US FDA extends review of Applied Therapeutics' genetic disease drug (updated)

Applied Therapeutics

28/03/2024 21:12

(Adds details throughout)
       March 28 (Reuters) - The U.S. Food and Drug
Administration has extended its review of Applied Therapeutics'
 APLT.O  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)
 ((Mariam.ESunny@thomsonreuters.com;))