Applied Therapeutics’ Rare Disease Drug Fails Another Phase 3 Trial After FDA Rejection

Applied Therapeutics' rare disease drug govorestat, targeted for classic galactosemia, has suffered another phase 3 failure after the FDA rejected the drug in late 2024 due to 'deficiencies in the clinical application.'24

The pivotal phase 3 trial missed its primary endpoint in 2023, which contributed to the FDA's decision to deny approval.45

Despite some perceived clinical benefits on various measures, the FDA concluded that the drug's data package did not meet its standards for rare disease approval; this triggered a steep decline in Applied's stock price by over 75%.45

Post-rejection, the FDA also issued a warning letter to Applied Therapeutics, highlighting problems in trial conduct, including the deletion of electronic data by a third-party vendor and mishandling of a dosing error affecting some patients.5

Applied Therapeutics has stated intentions to meet with the FDA to discuss requirements for potential resubmission or an appeal, but the clinical and procedural deficiencies create significant uncertainty about the path forward.245

Sources:

2. https://www.biospace.com/fda/applied-therapeutics-crashes-after-fda-rejection-of-rare-disease-drug

4. https://www.fiercebiotech.com/biotech/fda-rejects-applied-filing-rare-disease-approval-clinical-grounds-sinking-stock

5. https://www.fiercebiotech.com/biotech/applied-therapeutics-trial-conduct-questioned-fda-warning-letter-days-after-rare-disease