FDA Rejects Govorestat for Classic Galactosemia, Citing Clinical Deficiencies

FDA Rejection:
The FDA has rejected the New Drug Application (NDA) for govorestat (AT-007), an investigational treatment for classic galactosemia, citing deficiencies in the clinical application12.

Clinical Deficiencies:
The Complete Response Letter (CRL) from the FDA indicates that the application cannot be approved in its current form due to these deficiencies2.

Applied Therapeutics' Response:
Applied Therapeutics plans to meet with the FDA to address the issues and explore options for resubmission or appeal12.

Disease Background:
Classic galactosemia is a rare genetic condition that prevents proper metabolism of the sugar galactose, leading to neurological complications including cognitive and motor impairments1.

Clinical Trials:
Despite promising results from clinical trials, including significant benefits in cognitive function, behavior, motor skills, and daily living activities, the FDA found the application lacking1.

Urgent Need:
The Galactosemia Foundation has expressed disappointment and stressed the urgent need for treatment, urging the FDA to reconsider its decision1.

Future Plans:
Applied Therapeutics is also developing govorestat for other rare conditions, including sorbitol dehydrogenase deficiency, with plans to submit another application in early 20251.

Sources:

1. https://xtalks.com/govorestat-hits-roadblock-fda-denies-approval-for-applied-therapeutics-rare-disease-drug-3983/

2. https://appliedtherapeutics.gcs-web.com/news-releases/news-release-details/applied-therapeutics-receives-complete-response-letter-us-fda