Govorestat FDA Approval Status
Last updated by Judith Stewart, BPharm on March 19, 2024.
FDA Approved: No
Generic name: govorestat
Company: Applied Therapeutics, Inc.
Treatment for: Galactosemia
Govorestat is a central nervous system penetrant Aldose Reductase Inhibitor (ARI) in development for the treatment of galactosemia.
- Galactosemia is a rare, slowly progressing metabolic disease caused by a genetic inability to break down the sugar galactose. Galactose is found in foods, but is also produced endogenously by the body. When galactose is not metabolized properly, it is converted to the toxic metabolite galactitol which causes neurological complications including deficiencies in speech, cognition, behavior, and motor skills. It can also cause juvenile cataracts and ovarian insufficiency in women.
- Govorestat works by inhibiting the enzyme aldose reductase, which converts galactose into galactitol and plays a crucial role in the pathogenesis of galactosemia.
- Govorestat is also being studied for the treatment of sorbitol dehydrogenase (SORD) deficiency and phosphomannomutase 2 deficiency (PMM2-CDG).
- Govorestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of galactosemia, PMM2-CDG, and SORD deficiency; Pediatric Rare Disease designation for galactosemia and PMM2-CDG; and Fast Track designation for galactosemia.
- The FDA has accepted the filing of the New Drug Application (NDA) for govorestat for the treatment of classic galactosemia. The NDA was granted Priority Review status, and the FDA has assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024.
Development timeline for govorestat
Further information
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