FAQs

If you have questions about NITYR® (nitisinone) Tablets, you are not alone. We have compiled some Frequently Asked Questions (FAQs) to help, but if you have any other questions then get in touch with Cycle Vita™.

NITYR (nitisinone) Tablets are an option for the treatment of Tyrosinemia Type 1. NITYR is bioequivalent1 to Orfadin®* and contains the same nitisinone (NTBC) you are used to, but NITYR has been formulated in a way that can provide greater convenience for patients and their families.

Yes, NITYR contains the active ingredient nitisinone. NTBC is an abbreviation for nitisinone and the two terms mean the same thing.

Cycle Pharmaceuticals manufactures NITYR in UK and Switzerland.

The medication is delivered directly to a location of your choice and comes with a convenient proactive refill program.

No, NITYR is stable at room temperature between 68°F and 77°F (20°C to 25°C), allowing for excursions between 59°F and 86°F (15°C and 30°C) until its expiry date.

NITYR has the same efficacy and safety profile as your current nitisinone (NTBC) treatment.1

Adverse reactions

The most common adverse reactions (≥1%) in patients with HT-1 taking nitisinone are elevated tyrosine levels, low platelets (thrombocytopenia) or white cells in the blood (leukopenia), and complaints related to the eyes, including conjunctivitis, corneal opacity, inflammation of the cornea, eye pain, blepharitis, cataracts, granulocytopenia and extreme sensitivity to light (photophobia), nosebleed (epistaxis), itching (pruritus), skin inflammation (exfoliative dermatitis), rash (maculopapular rash), dry skin and alopecia.

NITYR tablets are very small2 (baby aspirin sized) and can be swallowed by patients from 3 years old. However, the ability of the patient to swallow the tablet should be established by your healthcare provider.

For those unable to swallow a tablet, NITYR can be converted to a suspension that can be administered via an oral syringe. The suspension can be stored at room temperature for up to 24 hours before use. If your child can swallow soft food, NITYR Tablets can also be crushed and mixed with applesauce. Further information and a short video are available here.

Generally, children aged 3 years and older can swallow NITYR Tablets (baby aspirin sized2). However, check with your healthcare provider before administering NITYR Tablets to young children.

Children under 3 years old can take NITYR by creating a suspension using an oral syringe (watch video here) or by crushing the tablets and mixing them with applesauce. The suspension can be stored at room temperature for up to 24 hours before use. Our Cycle Vita Team can provide training, oral syringes and tablet crushers free of charge and give you tips on how to teach your child to swallow NITYR Tablets. Please call 888-360-8482.

Yes, we provide the Cycle Vita support program for US patients and caregivers. You can call Cycle Vita and gain advice and support when you need help or have any questions. The program also includes delivery of your medication to your home and proactive refills. For more information call 888-360-8482 or visit www.cyclevita.life.

If you forget a dose, take the next dose as planned. Do not take a double dose to make up for a forgotten dose. Contact your healthcare provider or Cycle Vita on 888-360-8482 for further advice.

You or your child should follow a low-protein diet and avoid foods and drinks that contain phenylalanine or tyrosine. You should work with your healthcare provider and consult a metabolic dietitian skilled in helping people manage amino acid disorders for specific dietary instructions. Dietitian consultations are available through the NITYR Patient Nutrition Program**.

No. Tyrosinemia Type 1 is a lifelong condition and it’s important to maintain recommended medication and dietary routines to lower the risk of adverse health outcomes.

NITYR Tablets contain nitisinone, which prevents the build-up of toxic substances which cause severe liver and kidney problems in patients with Tyrosinemia Type 1. Patients still need to follow a low-protein diet, restricting the intake of phenylalanine and tyrosine to control tyrosine levels in the body.

For NITYR availability in other countries please contact: sales-distribution@cyclepharma.com

Additional FAQs for Patients Moving to NITYR

NITYR (nitisinone) Tablets may be more convenient for you and your family. Moving to NITYR is easy to do. Discuss with your healthcare provider. A Consultation Guide is available to help you navigate questions with your healthcare provider about choosing NITYR.

NITYR (nitisinone) Tablets are bioequivalent to Orfadin®* (nitisinone) capsules, which means they have the same efficacy, safety and side effects, and may offer patients and their families greater convenience.1

NITYR is also stable at room temperature between 68°F to 77°F (20°C to 25°C), allowing for excursions between 59°F and 86°F (15°C and 30°C), until its expiry date, freeing you from having to refrigerate the medication.

NITYR tablets are very small2 (baby aspirin sized) and come in 2 mg, 5 mg and 10 mg tablets. NITYR can also be taken with or without food.

NITYR is bioequivalent to Orfadin®*, which means that it has the same efficacy, safety and side effects, and may offer you and your family greater convenience.1

Because NITYR (nitisinone) Tablets are bioequivalent to Orfadin®* capsules, NITYR has the same efficacy, safety and adverse reactions.1

Our Cycle Vita team will complete a benefits investigation to help understand your coverage and how much NITYR may cost you. We also offer $0 co-pay for eligible patients.**

No, patients who move to NITYR are not expected to experience any new adverse reactions. The most common adverse reactions (≥1%) in patients with HT-1 taking nitisinone are elevated tyrosine levels, low platelets (thrombocytopenia) or white cells in the blood (leukopenia), and complaints related to the eyes, including conjunctivitis, corneal opacity, inflammation of the cornea, eye pain, blepharitis, cataracts, granulocytopenia and extreme sensitivity to light (photophobia), nosebleed (epistaxis), itching (pruritus), skin inflammation (exfoliative dermatitis), rash (maculopapular rash), dry skin and alopecia. Click here for full adverse events and prescribing information.

* Orfadin is a registered trademark licensed by Sobi, Inc. or its affiliates.
** Some areas of support may not be accessible to all patients. Eligibility criteria may apply to ensure compliance with all applicable federal and state requirements, and benefits may be limited to commercially insured patients only. For more detailed information about eligibility, terms and conditions, please contact the Cycle Vita team at 888-360-8482.

References

1. Data on file: REF-00047
2. Data on file: REF-00041