WASHINGTON (dpa-AFX) - Alexion Pharmaceuticals Inc. (ALXN) announced that the Committee for Medicinal Products for Human Use or CHMP of the European Medicines Agency (EMA) has adopted positive opinions recommending marketing authorization of Strensiq (asfotase alfa) and Kanuma (sebelipase alfa).
The proposed indication for Strensiq is for long-term enzyme replacement therapy in patients with pediatric-onset hypophosphatasia (HPP) to treat the bone manifestations of the disease.
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 HPP or LAL-d.
HPP is a genetic, progressive, ultra-rare metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to debilitating or life-threatening complications. It is characterized by defective bone mineralization that can lead to deformity of bones and other skeletal abnormalities, as well as systemic complications such as profound muscle weakness, seizures, pain and respiratory failure leading to premature death in infants.
Copyright RTT News/dpa-AFX