Exciting News from Denali Therapeutics Inc.
Accelerated Approval Pathway for Treating MPS II
SOUTH SAN FRANCISCO, Calif., Sept. 03, 2024 (GLOBE NEWSWIRE) —
Denali Therapeutics Inc. (Nasdaq: DNLI) has recently announced a breakthrough in their meeting with the Center for Drug Evaluation and Research (CDER) division of the U.S. Food and Drug Administration (FDA). This milestone paves the way for filing a biologics license application (BLA) for accelerated approval and subsequent conversion to full approval for tividenofusp alfa (DNL310) in the treatment of MPS II (Hunter syndrome).
During the meeting, agreement was reached that cerebrospinal fluid heparan sulfate (CSF HS) can be used as a surrogate endpoint to predict clinical benefit, supporting the accelerated approval of tividenofusp alfa for MPS II. Denali will include preclinical and clinical data on biomarkers such as CSF HS and neurofilament light (NfL) in the BLA, set to be submitted under the accelerated approval pathway early in 2025.
This breakthrough opens up new possibilities for treating MPS II and marks a significant step forward in addressing this rare genetic disorder. Denali’s innovative approach and dedication to advancing treatments for rare diseases are commendable.
Impact on Individuals
For individuals with MPS II and their families, the potential approval of tividenofusp alfa under the accelerated pathway offers hope for a more effective treatment option. This could lead to improved quality of life and better disease management for those affected by MPS II.
Global Implications
On a global scale, the approval of tividenofusp alfa for MPS II could impact the way rare diseases are treated and managed. It sets a precedent for using biomarkers as surrogate endpoints in drug development and may accelerate the approval process for other rare disease treatments in the future.
Conclusion
The outcome of Denali Therapeutics Inc.’s recent meeting with the FDA is a significant milestone in the journey towards finding effective treatments for rare diseases like MPS II. The potential approval of tividenofusp alfa under the accelerated pathway represents a promising step forward in the field of rare disease therapeutics, offering hope to individuals and families affected by MPS II worldwide.
