Key Takeaways FDA accepted DNLI's BLA for tividenofusp alfa in Hunter syndrome, with Priority Review granted. Tividenofusp alfa uses DNLI's TV platform to address both cognitive and physical symptoms of MPS II. A potential approval would be DNLI's first, significantly advancing its move to a commercial biotech.Denali Therapeutics, Inc. (DNLI) announced that the FDA has accepted for review its biologics license application (BLA) for lead-pipeline candidate tividenofusp alfa.The BLA is seeking accelerated a ...

Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 DeficiencyFirst Pivotal Data Readout in Children Expected in Early 2026 with Potential Launch in 2027; Additional Clinical Programs to Expand to Patients of all Ages Potential First-in-Disease Treatment for ENPP1 DeficiencyConference Call and Webcast Scheduled Today at 8:45 a.m. ETSAN RAFAEL, Calif. and BOSTON, May 16, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. ...