The U.S. Food and Drug Administration extended on Monday Denali Therapeutics Inc.’s (NASDAQ:DNLI) review timeline of the Biologics License Application (BLA), seeking accelerated approval of tividenofusp alfa for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
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The Prescription Drug User Fee Act (PDUFA) target date has been extended from Jan. 5, 2026, to April 5, 2026.
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The extension follows Denali’s submission of updated clinical pharmacology information in response to an information request from the FDA as part of the standard review process and is not related to efficacy, safety or biomarkers. The FDA requested no additional data.
Denali believes that the updated information submitted in the amendment does not affect the clinical pharmacology or benefit-risk conclusions of the BLA.
The Phase 2/3 COMPASS study is enrolling participants with MPS II in North America, South America and Europe to support global approval.
William Blair noted that the company found a molecular weight discrepancy for tividenofusp in a public database, which it reported to the FDA. The issue impacts several label-related calculations. Management said that since the discrepancy surfaced late in the review process — after the midcycle review — it was procedural for the FDA to issue a major amendment.
On Monday, analyst Myles Minter wrote, “While the PDUFA extension is disappointing, we view this as a technicality and remain confident in the approvability of tivi with the current clinical datasets. We continue to believe that there is regulatory precedence from the prior approval in Hunter syndrome of Takeda's peripherally restricted Elaprase, and are positive on an eventual approval…”
DNLI Price Action: Denali Therapeutics stock is down 0.87% at $14.85 at publication on Tuesday.
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