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FAQs

What is Orfadin (Nitisinone)?

Nitisinone is a prescription medication used to treat hereditary tyrosinemia type 1 (HT-1), a rare metabolic disorder. It works by blocking the breakdown of tyrosine, reducing the toxic byproducts that cause organ damage.

Learn more about Orfadin here

What conditions does Nitisinone treat?

Nitisinone is primarily prescribed for:

  • Hereditary tyrosinemia type 1 (HT-1): A metabolic disorder that can lead to liver and kidney damage if untreated.

It is also being studied for off-label use in other conditions, such as alkaptonuria, a rare metabolic condition. Read about it here.

Is there a generic version of Nitisinone available?

Yes, generic versions of Orfadin and/or Nityr and may be less expensive than brand-name versions like Orfadin or Nityr. Availability and pricing can vary depending on your location and pharmacy as this generic was recently approved (Nov. 2024) and may take time to reach the entire market. Either way, we guarantee we’ll always give you the best price possible along with aiding access to copay assistance.

Is there a brand name for Nitisinone?

Yes, nitisinone is sold under the brand names Orfadin and Nityr, depending on the manufacturer and formulation.

What if I can't afford Orfadin or Nityr?

Copay Patient Assistance may be right for you! We believe that high deductibles and copays shouldn’t prevent anyone from receiving the medications they need. That’s why at QuickRx, we offer assistance accessing Orfadin or Nityr Copay Assistance Programs. This is designed to help patients reduce their monthly out-of-pocket costs.

Our program partners with many foundations and manufacturer assistance programs to help make medication affordable. Fill out the form on this page and someone from our dedicated team will be in touch within one business day. 

Give us a call to see if you qualify. Not only are we dedicated to helping all who may qualify with access to copay assistance programs, but we are also committed to giving every person their lowest price possible, even if you aren’t using prescription coverage.

What is Orfadin or Nityr Copay Assistance?

Orfadin/Nityr Copay Assistance comes in the form of various savings programs, which are often referred to as “Orfadin copay cards.” These patient assistance programs can be very difficult for patients to find, enroll, and keep an active status.
Although these programs are free, many people are not aware they exist. That is where QuickRx comes in. We have a highly-skilled team dedicated to helping make prescription copays affordable for all who qualify by facilitating access to these programs.

How does the Orfadin/Nityr Copay Card Assistance program work?

The Orfadin/Nityr Copay Assistance programs each have their own set of rules and regulations. This is the part of the process that has proven to be frustrating for many people seeing copay assistance.

However, with QuickRx your experience will be anything but! Our dedicated team will work to make the Copay Card process as seamless as possible for you. In fact, your part of the process involves just a few short, easy steps to find out if you qualify for copay assistance.

 

What is the difference between Orfadin and Nityr?

In formula there is no difference between these two medications. However, on Nityr’s website the manufacturer highlights that Nityr is “baby asprin-sized” and is 20% smaller than Orfadin.

See the Nityr site here

See the Orfadin site here

Are these medications covered by insurance?

Orfadin and/or Nityr insurance coverage may vary depending on the individual’s insurance plan and the specific circumstances of their treatment. It is recommended to check with your insurance provider to determine your personal coverage details and copay amount.

What are the steps to find out if I qualify for Orfadin or Nityr (Nitisinone) copay assistance?

Step 1:
Get started using one of these options;

Step 2:
Fill out some easy forms

Step 3:
Submit a few pieces of documentation to our team.

That’s it! From there, our team will handle everything to see if you qualify. If you meet the qualification guidelines, our team will ensure that your discounts are automatically applied for you at the pharmacy, coordinate pickup or home delivery of your medications, and they’ll even continue to monitor your case! With QuickRx you will have peace of mind knowing that you will never have any surprise lapse in copay assistance benefits.

What are the side effects of Nitisinone?

Common Side Effects

  • Elevated tyrosine levels (tyrosinemia type II-like symptoms), which may lead to:
    • Sensitivity to light (photophobia)
    • Eye pain or discomfort
    • Corneal clouding
    • Dryness or redness in the eyes
    • Keratitis (inflammation of the cornea)
  • Headache
  • Skin irritation or redness
  • Itchy skin (pruritus)

Gastrointestinal Side Effects

  • Nausea
  • Diarrhea
  • Vomiting
  • Abdominal pain

Liver and Kidney Effects

  • Increased liver enzyme levels (requires regular monitoring)
  • Potential for kidney issues with long-term use

Other Side Effects

  • Fatigue or weakness
  • Fever

Rare but Serious Side Effects

  • Severe eye complications:
    • Corneal ulcers or deposits (if tyrosine levels remain elevated and unmanaged)
  • Liver damage (if improperly monitored)
  • Hypersensitivity or allergic reactions, including:
    • Swelling of the face or throat
    • Difficulty breathing
    • Rash or hives

If you believe you may have these or any of side effects do not wait to contact your doctor. It is important to manage side effects through regular monitoring of tyrosine levels, adhering to a low-tyrosine and low-phenylalanine diet. Consult a healthcare provider for your specific plan of care.

Is monitoring required while on Nitisinone?

Yes, regular monitoring is essential while on nitisinone. Your healthcare provider will monitor:

  • Tyrosine levels in the blood
  • Liver function
  • Kidney health
  • Eye health
Can children take Nitisinone?

Yes, nitisinone is approved for use in children, including infants, for the treatment of hereditary tyrosinemia type 1 (HT-1). Dosage may vary depending on weight and specific needs. Need and ability to tolerate any medication should always be determined by a qualified healthcare professional.

How should Nitisinone be taken?

Nitisinone is usually taken orally in capsule or liquid suspension form. It should be taken as directed by your healthcare provider, either with or without food. Patients on nitisinone typically follow a tyrosine- and phenylalanine-restricted diet.