Alexion's Kanuma becomes first LAL-D therapy to be approved by FDA

An innovative enzyme replacement therapy (ERT) has become the first treatment for patients with lysosomal acid lipase deficiency (LAL-D) to be approved by the US Food and Drug Administration, it has been announced.

Kanuma (sebelipase alfa), made by US company Alexion Pharmaceuticals (Nasdaq: ALXN), was approved under the Priority Review process after it was granted Breakthrough Therapy designation.

LAL-D is a rare genetic and progressive metabolic disease in which patients suffer multi-organ damage and premature death. It is defined as an ultra-rare disease, which means it affects fewer than 20 patients per one million of the general population.