Data from Pharmawand - Curated by EPG Health - Date added 02 September 2015
Alexion Pharmaceuticals, Inc. announced that the European Commission has approved Kanuma (sebelipase alfa) for long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency (LAL-D). Kanuma is the first approved treatment in the European Union for patients with LAL-D, a genetic and progressive ultra-rare metabolic disease in which patients suffer multi-organ damage and premature death. Alexion expects to begin serving patients in Germany in October and is now commencing reimbursement processes with healthcare authorities in each of the major European countries.
The approval of Kanuma in the EU was based on data from two clinical studies and a supporting open-label extension study comprising infant, paediatric, and adult patients with LAL-D. Study results showed significant benefit in terms of survival (67%, or 6 out of 9) in patients with the infant form of LAL-D beyond 12 months, compared with 0 out of 21 patients in an untreated historical cohort. Infant patients treated with Kanuma also had improvements in liver parameters, including ALT and AST, as well as weight gain within the first several weeks of treatment. In paediatric and adult patients with LAL-D, treatment with Kanuma resulted in normalisation of ALT, reduction in liver fat content and other markers of liver injury compared to placebo, as well as significant improvements in lipid accumulation as measured by LDL-C and HDL-C. In patients who received Kanuma during the double-blind period and subsequently entered the open-label extension period, reductions in ALT levels were maintained and further improvements were seen in LDL-C and HDL-C.