FDA Extends Review Period for Applied Therapeutics’ Govorestat by Three Months

FDA Entrance_iStock, Grandbrothers

Pictured: Facade of the FDA's office in Maryland/iStock, Grandbrothers

Applied Therapeutics on Thursday announced that the FDA is extending the review period for its investigational aldose reductase inhibitor govorestat for the treatment of classic galactosemia. The biotech now expects a verdict by Nov. 28, 2024.

The FDA will need more time to review supplemental analyses and data that Applied Therapeutics provided in response to the regulator’s routine information requests, which the FDA considered a major amendment to govorestat’s New Drug Application (NDA), according to the company.

Shoshana Shendelman, founder and CEO of Applied Therapeutics, said in a statement that despite the delay the company remains “confident in the potential for govorestat approval for galactosemia and we will continue to work closely with the FDA throughout the review process.”

Classic galactosemia is a rare and genetic metabolic disease characterized by the inability to properly metabolize galactose. Patients with galactosemia have a surplus of the toxic metabolite galactitol, which accumulates in tissues and organs, leading to neurological problems, such as speech, cognitive, behavior and motor deficiencies.

In galactosemia, the aldose reductase enzyme is responsible for converting galactose into galactitol, according to Applied Therapeutics’ website. The biotech’s potential solution to this pathway is govorestat, a novel and selective aldose reductase inhibitor that has been designed to be able to cross the blood-brain barrier and penetrate into the central nervous system.

Applied Therapeutics is supporting govorestat’s NDA—which the FDA accepted and granted Priority Review in February 2024—with data from the Phase I/II ACTION-Galactosemia and Phase III ACTION-Galactosemia Kids trials, alongside preclinical studies.

ACTION-Galactosemia demonstrated that govorestat was safe to use in adult patients and elicited a reduction in galactitol levels. Govorestat’s action was rapid and its effects were apparent starting from the first day of treatment and was sustained over one month, according to the results. Govorestat also did not induce an increase in concentrations of galactose and other galactose metabolites.

In ACTION-Galactosemia Kids, which enrolled more than 47 pediatric patients, govorestat treatment led to clinical benefits in terms of activities of daily living, behavioral symptoms, cognition and tremors.

The original target action date for govorestat was Aug. 28, 2024, and the FDA has indicated that it plans to convene an advisory committee to discuss Applied Therapeutics’ data package.

If approved, govorestat would become the first authorized therapy for galactosemia—and it would be Applied Therapeutics’ first commercial product.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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