Accelerated ApprovalThe FDA’s CDER division has opened a path for accelerated approval for tividenofusp alfa for the treatment of MPS II, potentially launching it ~2 years earlier than traditional approval.
Clinical DataDNL310 has shown significant reductions in cerebrospinal fluid heparan sulfate levels, normalizing in 100% of participants by week 24, suggesting strong clinical efficacy.
Regulatory AlignmentDenali and the FDA have reached alignment on a registrational path that includes early 2025 BLA submission under the accelerated approval pathway for DNL310.