FDA Rejection of Applied Therapeutics' Galactosemia Drug Causes Stock Crash, Future Plans Unveiled
The FDA has rejected Applied Therapeutics' application for the approval of govorestat, an aldose reductase inhibitor intended to treat the rare genetic disorder of galactosemia, citing deficiencies in the clinical application[1][2]. This rejection follows the drug's failure to meet its primary efficacy endpoint in the Phase III ACTION-Galactosemia Kids study, despite showing some post-hoc benefits[1][3]. Following the FDA's decision, Applied Therapeutics' stock experienced a steep decline of over 80%[1][2]. Despite the setback, the company plans to work with the FDA to address these concerns and explore potential resubmission or appeal of the application[1][3].
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What specific deficiencies did the FDA cite in the clinical application of govorestat that led to its rejection?
How does Applied Therapeutics plan to demonstrate govorestat's efficacy and safety in future FDA submissions?
What are the potential implications for Applied Therapeutics' financial health following the significant decline in their stock after the FDA's decision?
How might the company's future plans to pursue regulatory approvals for SORD deficiency impact their strategy and portfolio?
What is the likelihood of govorestat eventually gaining FDA approval for galactosemia based on the current post-hoc benefits observed?