FX.co ★ Applied Therapeutics Says FDA Extends Review Period Of NDA For Govorestat

On Friday, Applied Therapeutics Inc. reported that the United States Food and Drug Administration (FDA) extended the analysis period for the New Drug Application (NDA) of Govorestat, known as AT-007, by three months to November 28th. This treatment is for Classic Galactosemia.

The FDA indicated the need for more time to examine supplemental analyses of previously submitted data since this additional information constitutes a significant amendment to the previously submitted NDA.

In February 2024, Applied Therapeutics announced that the FDA had accepted and prioritized the review of the NDA.

Govorestat, an investigational, novel aldose reductase inhibitor (ARI) under development for numerous rare diseases, had previously acquired a pediatric rare disease designation. Upon approval, it will be eligible for a priority review voucher.

The NDA application for Govorestat was bolstered by a quick and sustained reduction in galactitol levels shown to benefit pediatric patients with a favorable safety profile.

The submission package was conceived with clinical outcomes data from the Phase 3 registrational ACTION-Galactosemia Kids study in children from 2 to 17 years old with galactosemia. Also included were results from the Phase 1/2 ACTION-Galactosemia study in adult patients with the same disorder, along with preclinical data.

Govorestat, if approved, would be the first medical intervention for Galactosemia and mark the debut of Applied Therapeutics' first commercial product.

Following market close on Thursday, Applied Therapeutics' shares were down by 14.71% at $5.80 on the Nasdaq.