US FDA extends review of Applied Therapeutics' genetic disease drug

FILE PHOTO: FILE PHOTO: Signage is seen outside of FDA headquarters in White Oak, Maryland

(Reuters) -The U.S. Food and Drug Administration has extended its review of Applied Therapeutics' experimental drug to treat galactosemia, a rare genetic metabolic disease, the company said on Thursday.

The health regulator will now give its decision by Nov. 28, 2024, compared with its previous action date of Aug. 28.

Galactosemia results in an inability to metabolize the simple sugar galactose, causing it to build up to toxic levels in the blood.

There are currently no approved treatments for the disease in the United States, which has about 3,000 Galactosemia patients.

The FDA delayed its decision on the drug, govorestat, saying it requires additional time to review supplemental analyses submitted by the company.

Applied's application for govorestat is supported by data from a late-stage study in which it helped significantly reduce a toxic substance called galactitol in pediatric patients, and a mid-stage study in adult patients, the company said.

The company said it will work with the regulator throughout the review process.

(Reporting by Mariam Sunny in Bengaluru; Editing by Krishna Chandra Eluri)