Rare Disease Report

FDA Approves Nityr (nitisinone tablets) for Tyrosinemia

JULY 31, 2017
James Radke
The U.S. Food and Drug Administration (FDA) has approved Nityr (nitisinone tablets) for the hereditary tyrosinemia type 1 (HT-1), a rare metabolic disorder. The drug is to be taken in combination with dietary restriction of tyrosine and phenylalanine.
HT- I is due to a deficiency of the enzyme fumarylacetoacetate hydrolase (FAH). The absence of FAH leads to an accumulation of toxic metabolic products as the body is unable to properly breakdown tyrosine.
Nitisinone inhibits 4-hydroxyphenylpyruvate dioxygenase so that toxic by-products of tyrosine do not accumulate in the body.

Nityr is the second drug approved for HT-1 patients. Another formulation of nitisinone, Orfadin, had been available for HT-1 for several years and comes in capsule and oral suspension form. The approval of Nityr provides additional treatment options for people with HT-1. Unlike Orfadin, Nityr does not require refrigeration and can be taken with or without food.
“The FDA approval of Nityr for HT-1, is an exciting new treatment option for patients in the United States,” said Jon Miller, President of the Network of Tyrosinemia Advocates (NOTA) in a news release. “Since this new treatment is a small tablet and does not need refrigeration, it may offer convenience and a better quality of life to patients and families who struggle with the debilitating effects of HT-1. Cycle Pharmaceuticals has taken an innovative approach to help address unmet needs and I hope this will be an incentive for pharmaceutical companies to further invest and innovate in HT-1 treatments and support.”
"Cycle Pharmaceuticals has interpreted the modern health needs of HT-1 patients and caregivers, by developing a treatment that may improve their quality of life. Nityr is the first milestone in the long-term engagement of Cycle Pharmaceuticals with the HT-1 community. We look forward to working with physicians, caregivers and the patient community to help HT-1 patients benefit from the developments that Nityr brings," said Antonio Benedetti, CEO, Cycle Pharmaceuticals.
Nitry tablets are available 2 mg, 5 mg and 10 mg tablets that can be dissolved in water for younger patients, if necessary.
Approximately 150 in the U.S. have HT-1. Below, Jon Miller describes the disease:

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