Synageva Hits Enrollment Target

Synageva BioPharma has met the enrollment target of 50 patients in its global Phase III trial of sebelipase alfa in children and adults with lysosomal acid lipase deficiency (LAL Deficiency). To accommodate individuals still in the screening process, total enrollment will exceed the planned number of 50 patients, with the final patient expected to be randomized by the end of this year. As a result of this enrollment update, the company expects to report top-line results from the ARISE trial during the 2H14.

The ARISE trial is a randomized, double-blind, placebo-controlled study of sebelipase alfa in children and adults with LAL Deficiency and is designed to assess the effects of sebelipase alfa on a broad range of abnormalities associated with the disease. Patients enrolled in the trial are randomized to infusions of sebelipase alfa (1 mg/kg, every other week) or placebo for the 20-week, double-blind treatment period and then allowed to enter into a long-term, open-label extension period. The efficacy and safety results from the double-blind treatment period are expected to support global submissions for product registration.