Nityr Approved for Hereditary Tyrosinemia Type 1
August 2, 2017 – The U.S. FDA has approved Nityr™ (nitisinone) tablets, manufactured by Cycle Pharmaceuticals, for the treatment of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. Hereditary tyrosinemia (HT) is a genetic condition that leads to harmful accumulation of tyrosine, an amino acid, in the bloodstream. This is due to a deficiency in the enzyme responsible for metabolizing tyrosine from proteins. Left untreated, HT causes diarrhea, vomiting, pain, rickets, irritability, and fatigue, as well as eventually fatal damage to organs, including the eye, liver, kidney, and nerves. HT-1, the most severe of three identified types, is typically diagnosed within three months of birth. Children whose HT-1 goes untreated have a life expectancy of less than 10 years. Approximately 150 patients in the United States have been diagnosed with HT-1.
Recommended dosing for Nityr starts at 0.5mg/kg twice a day, and is not to exceed 2mg/kg per day. The tablets can be given by oral syringe after being dissolved in water, or they can be crushed and mixed with a small amount of applesauce.
Nityr is a bioequivalent Orfadin® (nitisinone) capsules, which are manufactured by Sobi. Although Orfadin has been available in the U.S. to treat HT-1 since 2002, the capsules are larger than Nityr tablets and must be refrigerated, while Nityr can be stored at room temperature. Plans for the pricing and launch of Nityr have not yet been disclosed.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by U.S. mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- U.S. Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
Actual drug patent expiration dates and availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.
*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.