FDA accepts for review Synageva's Biologics License Application for Kanuma

The US Food and Drug Administration has accepted for review the Biologics License Application for Kanuma (sebelipase alfa), produced by Synageva BioPharma (Nasdaq: GEVA).

It has been accepted for review for lysosomal acid lipase deficiency, and the FDA has granted Synageva’s request for priority review, shortening the regulatory review period. The FDA has established a target action date of September 8 2015 under the Prescription Drug User Fee Act.

The application included previously-reported data from the global, randomized, double-blind, placebo-controlled Phase III trial in children and adults with lysosomal acid lipase deficiency, and the Phase II/III study of Kanuma in infants with the deficiency. LAL deficiency causes progressive and multisystemic organ damage including cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications. It often manifests in childhood but can be diagnosed at all ages. LAL deficiency is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues, leading to the buildup of fatty material in the liver, blood vessel walls and other tissues.