If you have questions about NITYR, 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 the NITYR Patient Liaison Services.
Q: What is NITYR?
A: NITYR (nitisinone) Tablets are a new 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.
Q: Is NITYR also NTBC?
A: Yes, NITYR contains the active ingredient nitisinone. NTBC is an abbreviation for nitisinone and the two terms mean the same thing.
Q: Do the tablets have any flavor?
A: NITYR is tasteless.
Q: Who manufactures NITYR?
A: Cycle Pharmaceuticals manufactures NITYR in Switzerland.
Q: How do you deliver this medication?
A: The medication is delivered directly to a location of your choice and comes with a convenient proactive refill program.
Q: Can I get a free sample of NITYR to try?
A: Yes, with the NITYR Experience, you can try NITYR, compare it to your current nitisinone medication and decide if NITYR is right for you. You can start with the 30-Day Free NITYR Program** with:
- No financial obligation
- No commitment
- No impact on your current nitisinone
The 30-Day Free NITYR Program gives you the opportunity to decide if NITYR is right for you and your family. During the Program, you will receive calls from our NITYR Patient Liaison Services Team, including an initial call to discuss NITYR and explain how to administer the medication. If you have any questions before, during or after the Program, our NITYR Patient Liaison Services are available on 1-800-775-5783. For more information on the 30-Day Free NITYR Program click here.
Q: Do I need to keep NITYR in the fridge?
A: No, NITYR is stable at room temperature between 68° and 77°F (20° to 25°C), allowing for excursions between 59° and 86°F (15° and 30°C) until its expiry date.
Q: Are there any adverse reactions associated with taking NITYR?
A: NITYR has the same efficacy and safety profile as your current nitisinone (NTBC) treatment.
The most common adverse reactions (>1%) to taking NITYR are elevated tyrosine levels, thrombocytopenia, leukopenia, conjunctivitis, corneal opacity, keratitis, photophobia, eye pain, blepharitis, cataracts, granulocytopenia, epistaxis, pruritus, exfoliative dermatitis, dry skin, maculopapular rash and alopecia.
Q: How do you administer NITYR to small babies and toddlers?
A: NITYR tablets are very small2 (baby aspirin sized) and can be easily swallowed by patients from 3 years old. However, the ability of the patient to swallow the tablet should be established by your HCP (a 30-Day Free Program is available**).
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 added to applesauce. Further information and a short video are available here.
Q: From what age can children swallow NITYR tablets?
A: 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 (a 30-Day Free Program is available**).
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 Patient Liaison Services 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 1-800-775-5783.
Q: Does NITYR have a patient support program?
A: Yes, we provide the NITYR Patient Support Program for US patients and caregivers. You can call the Patient Support Team and gain advice and support from a specialist pharmacist 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 1-800-847-8714 or visit here.
Q: What other support do you offer to patients?
As part of the Low-Protein Food Program you will receive a $400 monthly contribution to spend on Cambrooke Therapeutics’ specialist foods.
For questions on this program, call the Cambrooke team on 1-866-456-9776.
Q: What do I do if I miss a dose?
A: 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 the NITYR Patient Support Program on 1-800-847-8714 for further advice.
Q: What can I or my child eat during treatment with NITYR?
A: 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 dietician skilled in helping people manage amino acid disorders for specific dietary instructions. Dietitian consultations are available through the NITYR Patient Nutrition Program§.
Q: My son/daughter has no symptoms and has been feeling good for a while, can I stop the medication?
A: 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.
Q: How does NITYR work?
A: 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.
Q: I am not in the United States, how can I receive NITYR?
A: For NITYR availability in other countries please contact: firstname.lastname@example.org
Additional FAQs for Patients Moving to NITYR
Q: Why should I move to NITYR? And how do I do it?
A: NITYR (nitisinone) Tablets may be more convenient for you and your family. Moving to NITYR is easy to do and a 30-Day Free NITYR Program** is available. Discuss with your healthcare provider. A new Consultation Guide is available to help patients and caregivers navigate questions with their HCP about choosing NITYR.
Q: How is NITYR different from the nitisinone I’ve been prescribed before?
A: 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° to 77°F (20° to 25°C), allowing for excursions between 59° and 86°F (15° and 30°C), until its expiry date, freeing you from having to refrigerate the medication.
NITYR tablets are tasteless and 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.
Q: Is NITYR the same as Orfadin* (nitisinone) capsules?
A: 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
Q: Are the adverse reactions different from Orfadin* capsules?
A: Because NITYR (nitisinone) Tablets are bioequivalent to Orfadin* capsules, NITYR has the same efficacy, safety and adverse reactions.1
Q: Will NITYR cost me more than my current nitisinone medication?
A: Our team can make a full benefit investigation to tell you how much NITYR will cost. We also offer $0 Co-pay for eligible patients.||
Talk to your healthcare provider and ask them to complete a 30-Day Free NITYR Program** enrollment form. The results will help you with the decision.
Q: Will there be adverse reactions associated with moving from my current nitisinone to NITYR?
A: No, patients who move to NITYR are not expected to experience any new adverse reactions. The most common adverse reactions for nitisinone (>1%) are elevated tyrosine levels, thrombocytopenia, leukopenia, conjunctivitis, corneal opacity, keratitis, photophobia, eye pain, blepharitis, cataracts, granulocytopenia, epistaxis, pruritus, exfoliative dermatitis, dry skin, maculopapular rash and alopecia. Click here for full adverse events and prescribing information.
*Orfadin is a registered trademark licensed by Sobi, Inc. or its affiliates. **Free NITYR Program: Patients can try NITYR for 30 days, one time, for free, as long as their doctor prescribes it. Subject to terms and conditions, eligibility criteria, and other federal and state law. Terms and Conditions and eligibility criteria are available at www.nityr.us/offertcs. §Patient Nutrition Program: Subject to terms and conditions and eligibility criteria. This program is free of charge for patients with a valid prescription for NITYR issued by their physician or other HCP, or commercially insured patients on the 30 Day Free NITYR Program and is subject to other federal and state law. Terms, conditions and eligibility criteria are available at www.nityr.us/offertcs. ||$0 co-pay for eligible commercially insured patients within specified limits and relevant terms and conditions. A prescription is needed. References: 1. Data on file: NIT/02/20 2. Data on file: NIT/03/18
(Es usted un Paciente/familiar residente en Estados Unidos?)NO * YES (Sí)
* If you select "no" then you will be directed to the Cycle Pharmaceuticals website.