EU approves Kanuma (sebelipase alfa) an enzyme replacement therapy for patients with lysosomal acid lipase deficiency.-Alexion Pharmaceuticals

Alexion Pharmaceuticals, Inc. announced that the European Commission (EC) has approved Kanuma (sebelipase alfa) for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase deficiency (LAL-D). Kanuma, an innovative ERT, is the first approved treatment in the European Union for patients with LAL-D, a genetic and progressive ultra-rare metabolic disease. 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.

LAL-D is a genetic, chronic and progressive metabolic disease in which infants, children and adults suffer multi-organ damage and premature death. It is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million of the general population. Patients with LAL-D often experience a rapid onset of life-threatening disease manifestations, and similar to other liver diseases, many patients may be asymptomatic until they experience a severe consequence of the disease. LAL-D is caused by genetic mutations that result in a marked decrease or loss in LAL enzyme activity in the lysosomes across multiple body tissues, leading to the chronic build-up of cholesteryl esters and triglycerides in the liver, blood vessel walls and other tissues.