Applied Therapeutics says it received FDA's warning letter for genetic disease drug study (updated)
02/12/2024 23:28
(Adds background on drug in paragraphs 2 and 3, details in
paragraphs 4 to 7)
Dec 2 (Reuters) - Applied Therapeutics APLT.O 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)
((Sriparna.Roy@thomsonreuters.com))