7:01AM Synageva Biopharma highlights data from Phase 1/2 extension study with sebelipase alfa in adults with LAL Deficiency; results show sustained improvements in biochemical markers of liver damage (GEVA) 58.91 : Co reportes 78-week results from an ongoing extension study with sebelipase alfa in adults with Lysosomal Acid Lipase Deficiency.
Nine adults with LAL Deficiency with a median age of 29 years (range 19-45) enrolled in the Phase 1/2 trial. Seven of nine patients had a history of hepatomegaly and/or splenomegaly, and two of nine patients had evidence of more advanced liver disease, including cirrhosis and portal hypertension.
At 78 weeks of treatment with sebelipase alfa, patients continued to have sustained reductions in both ALT and AST, frequently into the normal range, from the pre-treatment baseline. In addition, sebelipase alfa maintained improvement in dyslipidemia associated with LAL Deficiency with decreases in LDL and triglycerides and increases in HDL from the pre-treatment baseline to week 78 of the extension study. Sebelipase alfa also reduced patients' liver fat fraction and liver volume from the beginning of the extension study to week 52, the latest time point for these assessments. Liver fat fraction and liver volume were measured by multi-echo magnetic resonance imaging.
Sebelipase alfa was generally well tolerated through 78 weeks of the extension study. Most adverse events were mild and unrelated to sebelipase alfa.
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