Lysosomal Acid Lipase Deficiency (LAL)

July 14, 2014

Synageva BioPharma issued results of a phase III trial of sebelipase alfa in 66 children and adults with lysosomal acid lipase deficiency (LAL Deficiency). The global, randomized, double-blind, placebo-controlled study met the primary endpoint of normalization of alanine aminotransferase (ALT), a marker of liver injury (p=0.027). Patients enrolled in the trial were randomized on a one-to-one basis to every other week infusions of sebelipase alfa (1mg/kg) or placebo for the double-blind treatment period of 20 weeks. 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 also were 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). Together with safety and efficacy data from the phase II/III study in infants with rapidly progressive LAL Deficiency, data from this phase III study in children and adults will be used to support global submissions for product registration.