Denali(US:DNLI) Newsfilterยท2025-04-02 12:00Core Viewpoint - Denali Therapeutics has initiated a rolling submission of a biologics license application (BLA) for accelerated approval of tividenofusp alfa for Hunter syndrome, with expectations to complete the submission by May 2025 and a potential commercial launch in late 2025 or early 2026 [1][7]. Company Developments - The company is engaged in regular collaboration with the FDA's Center for Drug Evaluation and Research (CDER) regarding the BLA data package, including the use of cerebrospinal fluid heparan sulfate as a surrogate endpoint for accelerated approval [1][2]. - Denali's Chief Medical Officer expressed gratitude for the FDA's support and highlighted the importance of this regulatory milestone for the Hunter syndrome community [2]. - The company is also preparing for the development and approval path for DNL126, a treatment for Sanfilippo syndrome, under the START program with CDER [2][7]. Product Information - Tividenofusp alfa (DNL310) is designed to deliver the iduronate 2-sulfatase enzyme to the brain and body, addressing cognitive, behavioral, and physical symptoms of Hunter syndrome [4]. - The product has received Fast Track and Breakthrough Therapy designations from the FDA, as well as Priority Medicines designation from the European Medicines Agency [4]. - The ongoing Phase 2/3 COMPASS study is enrolling participants to support global approval, comparing tividenofusp alfa with idursulfase [5]. Disease Context - Hunter syndrome (MPS II) is a rare genetic disorder affecting over 2,000 individuals worldwide, primarily males, leading to various physical and cognitive symptoms due to a deficiency of the IDS enzyme [3]. - Current standard treatments only partially address physical symptoms and do not cross the blood-brain barrier, leaving cognitive and behavioral symptoms unmet [3].