Latest on govorestat fda clr

The latest updates on the FDA review of Govorestat highlight both the challenges and the ongoing efforts of Applied Therapeutics to address the Complete Response Letter (CRL) and move forward with the drug's development. 1. **FDA CRL and Company Response**: - **November 26-28, 2024**: Applied Therapeutics received a Complete Response Letter (CRL) from the FDA regarding the New Drug Application (NDA) for Govorestat in Classic Galactosemia. The CRL indicated deficiencies in the clinical application, leading to the FDA's inability to approve the NDA in its current form. - **Company Response**: Applied Therapeutics is reviewing the FDA's feedback and plans to immediately request a meeting to discuss the requirements for either a resubmission of the NDA or an appeal of the decision, along with appropriate next steps. 2. **Previous Expectations and Setbacks**: - **September 2024**: Prior to the CRL, expectations for Govorestat's approval were high. The FDA had signaled a positive direction by canceling an advisory committee meeting scheduled for October 9, which was interpreted as a favorable sign for an approval decision by the November 28 PDUFA target date. - **November 27, 2024**: The unexpected CRL led to a significant drop in Applied Therapeutics' stock price, reflecting investor disappointment and the impact of the FDA's decision. 3. **Future Steps and Pipeline Implications**: - **SORD Deficiency**: Despite the setback with Govorestat, Applied Therapeutics is moving forward with preparations to submit an NDA for Govorestat in the treatment of Sorbitol Dehydrogenase (SORD) Deficiency in early 2025. - **Pipeline Progress**: The company's ongoing participation in healthcare conferences and its focus on advancing its pipeline indicate a strategic effort to maintain momentum and explore new opportunities. In summary, Applied Therapeutics is currently addressing the FDA's concerns regarding Govorestat and preparing for potential resubmissions or appeals. The company's broader pipeline progress and participation in industry events suggest a proactive approach to navigating regulatory challenges and moving forward with its mission to develop transformative treatments for rare diseases.