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 US Food and Drug Administration (FDA) has approved Alexion Pharmaceuticals' Kanuma (sebelipase alfa) to treat patients with lysosomal acid lipase deficiency (LAL-D).
Subscribe to our email newsletter
LAL-D is caused by genetic mutations that lead to a marked decrease or loss in LAL enzyme activity in the lysosomes across several body tissue.
Kanuma reduces substrate accumulation in the lysosomes of cells throughout the body.
In clinical studies, treatment with Kanuma enhanced survival in infants with LAL-D and led to reductions in ALT and liver fat content, apart from improvements in lipid parameters, in children and adults with LAL-D.
Kanuma is expected to go on sale in the US in the first week of January 2016.
Alexion Pharmaceuticals CEO David Hallal said: "We are pleased with the FDA approval of Kanuma, a transformative treatment for patients with LAL-D, a devastating, ultra-rare disease that causes premature death in infants and multi-organ damage in those who survive.
"Importantly, the label includes a survival benefit in infants and reductions in important markers of liver disease, including ALT and liver fat content, as well as significant improvements in lipid parameters, in children and adults."
Kanuma is also approved in the European Union. A new drug application for the enzyme replacement therapy has been submitted to Japan’s Ministry of Health, Labour and Welfare.