Applied Therapeutics' rare disease drug receives CRL

The FDA has rejected Applied Therapeutics' application for their experimental treatment, govorestat, aimed at addressing classic galactosemia, a rare genetic disorder. This condition hampers the body's ability to metabolize galactose, a sugar predominantly found in dairy products. Infants diagnosed with the disease often exhibit symptoms such as feeding difficulties, vomiting, and jaundice.

Despite the setback, Applied Therapeutics expressed disappointment over the FDA’s decision but remains determined to pursue further discussions with the regulatory body. The company aims to explore potential pathways for resubmission or an appeal regarding the rejection. Following the announcement of the FDA's complete response letter (CRL), Applied's stock ($APLT) plummeted by approximately 80% on Wednesday.

Govorestat functions by inhibiting aldose reductase, an enzyme involved in the galactose metabolism pathway. However, the drug had previously failed to achieve its primary endpoint in clinical trials, which assessed a range of clinical measures associated with the disease. In their application for FDA approval, Applied Therapeutics highlighted secondary and post-hoc analyses focusing on tremors and behavioral aspects.

Initially, the FDA had planned to convene an advisory committee to review govorestat’s application for treating galactosemia. However, the timeline for this meeting, initially set for August, was postponed indefinitely by the agency. Then, in September, Applied Therapeutics revealed that the FDA had decided against holding a meeting with external experts to discuss the treatment. On that particular day, Applied's stock saw a significant increase, closing 69% higher at $7.85 per share.

Looking ahead, Applied Therapeutics has announced plans to seek FDA approval for govorestat in early next year for another rare disorder, SORD deficiency. Despite the challenge posed by the recent rejection, the company remains committed to advancing its pipeline and bringing new treatments to market for rare genetic diseases.