FDA Scrutiny and Data Issues Plague Applied Therapeutics' Govorestat Trials

The FDA's rejection of Govorestat, Applied Therapeutics' drug candidate for treating galactosemia, is rooted in multiple trial conduct issues that came to light after a comprehensive inspection. The FDA issued a warning letter pointing to significant problems such as the deletion of essential electronic data by a third-party vendor and mishandling of dosing errors, which led to patients initially receiving lower doses than prescribed[1]. Additionally, the FDA criticized the company for failing to adequately inform the agency about the clinical trial discrepancies, including improperly recorded doses which affected at least 19 patients[2]. Despite attempts to address these issues with prior warnings, the persistent problems led to a failed phase 3 trial in 2023, contributing to a significant drop in Applied Therapeutics' share price and the subsequent regulatory refusal[1].