(Reuters) -Fortress Biotech on Wednesday said the U.S. Food and Drug Administration declined to approve the drug it is developing with Indian drugmaker Zydus Lifesciences for a rare genetic disease affecting copper absorption in children. Shares of Florida-based Fortress dropped 34% to $2.43 in morning trade. The FDA in its “complete response letter” pointed to […]
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US FDA declines to approve Fortress Bio and Zydus’ treatment for a rare pediatric disease
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(Reuters) -Fortress Biotech on Wednesday said the U.S. Food and Drug Administration declined to approve the drug it is developing with Indian drugmaker Zydus Lifesciences for a rare genetic disease affecting copper absorption in children.
Shares of Florida-based Fortress dropped 34% to $2.43 in morning trade.
The FDA in its “complete response letter” pointed to deficiencies at the facility where the drug, CUTX-101, is manufactured, the company said, adding that the regulator did not cite issues with efficacy and safety data.
The drug is being developed to treat Menkes disease, where patients are born without the ability to absorb copper from their diet. It is caused by mutations in a gene responsible for the transportation of copper in the body.
The disease primarily affects male infants. According to the companies, recent estimates suggest a prevalence of 1 in 34,810 to as high as 1 in 8,664 live male births.
Sentynl Therapeutics, a company wholly owned by Zydus, had taken over development and commercialization of CUTX-101 in December 2023. Sentynl plans to meet with the FDA and resubmit the application after addressing the issues, Fortress added.
The experimental drug had been granted priority review and is backed by studies that showed improved overall survival with early treatment initiation, according to Fortress.
(Reporting by Padmanabhan Ananthan, Sneha S K and Christy Santhosh in Bengaluru; Editing by Vijay Kishore)