Denali Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to tividenofusp alfa (DNL310) for the treatment of individuals with Hunter syndrome (MPS II). This designation is a critical regulatory milestone for the company's lead program.
The Breakthrough Therapy Designation is in addition to other regulatory recognitions previously granted to tividenofusp alfa, including Fast Track, Orphan Drug, and Rare Pediatric Disease designations. These designations collectively aim to expedite the development and review process for therapies addressing serious conditions with unmet medical needs.
Denali Therapeutics expects to submit a Biologics License Application (BLA) for tividenofusp alfa in early 2025. This submission will be pursued under the accelerated approval pathway, reflecting the potential of DNL310 to offer significant clinical benefit to the Hunter syndrome community.
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