USFDAMay 7, 2026, 04:04 PM
Denali's AVLAYAH Receives FDA Approval for Hunter Syndrome
AI Summary
Denali Therapeutics announced the FDA approval of AVLAYAH (tividenofusp alfa-eknm) for Hunter syndrome, marking the first medicine to utilize the transferrin receptor to cross the blood-brain barrier. The company has successfully launched AVLAYAH in the U.S., with the first patients already receiving commercial treatment. Denali also reported progress across its clinical pipeline, including dosing the first patient with DNL628 for Alzheimer's disease and advancing DNL593 for FTD-GRN after regaining full rights. For the first quarter of 2026, the company reported a net loss of $128.4 million and maintained a strong cash position of approximately $1.05 billion.
Key Highlights
- FDA approved AVLAYAH for Hunter syndrome, the first medicine to leverage transferrin receptor to cross the blood-brain barrier.
- AVLAYAH launched in the U.S. with first patients treated commercially in April.
- First patient dosed with OTV-enabled DNL628 (OTV:MAPT) for Alzheimer's disease.
- DNL593 (PTV:PGRN) Phase 1/2 study for FTD-GRN completed enrollment, with results expected by end of 2026.
- Net loss for Q1 2026 was $128.4 million, compared to $133.0 million in Q1 2025.
- Research and development expenses decreased by $12.4 million to $103.8 million in Q1 2026.
- Cash, cash equivalents, and marketable securities totaled approximately $1.05 billion as of March 31, 2026.
- Received $200 million from Royalty Pharma Investments 2023 ICAV in connection with a synthetic royalty funding agreement.