Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted positive opinions recommending marketing authorization of Alexion Pharmaceuticals' Strensiq (asfotase alfa) and Kanuma (sebelipase alfa).
Subscribe to our email newsletter
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.
anuma is proposed for long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency (LAL-d).
Based on the positive recommendations from CHMP, final decisions from the European Commission (EC) are expected in the third quarter of this year.
There are currently no therapies approved to treat HPP or LAL-d, which are genetic, progressive, ultra-rare metabolic diseases.
Alexion CEO David Hallal said: "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.
"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."
The CHMP positive opinion for Strensiq is based on clinical data from 68 patients with pediatric-onset HPP (ranging from newborns to 66 years of age) enrolled in three pivotal prospective studies and their extensions.
The CHMP reviewed Kanuma under accelerated assessment and the positive opinion is 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.