platelets).
  • Low white blood cells (leukopenia).
  • Pruritus or bronchitis.

Some individuals may experience serious side effects while using Orfadin. These side effects may require immediate medical attention. If you experience any of the following, contact your healthcare provider or seek emergency medical help:

  • Symptoms of an allergic reaction such as hives, trouble breathing, or swelling of your face, lips, tongue, or throat.
  • Developmental delays in children (e.g., sitting up, crawling, walking, talking, etc.).
  • The liquid form of nitisinone may contain glycerol and can cause a headache, upset stomach, or diarrhea.
  • Unusual bleeding from the mouth, nose, vagina, or rectum, easy bruising, and red or purple pinpoint areas beneath the skin.
  • Jaundice (eye or skin yellowing).

These are not all of the possible adverse events of Orfadin. You should always seek medical advice from your healthcare provider for any questions or concerns about your medical condition or treatment. Read all patient information, medication guides, or drug information sheets that come with this medication. You can also report adverse effects to the Food and Drug Administration at www.fda.gov/medwatch or 1-800-FDA-1088.

What are the warnings or precautions for Orfadin?

Before starting Orfadin, it is crucial to discuss any existing health conditions with your healthcare provider. You should be sure that your healthcare provider is aware of all your medical conditions, including whether you have:

  • Medical conditions affecting your eyes.
  • Low blood counts, including low platelet or white blood cell counts.

Are there any contraindications with Orfadin?

There are no specific contraindications of Orfadin. However, if you are allergic or have any other adverse reactions to the medication, you should avoid taking it.

How long does it take Orfadin to work?

It usually takes about one week for patients to respond to the medication.

What interacts with Orfadin?

When Orfadin is taken with other prescription drugs, over-the-counter medications, vitamins, and supplements, it may change how they work or increase the frequency or severity of side effects. Make sure that you tell your healthcare professional about anything that you are taking to avoid any negative drug interactions, including:

Celecoxib (Celebrex), phenytoin (Dilantin), warfarin (Coumadin), and methotrexate are among the medications that interact with nitisinone.

What doses of Orfadin are available?

Orfadin is available in 2 mg, 5 mg, 10 mg, and 20 mg capsules. It is also available as a 4 mg/mL oral suspension.

For the treatment of hereditary tyrosinemia type 1, your body weight determines how much nitisinone you need to take. The usual starting dose of Orfadin is 0.5 mg/kg/day twice daily.

Your doctor may change your dosage in response to side effects and blood levels of specific substances.

How do I store Orfadin?

Do not freeze the liquid or capsules; instead, keep them in the refrigerator. After using Orfadin liquid for the first time, you may store the medicine at room temperature for up to 60 days.

Which pharmaceutical company manufactures Orfadin?

Swedish Orphan Biovitrum (Sobi) is a pharmaceutical company that manufactures Orfadin.

Is there a generic version of Orfadin?

There are several lower-cost generics of Orfadin available.

The drug nitisinone is marketed under the brand names Nityr and Orfadin, and both are effective in treating HT-1 symptoms.

How do I stop taking Orfadin?

Consult your doctor before stopping or reducing your dosage of Orfadin. Your disease can get worse, or you might get unintended adverse effects if you abruptly stop taking it.

Can Orfadin be used if I am pregnant?

Talk to your doctor if you are already pregnant or if you have any plans to become pregnant. They will know or recommend what is best for you.

Can Orfadin be used if I am breast feeding?

It is not known if Orfadin is found in breast milk during lactation. Talk to your doctor if you are already breastfeeding, or if you have any plans to breastfeed. They will know or recommend what is best for you.

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Important information about buying Orfadin from UniversalDrugstore™

What happens after I place my order for Orfadin with Universal Drugstore?

Once you complete your order, we process, verify, and dispatch your medication. You’ll receive email updates at each stage, including tracking details once your order ships. If any additional documentation is needed (such as a valid prescription), we’ll contact you promptly. Once approved, your order is charged and delivered directly to your doorstep.

How long does it take to process and deliver my order for Orfadin?

Orders are typically processed within 1–3 business days once all required documents are received and approved. Standard delivery times range from 10–18 business days, depending on your location.

Can I track my order of Orfadin?

Yes! Once your medication ships, you will receive an email with tracking information so you can monitor your shipment every step of the way.

What should I do if there is an issue with my order for Orfadin?

Our Customer Care team is available 24/7 to assist with any order-related concerns or questions. If you experience delays, missing items, or need support, contact us using the details provided in your confirmation email.

How is my medication packaged to ensure privacy and safety?

All orders are shipped in discreet, plain packaging to protect your privacy. Medications are securely packaged to prevent damage and ensure they arrive in top condition.

Does Universal Drugstore ship medications that require refrigeration or special handling?

Yes. We use cold-chain shipping methods to safely transport temperature-sensitive medications, ensuring they maintain proper storage conditions throughout delivery.

What are the shipping costs, and does Universal Drugstore offer free delivery?

Shipping costs vary depending on your location and shipping method. Free shipping promotions may be available—check the checkout page for the latest offers. Orders above $100 going to a US destination will receive free shipping.

Does Universal Drugstore accept Medicare or Medicaid for prescriptions?

Currently, we do not bill Medicare or Medicaid directly. However, we provide detailed receipts and documentation that you can submit to your insurance provider for potential reimbursement.

Can I use private health insurance to cover my prescription from Universal Drugstore?

Universal Drugstore does not bill private health insurance directly, but we provide a detailed invoice that you can submit to your insurer for possible reimbursement. Many insurers will accept Unievrsal Drugstore’s documents, but if you have any questions, check with your insurance company prior to purchasing.

How secure is my payment information when ordering from Universal Drugstore?

We use advanced SSL encryption and secure payment processing to ensure that all transactions are 100% protected against fraud and unauthorized access.

Can I order a generic alternative to Orfadin?

Universal Drugstore offers many generic alternatives that provide the same active ingredients at a lower cost. Search for your medication and compare pricing for generic vs. brand-name options. Generic availability depends on the medication.

Do I need a prescription to order Orfadin?

If your medication requires a prescription, you must upload it online, via email, or fax. Over-the-counter (OTC) medications do not require a prescription. All prescription medication purchases require a valid and approved prescription.

Can I order multiple medications at once?

Yes! You can add multiple medications to your cart, including prescription, OTC, and pet medications, and complete your purchase in a single checkout process.

Can I refill my prescription through Universal Drugstore?

Yes! If your prescription includes refills, you can reorder quickly by logging into your account and selecting your medication for a hassle-free refill process. Always refill your medication proactively and ontime, to ensure you do not run out of important medication.

Does Universal Drugstore offer discounts or price matching?

Yes! We offer:

  • Up to 80% savings on medications
  • Price match guarantee – we’ll match competitor pricing on qualifying medications
  • 10% off your first order for new customers

How can I contact Universal Drugstore for additional support?

We offer 24/7 customer support via phone, email, and chat. Visit our Contact Us page for assistance with ordering, shipping, or medication questions.

Important

The information, including but not limited to, text, graphics, images and other material contained on this website are for informational purposes to increase health awareness only. No material on this site is intended to be a substitute for professional medical advice, diagnosis or treatment. 



Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a medical condition or treatment and before undertaking a new health care regimen, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.