Applied Therapeutics Inc. has encountered a significant hurdle in its efforts to bring a new treatment for galactosemia to market. The U.S. Food and Drug Administration (FDA) has issued a warning letter to the company, citing deficiencies in the clinical application for its drug, govorestat. This development follows the FDA's recent decision to deny approval for the drug, which was intended to treat the rare genetic metabolic disorder galactosemia.

Galactosemia is a condition that affects approximately 3,000 patients in the United States. It is characterized by the body's inability to metabolize the sugar galactose, leading to the accumulation of a toxic byproduct called galactitol. This can result in severe complications, including cognitive and motor dysfunctions. Currently, there are no approved treatments for this disease, making Applied Therapeutics' govorestat a potentially groundbreaking therapy.

The FDA's warning letter highlighted issues related to electronic data capture and a dosing error during the trial's dose-escalation phase. Applied Therapeutics has stated that these issues were addressed in previous communications with the FDA, and the company plans to respond to the letter within the permitted 15 business days.

The denial of govorestat's approval has had a significant impact on Applied Therapeutics, with the company's stock plummeting by over 80%. Despite this setback, the company remains committed to working with the FDA to resolve the identified issues and determine a path forward for govorestat. CEO Soshana Shendelman expressed disappointment over the rejection but emphasized the company's intention to address the concerns raised in the FDA's Complete Response Letter (CRL).

In addition to its efforts in galactosemia, Applied Therapeutics is also developing govorestat for another rare disease, sorbitol dehydrogenase (SORD) deficiency. The company plans to file a New Drug Application for this indication in early 2025, and analysts believe that the recent CRL will not impact this submission.

The path forward for govorestat in treating galactosemia remains uncertain, with the possibility of additional clinical trials being necessary. However, Applied Therapeutics is determined to continue its pursuit of approval, driven by the potential benefits the drug could offer to patients with this challenging condition.