FDA Presses Delay Button For Applied Therapeutics' First Potential Marketed Drug For Rare Disease

Thursday, the FDA extended the review period for Applied Therapeutics Inc’s (NASDAQ:APLT) New Drug Application (NDA) for govorestat (AT-007) for the treatment of Classic Galactosemia by three months. 

The FDA has set a new Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024, from the initial date of August 28, 2024.

The FDA notified Applied Therapeutics that it required additional time to review supplemental analyses of previously submitted data provided by Applied in response to the FDA’s routine information requests and determined that the additional information constitutes a Major Amendment to the NDA. 

In February 2024, the company announced that the FDA accepted and granted Priority Review to the NDA. Govorestat was previously granted Pediatric Rare Disease designation and will qualify for a Priority Review Voucher (PRV) upon approval.

If approved, govorestat would be the first medication indicated for the treatment of Galactosemia and would be Applied Therapeutics’ first commercial product. 

The company has also submitted a Marketing Authorization Application (MAA) for govorestat for Classic Galactosemia to the EMA, which was validated in December 2023 and is under review by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The company expects a decision by the EMA in the fourth quarter of 2024.

Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. 

There are approximately 3,000 patients with Galactosemia in the U.S. and 80 new births per year, and approximately 4,000 patients with Galactosemia in the EU and 120 new births per year. 

With approximately $153.5 million cash as of March 1, 2024, the company expects a cash runway into the first half of 2026.

In February, Applied Therapeutics released interim 12-month results from the ongoing Phase 3 INSPIRE trial, in which the primary and several key secondary endpoints were achieved. The INSPIRE Phase 3 trial evaluates the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency.

Price Action: APLT shares are down 6.99% at $6.325 on the last check Monday.