First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage disease.
The New England Journal of Medicine Publishes Phase 1/2 Study of Denali Therapeutics’ Tividenofusp Alfa (DNL310) for Hunter Syndrome (MPS II)
FDA assigns PDUFA target action date of January 5, 2026, for decision on accelerated approval.
Denali Therapeutics Announces Primary Analysis and Long-Term Follow-Up of Phase 1/2 Study in Hunter Syndrome (MPS II) with Tividenofusp Alfa
Denali expects to submit a Biologics License Application for tividenofusp alfa in early 2025 for regulatory review under the accelerated approval pathway
START is a pilot program newly launched jointly by the FDA CDER and CBER divisions to further accelerate the development of novel drug and biological products for rare diseases