CHMP recommends Kanuma (sebelipase alfa) to treat lysosomal acid lipase deficiency - Alexion Pharma

Alexion Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted positive opinion recommending marketing authorization of Kanuma (sebelipase alfa). The proposed indication for Kanuma is for long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency (LAL-d). Based on the CHMP’s positive recommendations, final decisions from the European Commission are expected in the third quarter of 2015, after which the Company will begin the country-by-country reimbursement processes. Currently, there are no therapies approved for the treatment of LAL-d.

LAL-d is a genetic, progressive, ultra-rare metabolic disease in which patients ranging from infants to adults experience chronic lipid accumulation causing multi-systemic organ damage and premature death. It is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues, leading to the chronic build-up of fatty material in the liver, blood vessel walls and other tissues.The proposed indication for Kanuma is for long-term enzyme replacement therapy in patients of all ages with LAL-d. As noted in the CHMP Summary of Opinion, the benefits of Kanuma are its ability to replace the activity of the missing enzyme resulting in reduced liver fat content and reduced levels of blood transaminases, low-density lipoprotein (LDL) cholesterol, non-high-density lipoprotein (HDL) and triglycerides. In addition, there was a significant benefit in terms of survival (67%) in patients with the infant form of LAL-d beyond 12 months. The CHMP reviewed Kanuma under accelerated assessment. The CHMP based its opinion on clinical data from four clinical studies in which 84 patients with LAL-d (including infants, children, and adults) were treated with Kanuma.

Comment; Kanuma is filed with the FDA and was granted a Priority Review with target action date of 8 September 2015.The FDA is not planning an Advisory Committee Meeting for Kanuma.The drug has also been filed in Japan.