Synageva BioPharma reports positive top-line results from Phase III study of Sebelipase Alfa

Synageva BioPharma (Nasdaq: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, has announced that the global, randomized, double-blind, placebo-controlled Phase III ARISE trial of sebelipase alfa met the primary endpoint of normalization of alanineaminotransferase, a marker of liver injury.

In addition, sebelipase alfa significantly improved multiple other disease-related abnormalities as measured by a number of secondary endpoints. LAL Deficiency patients enrolled in the trial presented with multiple clinically important abnormalities at baseline. Fibrosis and/or cirrhosis was documented in 100% of patients who had baseline biopsies even though the median age of patients enrolled in the trial was only 13 years. Dyslipidemia was common at baseline, with a median LDL cholesterol of 204 mg/dl (which is in the very high category of >190 mg/dl), and an abnormally low median HDL cholesterol of 32.5 mg/dl.

Impact on Dyslipidemia