Applied Therapeutics has shared an update on the FDA’s ongoing review of its New Drug Application (NDA) for govorestat, a potential treatment for Classic Galactosemia.
This week, the company said that the FDA has decided not to hold an advisory committee meeting, previously scheduled for October 9, and will continue with the Priority Review as planned. The Prescription Drug User Fee Act (PDUFA) target action date remains set for November 28, 2024.
The company completed its late-cycle review meeting with the FDA, which confirmed the continuation of the review process with discussions on post-marketing requirements expected in October. Govorestat, a CNS-penetrant aldose reductase inhibitor, has shown promise in clinical trials by reducing toxic metabolite levels and improving neurological symptoms in patients with Galactosemia.
Galactosemia is a rare genetic disorder in which the body cannot properly process galactose, a sugar found in milk and other foods. Due to an enzyme deficiency, galactose builds up in the body, leading to toxic effects that can cause liver damage, developmental delays, neurological issues, and other complications such as cataracts and ovarian insufficiency. The condition is typically detected through newborn screening, and treatment involves strict dietary management to avoid galactose-containing foods, though long-term complications may still arise even with intervention.
Govorestat has received several designations from both the U.S. and European regulatory bodies, including Orphan Drug and Fast Track status, positioning it as a potential first treatment option for Galactosemia.