Buy Nityr (nitisinone tablets)

What is Nityr (nitisinone) for?

Nityr (nitisinone) is a hydroxyphenyl-pyruvate dioxygenase inhibitor used to treat Hereditary Tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.^[1]

Nityr (nitisinone) is bioequivalent to Orfadin (nitisinone)* and contains the same nitisinone. This means that it has the same efficacy, safety and side effects.^[9]

It is available in tablet form containing 2 mg, 5 mg or 10 mg of nitisinone.^[1]

*Orfadin is a registered trademark, licensed by Swedish Orphan Biovitrum AB (publ) and is marketed by Sobi, Inc.

How does Nityr (nitisinone) work?

Hereditary Tyrosinemia type 1 (HT-1) is a genetic disease in which patients have elevated blood levels of the amino acid tyrosine, a building block of most proteins. Patients with HT-1 are missing an enzyme that is needed by the body to break down tyrosine. Instead, it’s converted into harmful substances.^[2,3]

The active ingredient in Nityr, nitisinone, blocks an enzyme that converts tyrosine into harmful substances. Because tyrosine is not broken down, tyrosine levels are higher than normal. To compensate for this, patients need to eat a special diet low in tyrosine. The patient also needs to reduce the consumption of phenylalanine, which is an amino acid that is converted to tyrosine in the body.^[3]

Where has Nityr (nitisinone) been approved?

Nityr (nitisinone) was approved for the treatment of people with HT-1 by:

• The Food and Drug Administration (FDA), USA on August 1, 2017.^[4]
• The European Medicines Agency (EMA) on July 26, 2018.^[3]
• The Chilean Institute of Public Health (Ministry of Health, MINSAL) on November 22, 2018.^[5]
• Saudi Food and Drug Authority (SFDA)on May 13, 2020.^[6]
• The State of Israel’s Ministry of Health on October 19, 2020.^[7]
• MedSafe, New Zealand on January 19, 2021.^[8]

This medicine is also being prescribed in many regions around the world such as Latin America, Middle East, North Africa, South Africa, and South-East Asia.^[8]

Please note that this medicine may have also been approved in other regions than the ones we’ve listed. If you have a question about its approval in a specific country feel free to contact our support team.

How is Nityr (nitisinone) taken?

The maximum total daily dosage is 2 mg/kg orally. The dose should be built up stepwise (titrated) based on the patient’s response, as described in the full prescribing information. The maximum dosage is 1 mg/kg orally twice daily.^[1]

Nityr (nitisinone) is used together with a diet that restricts the intake of tyrosine and phenylalanine. It can be taken with or without food.^[1]

For patients, including children, who have difficulty swallowing whole tablets, the tablets can be dissolved in water and given using an oral syringe. If patients are able to swallow semi-solid foods, the tablets can be crushed and mixed with applesauce.^[1]

Complete information about Nityr (nitisinone) dosage and (modified) administration can be found in the official prescribing information listed in our references section.^[1]

Note: Please consult with your treating doctor for personalised dosing.

Are there any known adverse reactions or side effects of Nityr (nitisinone)?

Common adverse reactions

The most common side effects (≥1% of patients) listed in the prescribing information include:^[1]

• Elevated tyrosine levels
• Thrombocytopenia
• Leukopenia
• Conjunctivitis
• Corneal opacity
• Keratitis
• Photophobia
• Eye pain
• Blepharitis
• Cataracts
• Granulocytopenia
• Epistaxis
• Pruritus
• Exfoliative dermatitis
• Dry skin
• Maculopapular rash
• Alopecia

Serious adverse reactions

The serious adverse reactions listed in the prescribing information include:^[1]

• Thrombocytopenia
• Leukopenia
• Liver disorder (porphyria)
• Ocular/visual complaints associated with elevated tyrosine levels

Use in a specific population

It is not known what the effects of Nityr (nitisinone) are on an unborn fetus. It is advised to avoid pregnancy and breastfeeding during treatment.^[1]

For a comprehensive list of side effects and adverse reactions please refer to the official prescribing information.^[1]

References

1. Full prescribing information: Nityr (nitisinone) [PDF] Cycle Pharmaceuticals, Aug 2017

2. Nityr product information
EMA, July 2018

3. Tyrosinemia type 1
Rarediseases.info.nih.gov, cited Jul 2021

4. Cycle Pharmaceuticals Receives FDA Approval of Nityr™ (nitisinone) Tablets for Treatment of HT-1
Cycle Pharma, Aug 2017

5. Cycle Pharmaceuticals’ NITYR (nitisinone) tablets receives marketing authorization in Chile
Cycle Pharma, Nov 2018

6. Cycle Pharmaceuticals’ NITYR® (nitisinone) Tablets Receives Marketing Authorization in the Kingdom of Saudi Arabia
Cycle Pharma, May 2020

7. Cycle Pharmaceutical’s Nityr (nitisinone) Tablets Marketing Authorization in Israel
Cycle Pharma, Oct 2020

8. NITYR (nitisinone) Tablets for Tyrosinaemia Type 1 receives Marketing Authorization in New Zealand
Cycle Pharma, Mar 2021

9. Clinical Review Report: Nitisinone (Nitisinone Tablets) – Appendix 5 Bioequivalence Study
Cycle Pharmaceuticals, Canadian Agency for Drugs and Technologies in Health; Aug 2018.