Denali: FDA approves AVLAYAH (tividenofusp alfa) for Hunter syndrome

FDA granted accelerated approval for AVLAYAH to treat neurologic manifestations of Hunter syndrome in pediatric patients ≥5 kg. The approval marks the first medicine using transferrin receptor to cross the blood-brain barrier. Commercial launch is underway with first patients treated in April 2026.

Key facts

Filed: May 7, 2026, 08:03 PM
Event: FDA approval
Direction: Positive
Company: DNLIDenali Therapeutics
Asset: tividenofusp alfa
Source: SEC 8-K
Confidence: Sourced
Calendar: FDA decision (PDUFA) · Apr 5, 2026 · Approved

Source excerpt

EX-99.1 2 ex991pressreleaseq12026.htm EX-99.1 Document Exhibit 99.1 Denali Therapeutics Reports First Quarter 2026 Financial Results and Business Highlights • FDA approved AVLAYAH™ (tividenofusp alfa-eknm) for treatment of Hunter syndrome (MPS II) and as first medicine to leverage transferrin receptor to cross blood-brain barrier • AVLAYAH launched in U.S. with strong momentum, vibrant community engagement, and first patients treated in commercial setting in April • Broad clinical pipeline progressing for lysosomal storage and neurodegenerative diseases, including first patient dosed with Oligonucleotide TransportVehicle™(OTV)-enabled DNL628 (OTV:MAPT) targeting tau for Alzheimer's disease • Advancing DNL593 (PTV:PGRN) in Phase 1/2 study for GRN-related frontotemporal dementia after regaining full rights, with data expected by end of 2026 SOUTH SAN FRANCISCO, Calif., – May 7, 2026 – Denali Therapeutics Inc. (Nasdaq: DNLI) today reported financial results for the first quarter ended March 31, 2026, and provided business highlights, including the recent U.S. Food and Drug Administration (FDA) approval of AVLAYAH™ (tividenofusp alfa-eknm). “The FDA approval of AVLAYAH is a major milestone for Denali, for the Hunter syndrome community, and for the field of biotherapeutics enabled to cross the blood-brain barrier. We are thrilled by the strong engagement with the community, seamless execution by our commercial team, and achievement of our first patient dosed in less than o

SEC 8-K