Applied Therapeutics has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for govorestat, intended for the treatment of galactosemia. The announcement has been met with considerable disappointment and uncertainty regarding the future of the drug's development for this indication.
Unexpected Regulatory Setback
Analysts at William Blair described the FDA's decision as "unexpected and disappointing," particularly due to the lack of detailed information provided in the CRL. This absence of clarity makes it difficult to determine the specific deficiencies that need to be addressed and whether further clinical trials will be required to secure approval for govorestat in treating galactosemia.
Uncertainty Surrounding Future Steps
The primary concern revolves around the path forward for govorestat. Without specific feedback from the FDA, Applied Therapeutics faces the challenge of identifying the necessary steps to address the agency's concerns. The possibility of needing to conduct an additional clinical trial represents a significant hurdle, potentially delaying the drug's availability to patients and increasing development costs.
Analyst Perspective
Despite the setback, William Blair maintains an optimistic outlook on Applied Therapeutics, retaining an 'Outperform' rating for the company. The firm suggests that a Type-A meeting with the FDA could provide crucial clarity on the deficiencies identified in the CRL and outline a viable path forward for govorestat. This meeting could help determine whether additional data or studies are needed to satisfy the FDA's requirements.
