Genzyme: Lumizyme is FDA approved for Pompe Disease

FDA approved Lumizyme (alglucosidase alfa) for late onset Pompe Disease subject to a risk evaluation and mitigation strategy (REMS) and post marketing study requirements. This is the first treatment in USA specifically for late onset (non infantile) Pompe Disease and is indicated for patients eight years or older who do not have cardiac hypertrophy. It is a new version of Myozyme which is indicated for all forms of the disease and available in Europe as well as USA. Approval was delayed due to manufacturing problems. Pompe Disease is a neuromuscular, autosomal,recessive, metabolic disorder caused by a deficiency in alfa-glucosidase which is needed to break down glycogen.The build up of glucogen causes progressive muscle weakness that can lead to heart, skeletal muscle, liver and nervous system problems. The product carries a black box warning about severe allergic reactions and is only available on a restricted distribution programme called Lumizyme ACE (Alglucosidase Alfa Control and Education) requiring mandatory registration of patients, prescribers and healthcare facilities.