FDA Rejects Applied Therapeutics' Govorestat for Galactosemia, Causing Stock Collapse, Plans for SORD Deficiency Approval Continue

The FDA has denied approval of Applied Therapeutics' govorestat, a treatment designed for classic galactosemia, citing deficiencies in the clinical application[1][2]. This decision came after the drug failed to meet its primary efficacy endpoint in a Phase III trial, despite showing some post-hoc benefits[1][3]. As a result of the rejection, the company's stock dropped significantly, alongside plans to meet with the FDA to discuss the potential for resubmission or appeal[1][2]. Despite this setback, Applied remains committed to continuing the development of govorestat for another rare condition, SORD deficiency, with regulatory filings anticipated in early 2025[1][2].