(Reuters) – Applied Therapeutics said on Monday it received a warning letter limited to a trial studying its genetic disease drug, after the U.S. health regulator declined to approve the treatment last week.
On Wednesday, the U.S. Food and Drug Administration said the marketing application for the drug, govorestat, cannot be approved in its current form due to certain deficiencies.
The company was seeking approval of the drug to treat galactosemia — a rare, genetic metabolic disease that causes inability to metabolize a simple sugar, galactose, which would instead get converted to a toxic substance called galactitol.
The letter identified issues related to electronic data capture, which Applied Therapeutics believes had been addressed in previous communications with the agency, including detailed paper and video records, it said in a filing.
The FDA letter also refers to a dosing error in the dose-escalation phase of the trial, resulting in slightly lower levels than targeted in a limited number of patients, which was remedied prior to achieving maintenance dosing.
The drug developer said detailed records were maintained and information was provided to the FDA.
The company intends to respond within the permitted 15 business days to address these issues, it added.
(Reporting by Sriparna Roy in Bengaluru; Editing by Alan Barona)
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