Alexion Pharmaceuticals reports that the FDA has approved Kanuma™ (sebelipase alfa) for the treatment of patients of all ages with a diagnosis of lysosomal acid lipase deficiency (LAL-D).
Kanuma, an enzyme replacement therapy, is the first therapy approved in the U.S. for the treatment of patients with LAL-D, a genetic and progressive ultra-rare metabolic disease in which patients suffer multi-organ damage and premature death.