FDA Requests Clinical Outcomes Data for Galactosemia Treatment

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Galactosemia is a rare genetic metabolic disease caused by an inability to metabolize galactose.

Following discussions with the Food and Drug Administration (FDA), Applied Therapeutics announced that it would hold on submitting the New Drug Application for AT-007 for the treatment of galactosemia until more data could be provided. 

The Company had hoped to submit the application for accelerated approval based on a surrogate endpoint (reduction in galactitol); however, the agency has now indicated that clinical outcomes data would be needed for approval.

Galactosemia is a rare genetic metabolic disease caused by an inability to metabolize galactose. AT-007 is an investigational compound that inhibits the enzyme aldose reductase. Inhibition of aldose reductase leads to a reduction in the formation of galactitol, a toxic metabolite that accumulates in tissues and organs. In a phase 2 registrational trial (ClinicalTrials.gov Identifier: NCT04117711), AT-007 was found to significantly reduce plasma galactitol levels vs placebo in adults with galactosemia.

The Company is currently evaluating the clinical benefit and safety of AT-007 in pediatric patients 2 to 17 years of age with classic galactosemia in the phase 3 ACTION-Galactosemia Kids study (ClinicalTrials.gov Identifier: NCT04902781). Initial study results are expected by the first quarter of 2022.

“While disappointed by this change in direction by the FDA, we remain committed to bringing this important treatment to patients with galactosemia,” said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. “We will continue to work with the FDA to determine the most expeditious path forward to regulatory approval and will provide an update on timing and plans accordingly.”


Applied Therapeutics provides regulatory update on galactosemia program. News release. Applied Therapeutics, Inc. January 3, 2022. Accessed January 5, 2022. https://ir.appliedtherapeutics.com/news-releases/news-release-details/applied-therapeutics-provides-regulatory-update-galactosemia