New York: Applied Therapeutics announced on Wednesday that the U.S. Food and Drug Administration (FDA) has declined to approve its drug, govorestat, designed to treat galactosemia, a rare genetic metabolic disorder. This setback has cast a shadow over the company’s ambitions for its first commercial product, leading to an 84% drop in its stock price after hours.
In a letter to Applied Therapeutics, the FDA stated that the marketing application for govorestat could not be approved in its current form due to certain deficiencies. The company is now reviewing the feedback from the FDA and plans to request a meeting to discuss the necessary steps for resubmitting the application or potentially appealing the decision.
The FDA had previously delayed its decision on govorestat in March, in order to further review supplemental analyses submitted by the company.
Galactosemia is a rare genetic disorder that prevents the body from metabolizing galactose, a simple sugar. The inability to properly process galactose leads to its conversion into a toxic substance called galactitol, which can cause severe neurological complications. These may include impairments in speech, cognition, behavior, and motor skills. The disease affects approximately 3,000 patients in the United States, according to Applied Therapeutics.
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Applied Therapeutics based its application for govorestat on data from a late-stage study, which showed that the drug significantly reduced galactitol levels in pediatric patients. The application also included data from a mid-stage study involving adult patients.
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Meanwhile, govorestat’s marketing application is under review by the European Union’s drugs regulator.
