Applied Therapeutics updates FDA on galactosemia drug

Applied Therapeutics has shared an update regarding the FDA’s ongoing review of its New Drug Application (NDA) for govorestat, a drug being investigated as a treatment for Classic Galactosemia. Recently, the company announced that the FDA has decided against holding an advisory committee meeting, which had been planned for October 9. Instead, the FDA will proceed with its Priority Review as initially scheduled. The target action date set by the Prescription Drug User Fee Act (PDUFA) remains November 28, 2024.

In a recent late-cycle review meeting, the FDA confirmed the continuation of the review process and mentioned that discussions about post-marketing requirements would occur in October. Govorestat, a Central Nervous System (CNS)-penetrant aldose reductase inhibitor, has demonstrated potential in clinical trials by decreasing toxic metabolite levels and alleviating neurological symptoms in patients with Galactosemia.

Galactosemia is a rare genetic condition where the body fails to properly process galactose, a sugar present in milk and various foods. This disorder results from an enzyme deficiency, causing galactose to accumulate in the body. The buildup can have toxic effects, leading to liver damage, developmental delays, neurological issues, and other complications such as cataracts and ovarian insufficiency. The condition is usually identified through newborn screening, and the current treatment involves strict dietary management to avoid galactose-containing foods. Despite this intervention, long-term complications may still occur.

Govorestat has garnered multiple designations from both U.S. and European regulatory agencies, including Orphan Drug and Fast Track status. These designations highlight its potential as the first treatment option specifically for patients with Galactosemia.