FDA approves Denali Therapeutics’ enzyme therapy for Hunter syndrome, breaking rare disease rejection streak

Denali Therapeutics has received FDA approval for Avlayah, an enzyme replacement therapy targeting Hunter syndrome, a rare genetic lysosomal storage disorder. This approval ends a recent pattern of FDA rejections for rare disease therapies, which had drawn criticism from the biotech community. Industry observers describe Avlayah as a 'game-changer' that could improve treatment options for patients with this debilitating condition and reinvigorate confidence in rare disease drug development.