Synageva BioPharma announced that the Phase 3 ARISE trial of sebelipase alfa in patients with lysosomal acid lipase deficiency (LAL Deficiency) met the primary endpoint.

ARISE is a global, double-blind, placebo-controlled trial of sebelipase alfa in 66 children and adults with LAL Deficiency. Patients were randomized 1:1 to every other week infusions of sebelipase alfa 1mg/kg or placebo for the double-blind treatment period of 20 weeks. Dyslipidemia was common at baseline, with a median LDL cholesterol of 204mg/dl, and an abnormally low median HDL cholesterol of 32.5mg/dl. The primary endpoint was the normalization of ALT (P=0.027). In addition, sebelipase alfa significantly improved multiple other disease-related abnormalities as measured by a number of secondary endpoints.

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In addition to demonstrating a statistically significant improvement in ALT normalization, sebelipase alfa improved dyslipidemia with statistically significant reductions in LDL cholesterol (P<0.001), non-HDL cholesterol (P<0.001) and triglycerides (P=0.038), as well as a statistically significant increase in HDL cholesterol (P<0.001), all compared with placebo from baseline to the completion of the double-blind treatment period of 20 weeks. Statistically significant improvements were also seen in AST normalization (P<0.001) and in liver fat fraction as assessed by multi-echo gradient echo (MEGE) magnetic resonance imaging (MRI) (P<0.001).

Synageva plans to submit a Biologic License Application (BLA) to the FDA for sebelipase alfa for the treatment of LAL.

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