Boost for Applied Therapeutics as FDA accepts govorestat NDA for priority review

USA-based rare disease Applied Therapeutics (Nasdaq: APLT) today announced that the US Food and Drug Administration (FDA) has accepted the filing of the New Drug Application (NDA) for govorestat (AT-007) for the treatment of classic galactosemia, with the news sending the firm’s shares up 46% to $8.16 by mid-morning trading.

The NDA was granted Priority Review status, and the FDA assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024. The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application. Govorestat was previously granted Pediatric Rare Disease designation, and will qualify for a Priority Review Voucher (PRV) upon approval.

The company noted that, if approved, govorestat would be the first medication indicated for the treatment of galactosemia and would be Applied Therapeutics’ first commercial product.