FDA extends review of Denali’s Hunter syndrome treatment

Published 13/10/2025, 21:06
FDA extends review of Denali’s Hunter syndrome treatment

SOUTH SAN FRANCISCO - Denali Therapeutics Inc. (NASDAQ:DNLI) announced Monday that the U.S. Food and Drug Administration has extended its review timeline for tividenofusp alfa, a treatment for Hunter syndrome. The company, currently valued at $2.2 billion, is trading near InvestingPro’s Fair Value estimate, with analysts setting price targets between $24 and $40.

The FDA pushed the target action date from January 5, 2026, to April 5, 2026, following the company’s submission of updated clinical pharmacology information. The agency classified this submission as a Major Amendment to the Biologics License Application (BLA), triggering the three-month extension.

Denali emphasized that the extension resulted from a standard information request and is not related to efficacy, safety, or biomarker concerns. No additional data were requested by the FDA in the Major Amendment letter.

"We appreciate the FDA’s continued collaboration throughout the review process," said Ryan Watts, Chief Executive Officer of Denali Therapeutics. "We continue to prepare for the potential approval and commercial launch of tividenofusp alfa."

Tividenofusp alfa, also known as DNL310, combines the iduronate 2-sulfatase enzyme with Denali’s proprietary TransportVehicle platform. The therapy is designed to cross the blood-brain barrier to address both neurological and physical symptoms of Hunter syndrome, a rare genetic disorder that current treatments cannot fully address.

The FDA has previously granted Fast Track and Breakthrough Therapy designations to tividenofusp alfa for Hunter syndrome treatment. The European Medicines Agency has also designated it as a Priority Medicine.

The announcement was based on a press release statement from the company.

In other recent news, Denali Therapeutics has been the focus of positive analyst ratings. Cantor Fitzgerald has reiterated its Overweight rating on the company, maintaining a positive outlook on Denali Therapeutics’ potential growth. The research firm suggests that Denali could become as significant as Genzyme, a company renowned for its enzyme replacement therapies. Additionally, BTIG has maintained its Buy rating on Denali Therapeutics, setting a price target of $32.00. This rating is attributed to the company’s advancements in blood-brain barrier shuttle technology. BTIG noted that Denali’s DNL310 has demonstrated a dosing capacity significantly higher than JCR’s approved version, with an acceptable safety profile. These developments indicate continued confidence in Denali Therapeutics from major research firms.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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