South San Francisco, California — October 30, 2026
Denali Therapeutics Inc. announced that AVLAYAH® (tividenofusp alfa-eknm) has received the 2026 Prix Galien USA Award for Best Biotechnology Product from The Galien Foundation. AVLAYAH is an enzyme replacement therapy approved by the U.S. Food and Drug Administration (FDA) in March 2026 for the treatment of neurologic manifestations of Hunter syndrome (mucopolysaccharidosis type II, or MPS II) in eligible pediatric patients weighing at least 5 kg before advanced neurologic impairment. It is the first FDA-approved biologic specifically engineered to cross the blood-brain barrier (BBB) and deliver therapeutic material to both peripheral tissues and the brain. The award was presented during a ceremony at the American Museum of Natural History in New York City, recognizing scientific innovation and contributions to patient care. The Galien Foundation selects award recipients through committees comprising leaders in science and medicine. AVLAYAH represents the first new FDA-approved treatment option in nearly 20 years for families affected by Hunter syndrome. However, its accelerated approval is based on a reduction in heparan sulfate in cerebrospinal fluid, and continued approval may depend on confirmation of clinical benefit in an ongoing confirmatory study
Prix Galien Recognition and TransportVehicle Technology
The Prix Galien USA award recognizes biotechnology products approved by the FDA within the previous five years and is regarded as a major distinction in the biopharmaceutical industry. Denali said the recognition reflects years of research focused on overcoming the BBB delivery challenge through its proprietary TransportVehicle™ (TV) technology. The platform uses engineered Fc domains that bind to naturally occurring transport receptors, including the transferrin receptor (TfR), to enable receptor-mediated transcytosis across the BBB. AVLAYAH is the first and only FDA-approved TfR-enabled medicine specifically engineered to cross this barrier. Its design allows delivery of an enzyme replacement therapy to the central nervous system as well as peripheral tissues. Denali CEO and co-founder Ryan Watts, Ph.D., said the award recognizes the scientific work behind the platform and the contributions of the Hunter syndrome community, including patients, families, advocates and clinicians. The company believes that the ability to deliver biologic medicines to the brain could support development of therapies for additional rare genetic and neurodegenerative diseases. The broader platform is designed to transport enzymes, antibodies and oligonucleotides, although the performance and clinical value of each candidate must be evaluated independently.
AVLAYAH Approval and Hunter Syndrome Treatment
AVLAYAH (tividenofusp alfa-eknm) is an intravenous enzyme replacement therapy consisting of the iduronate 2-sulfatase (IDS) enzyme fused to Denali’s TransportVehicle platform. Its engineered Fc component binds to the apical domain of the transferrin receptor, enabling transport across the BBB. Following delivery, the IDS enzyme is taken up by cells through the mannose-6-phosphate receptor and transported into lysosomes, where it is intended to break down accumulated glycosaminoglycans (GAGs). Hunter syndrome is a rare inherited lysosomal storage disorder caused by mutations in the IDS gene, resulting in deficiency of the IDS enzyme and accumulation of GAGs, including heparan sulfate and dermatan sulfate. This buildup can progressively damage multiple organs and tissues, including the brain, and lead to developmental delays, cognitive decline, behavioral abnormalities, joint stiffness, hearing loss and organ dysfunction. The disease primarily affects boys and is estimated to affect approximately 500 people in the United States and 2,000 worldwide. AVLAYAH is approved for pediatric patients weighing at least 5 kg who have neurologic symptoms before advanced neurologic disease. Its accelerated approval was based on reduction of heparan sulfate in cerebrospinal fluid; studies are ongoing to verify clinical benefit. The therapy is not approved outside the United States. The FDA has also granted AVLAYAH Rare Pediatric Disease, Breakthrough Therapy, Fast Track and Orphan Drug designations.
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Safety Information and Denali’s Expanding Pipeline
AVLAYAH carries important safety risks, including hypersensitivity reactions and anaphylaxis, which can be life-threatening and may occur early or after repeated treatment. Other warnings include infusion-associated reactions, anemia and membranous nephropathy. The most common adverse reactions, reported in at least 20% of patients, include infusion-associated reactions, upper respiratory infections, ear infections, fever, anemia, cough, vomiting, diarrhea, rash, COVID-19, nasal congestion, falls, headache, skin injuries and hives. Patients require monitoring during treatment, including attention to infusion reactions, hemoglobin levels and kidney function. Denali is also developing additional TransportVehicle-enabled candidates. These include zafinofusp alfa, an investigational enzyme replacement therapy for mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A); DNL628 (OTV), an antisense oligonucleotide designed to reduce tau by targeting the MAPT gene for Alzheimer’s disease; and DNL921 (ATV), an antibody-based candidate designed to target amyloid plaques. The company reports that preclinical studies of its platform have demonstrated increased brain exposure for engineered biologics and oligonucleotides compared with corresponding molecules without the technology. These investigational programs remain in development, and their potential clinical benefits have not yet been established. Denali is headquartered in South San Francisco, California, and focuses on developing therapies for neurodegenerative diseases, lysosomal storage disorders and other serious conditions.
Source:Denali Therapeutics, Press release
