By Dhirendra Tripathi
Investing.com – Applied Therapeutics stock (NASDAQ:APLT) sank 25% in Monday’s pre-market trading after receiving bad news from the FDA for the company’s lead drug candidate.
The company was earlier expecting an accelerated approval for the drug AT-007, which aims to treat galactosemia, a rare genetic metabolic disease, from the Food and Drug Administration, but the regulator has now indicated that clinical outcomes data will likely be required for it, according to a company release. The company has thus decided to hold on submitting a new drug application for AT-007 for treatment of galactosemia pending additional discussions with the FDA.
The first assessment of the clinical outcome will be completed in the first quarter of 2022, and then every 6 months thereafter until the study reaches statistical significance, the release said.
The company is developing AT-007 for treatment of galactosemia, a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior and motor skills.
AT-007 is currently being studied in a phase-3 clinical outcomes trial (ACTION-Galactosemia Kids) in 2-17-year-olds suffering from galactosemia. There are approximately 3,000 patients with galactosemia in the US and 80 new births per year.