MedCity News February 6, 2025
Currently available therapies for Hunter syndrome don’t cross the blood-brain barrier to address cognitive symptoms of the inherited enzyme deficiency. Denali Therapeutics’ technology gets its drug across the protective membrane, and the biotech plans to seek accelerated FDA approval in this rare disease.
A Denali Therapeutics drug for the rare enzyme deficiency Hunter syndrome is still in pivotal testing, but the company has guidance from the FDA on a pathway to get this therapy to the market sooner. Denali now has additional data from an earlier study that will be the main component of an application seeking accelerated approval.
The updated Phase 1/2 data, presented Thursday during the WORLDSymposium conference in San Diego, continue to show benefit for patients, some...
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