What is the story about?
What's Happening?
Applied Therapeutics has posted a new study for its aldose reductase inhibitor govorestat, indicating potential alignment with the FDA on a path forward for the drug in treating sorbitol dehydrogenase deficiency. The study aims to enroll 155 patients and will assess the long-term effects of govorestat on functional and clinical outcomes. This development follows the drug's failure in a previous trial, where it did not significantly improve primary outcomes but showed positive secondary results.
Why It's Important?
The potential alignment with the FDA suggests that govorestat could still receive accelerated approval based on its ability to lower blood sorbitol levels and improve patient outcomes. This is significant for patients with sorbitol dehydrogenase deficiency, a rare genetic condition that leads to sensory loss and muscle weakness. If successful, govorestat could provide a new treatment option for this underserved patient population, highlighting the importance of regulatory flexibility in drug development for rare diseases.
What's Next?
The confirmatory study is set to begin enrollment, with a primary completion date of October 2028. Applied Therapeutics will continue to work with the FDA to finalize the drug's development pathway, aiming for a 2025 filing. The company's financial situation, with limited cash reserves, poses a potential risk, but successful alignment with the FDA could attract additional investment and support the drug's advancement.
AI Generated Content
Do you find this article useful?
