Denali Therapeutics Inc. announced that the Center for Drug Evaluation and Research (CDER) of the U.S. Food and Drug Administration (FDA) has received the company’s initiation of a rolling submission of a Biologics License Application (BLA) for accelerated approval of tividenofusp alfa for the treatment of Hunter syndrome (MPS II). This marks a critical step towards potential market authorization for the company's lead therapeutic candidate.
Denali has achieved alignment with CDER on the content of the BLA data package, including the use of cerebrospinal fluid heparan sulfate (CSF HS) as a surrogate endpoint to support an accelerated approval. This agreement also covers the full approval conversion path, providing clarity on the regulatory trajectory for tividenofusp alfa.
The company expects to complete the BLA submission in the first half of May 2025 and continues to prepare for a potential commercial launch in the U.S. in late 2025 or early 2026. This accelerated pathway underscores the urgent need for new treatment options for Hunter syndrome patients.
Furthermore, Denali reported positive and ongoing collaboration with CDER through the Support for clinical Trials Advancing Rare disease Therapeutics (START) program for DNL126. This collaboration aims to align on an accelerated development and approval path for DNL126, which is being developed for the potential treatment of Sanfilippo syndrome Type A (MPS IIIA).
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