FDA extends review period for govorestat to treat classic galactosemia

FDA extends review period for govorestat to treat classic galactosemia | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.

The FDA review process for govorestat (AT-007; Applied Therapeutics), an investigational novel Aldose Reductase Inhibitor being developed for the treatment of classic galactosemia, has been extended by 3 months and has a new Prescription Drug User Fee Act (PDUFA) date of November 28, 2024.

According to a press release from Applied Therapeutics, the FDA required additional time to review previously submitted data. The federal agency accepted the New Drug Application (NDA) and granted Priority Review in February 2024. Additionally, govorestat was granted Pediatric Rare Disease designation and would qualify for a Priority Review Voucher if approved.

If approved, govorestat would be the first medication indicated for the treatment of galactosemia, a rare genetic metabolic disease resulting in an inability to metabolize simple sugar galactose, according to Applied Therapeutics.

If not metabolized properly, galactose is converted to toxic metabolite, galactitol, which can result in neurological complications such as deficiencies in speech, behavior, cognition, and motor skills.

Newborn screening for galactosemia is mandatory in the United States, which sees approximately 3000 patients with galactosemia and 80 new births per year.

The NDA filing for govorestat was supported by data from the phase 3 registrational ACTION-Galactosemia Kids study (NCT04902781) in children and adolescents aged 2 to 17 years with galactosemia. The trial demonstrated "clinical benefit on activities of daily living, behavioral symptoms, cognition, fine motor skills and tremor," stated the manufacturer.

The treatment also reduced plasma galactitol levels in children and adults.

Govorestat is also being studied in the ongoing phase 3 INSPIRE trial (NCT05397665), which is evaluating the effect of govorestat compared to placebo in patients with SORD deficiency on sorbitol reduction and clinical outcomes in approximately 50 patients aged 16 to 55 in the United States and Europe.

In a 12-month interim analysis, govorestat demonstrated statistical significance on the primary endpoint of correlation of sorbitol with the clinical outcome composite and demonstrated, "sustained, statistically significant reduction in sorbitol level vs. placebo," stated Applied Therapeutics.

Reference:

Applied Therapeutics provides FDA update on PDUFA target action date for govorestat for the treatment of classic galactosemia. Applied Therapeutics. Press release. March 28, 2024. Accessed March 29, 2024.