Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare disorders, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL Deficiency).  The FDA granted the company's request for Priority Review, which shortens the regulatory review period and is reserved for investigational therapies that treat serious conditions and, if approved, would provide a significant improvement in safety or effectiveness compared to available therapies.  The FDA established a target action date of September 8, 2015 under the Prescription Drug User Fee Act (PDUFA).  

The BLA for Kanuma included previously reported data from the global, randomized, double-blind, placebo controlled Phase 3 trial in children and adults with LAL Deficiency, and the Phase 2/3 trial of Kanuma in infants with LAL Deficiency.  Patients in these trials, combined with patients in other ongoing clinical trials with Kanuma, represent the largest patient population studied to date for this rare, devastating disease.

For more details, go to: http://ir.synageva.com/releasedetail.cfm?ReleaseID=897611