EU Recommends Approval for Alexion's Rare Disease Drugs
News Jun 30, 2015
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.
“The CHMP positive opinions for Strensiq and Kanuma are significant milestones in bringing these therapies to infants, children, and adults suffering from HPP and LAL-d in Europe,” said David Hallal, Chief Executive Officer of Alexion. “Both Strensiq and Kanuma are highly innovative enzyme replacement therapies that, if approved, will be the first treatments available for patients with HPP and LAL-d, two life-threatening and ultra-rare metabolic disorders.”
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.
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.