FDA Grants Review Date For Applied Therapeutics' First Commercial Product For Rare Metabolic Disorder

Applied Therapeutics Inc APLT shares are trading higher on Wednesday after the FDA accepted the filing of the New Drug Application (NDA) for govorestat (AT-007) for Classic Galactosemia. 

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 plans 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.

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 European Medicines Agency (EMA), which was validated in December 2023 and is under review by the EMA’s Committee for Medicinal Products for Human Use. 

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. 

When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women). 

Concurrently, Applied Therapeutics announced a private placement of $100 million with a sale of 12.3 million shares and 2 million pre-funded warrants at $6.999.

The company will use the net proceeds to fund govorestat (AT-007) commercial activities, develop other pipeline candidates, and work capital for general corporate purposes. 

The capital raised in the Private Placement and current cash and potential milestones expected from its Advanz European licensing partnership are expected to fund the business into 2026.

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

Price Action: APLT shares are up 36.20% at $7.60 on the last check Wednesday.