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 Support Program.
Q: What is NITYR?
A: NITYR (nitisinone) tablets are a new option for the treatment of HT-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, supplied by the NITYR Patient Support Program.
Q: Can I get a free sample of NITYR to try?
A: The NITYR Three-Week Free Trial2 gives you the opportunity to decide if NITYR is right for you and your family.
During the trial, you will receive calls from our NITYR Patient Support 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 trial, our Nityr Patient Support Team is available on 1-800-847-8714.
The Free Trial won’t stop your current deliveries of Orfadin*.
For more information on the Free Trial click here.
Q: Do I need to keep NITYR in the fridge?
A: No, NITYR is stable3 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. It has a shelf life of 36 months (3 years).
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%) of nitisinone 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.
The warnings and precautions that come with NITYR include:
Increased levels of plasma tyrosine, eye symptoms, developmental delay and skin changes
- Inadequate restriction of tyrosine and phenylalanine intake can result in elevations of plasma tyrosine
- At plasma tyrosine levels above 500 micromol/L, symptoms can occur including intellectual disability and developmental delay or painful hyperkeratotic plaques (thickening of the skin) on the soles and palms
- Do not adjust the dosage of NITYR in order to lower the levels of tyrosine in the blood
Changes in blood profile
- Patients can develop leukopenia (reduction in the number of white blood cells, which form part of the immune system) and severe thrombocytopenia (abnormally low levels of platelets, which help the blood clot)
- Your healthcare professional will monitor platelet and white blood cell counts, and will adjust your medication accordingly
For full detail please refer to the prescribing information
Q: How do you administer NITYR to small babies and toddlers?
A: NITYR tablets are very small4 (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 Free Trial is available2).
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 (tablets are baby aspirin sized4). However, check with your healthcare professional before administering NITYR tablets to young children (a Free Trial is available2).
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 Support Program can provide oral syringes and tablet crushers free of charge and give you tips on how to teach your child to swallow NITYR tablets. Please call 1-800-847-8714.
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?
A: Cycle Pharmaceuticals has partnered with Cambrooke TherapeuticsTM to support you with the protein formulas and low protein foods you rely on. As part of the NITYR Diet and Low Protein Food Program5 you will receive a $200 monthly contribution for two years 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 professional 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 professional and consult a metabolic dietician skilled in helping people manage amino acid disorders for specific dietary instructions. Dietitian consultations are provided free of charge through the NITYR Patient Support Program. A NITYR Diet and Low Protein Food Program5 is available to eligible patients.
Q: My son/daughter has no symptoms and has been feeling good for a while, can I stop the medication?
A: No. HT-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 HT-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:
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 patients and their families. Moving to NITYR is easy to do and a Free trial2 is available. Discuss with your healthcare professional. A new Consultation Guide is available to help patients and caregivers navigate questions with their HCP about choosing NITYR. Simply download the guide here.
Q: How is NITYR different from the nitisinone I’ve been prescribed before?
A: NITYR (nitisinone) tablets are bioequivalent1 to Orfadin* (nitisinone) capsules, which means that it has the same efficacy, safety and side effects, and may offer patients and their families greater convenience.
NITYR is also stable3 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. It has a shelf life of 36 months (3 years).
NITYR tablets are tasteless and very small4 (baby aspirin sized) and come in 2 mg, 5 mg and 10 mg tablets. NITYR can be taken with or without food, and comes with a price advantage and a NITYR Diet and Low Protein Food Program5.
Q: Is NITYR the same as Orfadin* (nitisinone) capsules?
A: NITYR is bioequivalent1 to Orfadin*, which means that it has the same efficacy, safety and side effects, and may offer patients and their families greater convenience.
Q: Are the adverse reactions different from Orfadin* capsules?
A: Because NITYR (nitisinone) tablets are bioequivalent1 to Orfadin* capsules, NITYR has the same efficacy, safety and adverse reactions.
Q: What do you mean by ‘improved stability’?
A: Improved stability 3 refers to how long NITYR can be stored at room temperature for (up to 77°F/25°C), allowing for excursions between 59° and 86°F (15° and 30°C), until its expiry date. It has a shelf life of 3 years. Because of this improved stability you do not need to refrigerate NITYR.
Q: Will NITYR cost me more than my current nitisinone medication?
A: NITYR should not cost you more and the NITYR Diet and Low Protein Food Program5 may help with the out of pocket expenses. Our team can make a full benefit investigation to tell you straight away how much NITYR will cost. We also offer $0 Co-pay for eligible patients6.
Talk to your healthcare professional and ask them to complete a Free Trial2 free trial 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.
1 Data on file: NIT/02/17 2 Free trial offer: Subject to terms and conditions and eligibility criteria. Patients can try NITYR for three weeks for free as long as their doctor prescribes it. Subject to terms and conditions, eligibility criteria, and to other federal and state law. 3 Data on file: NIT/01/17 4Data on file: NIT/03/18 5 Diet and Low Protein Food Program: Subject to terms and conditions and eligibility criteria. This program is for eligible commercially insured patients only. Not available for government-insured patients and subject to other federal and state law. 6 $0 co-pay for eligible commercially insured patients (up to $15,000 per year per patient). Cambrooke Therapeutics is a registered trademark of Cambrooke Therapeutics, Inc.
*Orfadin is a registered trademark licensed by Sobi, Inc. or its affiliates.