Details from the Phase 1/2 extension study with sebelipase alfa in adults with LAL Deficiency presented at NASPGHAN
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. All nine patients had a history of dyslipidemia, and seven of nine patients also had a history of other cardiovascular conditions. Seven of nine patients received treatment with lipid modifying therapies including statins, ezetimibe, and other medications.
Eight of nine patients continued treatment with sebelipase alfa as part of an ongoing, open-label extension study. The ninth patient delayed entering the extension study and, while off treatment, experienced progression of liver disease requiring an urgent liver transplant. As previously reported, one patient paused treatment but has recently been deemed eligible to resume treatment by an independent safety committee. One patient continues on treatment and has not yet reached the 78-week time point.
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 (MRI).
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