Denali Therapeutics Gains FDA Approval for AVLAYAH Therapy

An update from Denali Therapeutics ( (DNLI) ) is now available.

On March 25, 2026, Denali Therapeutics announced that the U.S. Food and Drug Administration granted accelerated approval to AVLAYAH (tividenofusp alfa-eknm), an enzyme replacement therapy for neurologic manifestations of Hunter syndrome in presymptomatic or symptomatic pediatric patients weighing at least 5 kg before advanced neurologic impairment. The therapy, the first new U.S. treatment for Hunter syndrome in nearly two decades and the first FDA-approved brain-penetrant biologic leveraging the transferrin receptor to cross the blood-brain barrier, is enabled by Denali’s TransportVehicle platform and earned the company a Rare Pediatric Disease Priority Review Voucher.

The approval is based on Phase 1/2 data showing a 91% reduction in cerebrospinal fluid heparan sulfate, with most treated children reaching biomarker levels comparable to individuals without the disease, while continued approval depends on confirmatory results from the ongoing global Phase 2/3 COMPASS trial. AVLAYAH, administered once weekly and expected to be available in the U.S. shortly after approval, is viewed by clinicians and patient advocates as a potential new standard of care that addresses long-standing neurologic needs in Hunter syndrome and exemplifies a biomarker-driven regulatory pathway that could speed development of therapies for other rare pediatric conditions.

The most recent analyst rating on (DNLI) stock is a Buy with a $30.00 price target. To see the full list of analyst forecasts on Denali Therapeutics stock, see the DNLI Stock Forecast page.

Spark’s Take on DNLI Stock

According to Spark, TipRanks’ AI Analyst, DNLI is a Neutral.

The score is held back primarily by weak financial performance (widening losses, zero revenue in 2024–2025, and ongoing cash burn). This is partly offset by strong technical momentum (price above key moving averages with positive MACD) and constructive corporate events that improve funding and highlight meaningful upcoming regulatory/clinical milestones, while valuation remains constrained by loss-making results and no dividend.

To see Spark’s full report on DNLI stock, click here.

More about Denali Therapeutics

Denali Therapeutics Inc., based in South San Francisco and listed on Nasdaq as DNLI, is a biopharmaceutical company focused on developing biologic medicines that can cross the blood-brain barrier to treat neurodegenerative and lysosomal storage diseases. Its proprietary TransportVehicle platform is designed to deliver enzyme replacement and other biotherapeutics to the whole body, including the central nervous system, targeting rare conditions such as Hunter syndrome (MPS II).

Average Trading Volume: 1,633,416

Technical Sentiment Signal: Buy

Current Market Cap: $3.34B

See more data about DNLI stock on TipRanks’ Stock Analysis page.