Washington: Applied Therapeutics announced on Monday that it received a warning letter from the U.S. Food and Drug Administration (FDA) regarding a clinical trial for its genetic disease drug, govorestat. The letter follows the FDA’s decision last week to decline approval of the treatment in its current form due to identified deficiencies.
Govorestat was being evaluated for the treatment of galactosemia, a rare genetic metabolic disorder. The disease hinders the body’s ability to metabolize galactose, a simple sugar, leading to the formation of a toxic compound called galactitol.
In the letter, the FDA raised concerns about the trial’s electronic data capture system. Applied Therapeutics noted that these issues had been previously addressed in communications with the agency, including providing detailed paper and video records.
Also Read | US Salmonella Outbreak Linked to Cucumbers: FDA and CDC Investigate
Additionally, the letter referenced a dosing error during the trial’s dose-escalation phase, resulting in slightly lower levels of the drug than targeted in a small group of patients. The company clarified that this issue had been resolved before the trial transitioned to its maintenance dosing phase.
Also Read | U.S. Greenlights $1.17 Billion Helicopter Equipment Sale to India
Applied Therapeutics emphasized that comprehensive records had been maintained and provided to the FDA throughout the process. The company plans to respond within the stipulated 15-business-day period to address the agency’s concerns.
