After recent successful meetings with the FDA, Denali Therapeutics announced its plan to file for accelerated approval of DNL310 for the treatment of MPSII, also known as Hunter Syndrome.
Carole Ho, MD, Chief Medical Officer of Denali, expressed gratitude towards the FDA's Center for Drug Evaluation and Research (CDER) for their positive and collaborative discussion. She highlighted the significance of their guidance on CSF HS as a surrogate biomarker, which is seen as a crucial step towards accelerating the development of medicines for individuals and families affected by MPS II.
This milestone is a testament to the collective effort across the patient community, academia, and industry to communicate the science and advocate for faster paths to effective treatments for these devastating rare diseases. Denali Therapeutics is excited about the potential to deliver a new MPS treatment sooner using the accelerated approval pathway. The company also looks forward to plans for conversion to full approval following the completion of the global Phase 2/3 COMPASS study.
Denali Therapeutics plans to submit a biologics license application (BLA) early in 2025 under the accelerated approval pathway, marking a significant step forward in the treatment of MPS II.