(Reuters) -Applied Therapeutics said on Wednesday that the U.S. FDA declined to approve its drug to treat galactosemia, a rare genetic metabolic disease, casting a cloud over the company’s hopes for its first commercial product.
The company’s shares fell 84% after the bell.
The Food and Drug Administration’s letter to the company said the marketing application for the drug, govorestat, cannot be approved in its current form, due to certain deficiencies.
The drug developer is reviewing the feedback from the FDA and plans to immediately request a meeting to discuss requirements for a potential resubmission of the application or appeal of the decision.
The health regulator previously delayed its decision on the drug in March to review supplemental analyses submitted by the company.
Galactosemia is a rare, genetic metabolic disease resulting in an inability to metabolize a simple sugar, galactose, which would then get converted to a toxic substance called galactitol.
It can cause neurological complications, including deficiencies in speech, cognition, behavior and motor skills.
There are about 3,000 galactosemia patients in the United States, according to the company.
Applied Therapeutics’ application for govorestat was based on data from a late-stage study in which the drug helped significantly reduce galactitol levels in pediatric patients, and a mid-stage study in adult patients, the company said.
The company’s marketing application for govorestat is also under review by the European Union’s drugs regulator.
(Reporting by Sneha S K, Mariam Sunny and Christy Santhosh in Bengaluru; Editing by Shailesh Kuber)
