Applied Therapeutics, Inc. has announced an extension in the review period by the U.S. Food and Drug Administration for their New Drug Application for govorestat, intended for treating Classic Galactosemia. The FDA has set a new target action date under the Prescription Drug User Fee Act (PDUFA) to November 28, 2024, extending the review period by three months.

This extension was prompted by the FDA's requirement for additional time to assess supplemental analyses of previously submitted data, provided by Applied Therapeutics in response to routine information requests. The FDA deemed this additional information a Major Amendment to the NDA. Notably, in February 2024, the FDA had accepted and granted Priority Review to the NDA. Govorestat, previously granted Pediatric Rare Disease designation, is expected to qualify for a Priority Review Voucher (PRV) upon approval.

Applied Therapeutics' Founder and CEO, Dr. Shoshana Shendelman, expressed confidence in govorestat's potential approval for Galactosemia despite the delay. She emphasized the company's commitment to collaborating closely with the FDA throughout the review process. Furthermore, the company remains dedicated to maintaining the expanded access program for govorestat, ensuring that patients with Galactosemia have access to this vital treatment option during the extended review period.

Govorestat, an investigational Aldose Reductase Inhibitor (ARI), has shown promise in addressing several rare diseases. The NDA filing is backed by compelling data demonstrating rapid and sustained reduction in galactitol, leading to significant clinical benefits across pediatric patients, coupled with a favorable safety profile. The submission package includes data from the Phase 3 registrational ACTION-Galactosemia Kids study, the Phase 1/2 ACTION-Galactosemia study, and preclinical data.

If approved, govorestat would mark the first medication indicated for treating Galactosemia and would serve as Applied Therapeutics' inaugural commercial product. Additionally, the company has submitted a Marketing Authorization Application (MAA) for govorestat to the European Medicines Agency (EMA), with an expected decision in the fourth quarter of 2024.

Galactosemia is a rare genetic metabolic disorder resulting in the body's inability to metabolize galactose, leading to various neurological complications and other health issues. The disease affects a small population, with approximately 3,000 patients in the US and 80 new births annually, along with 4,000 patients in the EU and 120 new births yearly.

Govorestat (AT-007) is a promising candidate for treating Galactosemia and other rare neurological diseases, including SORD Deficiency and PMM2-CDG. Clinical studies have demonstrated its efficacy in improving clinical outcomes and reducing toxic metabolite levels associated with these conditions. Moreover, govorestat has received several regulatory designations, highlighting its potential to address significant unmet medical needs.

 

Source: globenewswire.com

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