Fierce Pharma

FDA approves Denali's Hunter syndrome drug, handing rare disease community a win

The FDA has approved Denali Therapeutics’ enzyme replacement therapy for a genetic lysosomal storage disease, breaking the ...
STAT

FDA approves Denali Therapeutics drug for Hunter syndrome

The FDA approved a new medicine from Denali Therapeutics for Hunter syndrome, a notable decision by the agency after a string ...
Healio

FDA approves Avlayah to treat neurologic manifestations of Hunter syndrome

The FDA has given accelerated approval to Avlayah as a treatment for neurologic manifestations of Hunter syndrome, also known ...
BioSpace

Denali Delivers ‘Welcome Positive’ for Rare Disease Space With FDA’s Hunter Syndrome Nod

Avlayah is the first Hunter syndrome therapy approved to address the condition’s neurologic complications, according to Tracy ...
MPR

Avlayah Approved for Neurologic Manifestations of Hunter Syndrome

FDA accelerated approval of tividenofusp alfa-eknm offers a new treatment for neurologic manifestations in patients with Hunter syndrome.

Denali Therapeutics Announces U.S. FDA Approval of AVLAYAH™ (tividenofusp alfa-eknm) for Treatment of Hunter Syndrome (MPS II)

First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage diseaseFirst FDA-approved ...
MedPage Today on MSN

FDA Signals Regulatory Flexibility With Accelerated Approval of Rare Disease Drug

Tividenofusp alfa is the first approved treatment for neurologic complications of Hunter s ...
BioPharma Dive

FDA clears Denali drug in ‘clear step’ for rare disease biotechs

The approval of Denali’s Hunter syndrome treatment, Avlayah, comes after a series of drug rejections and delays that had led ...

Denali Wins FDA Nod for Hunter Syndrome Drug, Stock Up

DNLI surges after FDA approves Avlayah, its first commercial drug and a breakthrough Hunter syndrome therapy targeting the brain.

Denali Therapeutics CEO: 2026 Pivotal With Hunter Syndrome FDA Decision and New TV Trial Readouts

Denali Therapeutics (NASDAQ:DNLI) founder and CEO Ryan Watts said 2026 is shaping up to be a pivotal year for the company as ...
Technology Networks

FDA Approves Therapy for Neurological Hunter Syndrome Symptoms

A therapy for Hunter syndrome shows a significant reduction in disease-linked molecules in cerebrospinal fluid. Findings ...