Denali Therapeutics gains FDA accelerated approval for AVLAYAH in Hunter syndrome; CSF heparan sulfate drops 91% by week 24

Reuters03-25
Denali <a href="https://laohu8.com/S/LENZ">Therapeutics</a> gains FDA accelerated approval for AVLAYAH in Hunter syndrome; CSF heparan sulfate drops 91% by week 24
  • The FDA granted accelerated approval to Denali’s AVLAYAH (tividenofusp alfa-eknm) to treat neurologic manifestations of Hunter syndrome (MPS II) in presymptomatic or symptomatic pediatric patients weighing at least 5 kg before advanced neurologic impairment.
  • The approval was based on cerebrospinal fluid heparan sulfate reduction as a surrogate endpoint, including a 91% reduction by week 24 in a Phase 1/2 trial.
  • The FDA also granted Denali a Rare Pediatric Disease Priority Review Voucher in connection with the approval.


Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Denali Therapeutics Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: 202603251150PRIMZONEFULLFEED9678477) on March 25, 2026, and is solely responsible for the information contained therein.

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