FDA Approves Nityr (nitisinone tablets) for Tyrosinemia

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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