FDA APPROVED March 25, 2026 — accelerated approval, 11 days before PDUFA target date of April 5. First FDA-approved biologic engineered to cross the blood-brain barrier using Denali's TransportVehicle™ (TV) platform (targets transferrin receptor). First enzyme replacement therapy that reaches both the body and the brain for Hunter syndrome. Supported by Phase 1/2 data published in NEJM. Also received Rare Pediatric Disease Priority Review Voucher. Market cap ~$3.1B (sub-$7B). Launch planned within weeks of approval via Denali Patient Services. Next catalyst: BIIB122/DNL151 (LRRK2 inhibitor for Parkinson's) in Phase 3 with Biogen.
⚠️ Not Investment Advice: This information is provided for educational purposes only.
PDUFA dates are FDA target action dates, not guaranteed approval dates. The FDA may extend the review period,
issue Complete Response Letters, or take unexpected actions. Probability of approval scores are based on
historical base rates and do not account for drug-specific factors. Always consult a financial advisor
before making investment decisions.
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