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 extended Alexion Pharmaceuticals' prescription drug user fee act (PDUFA) date for priority review of its biologics license application (BLA) for Kanuma (sebelipase alfa), an investigational enzyme replacement therapy for the treatment of lysosomal acid lipase deficiency (LAL-D).
Subscribe to our email newsletter
The previously disclosed September 8, 2015 PDUFA date has been extended by the standard extension period of three months.
In response to a recent request from the FDA, Alexion submitted additional Chemistry, Manufacturing and Controls (CMC) information. Due to the timing of this submission, the FDA extended the PDUFA date to allow additional time for review of the new information. The FDA has not asked for additional clinical data.
On September 1, 2015, Alexion announced that the European Commission approved Kanuma for the treatment of patients of all ages with LAL-D. The FDA granted Breakthrough Therapy designation for Kanuma for LAL Deficiency presenting in infants and accepted the Kanuma BLA for Priority Review. In addition, a New Drug Application for Kanuma has been submitted to Japan’s Ministry of Health, Labour and Welfare.
Alexion is reiterating its 2015 financial guidance announced on July 30, 2015.
About Lysosomal Acid Lipase Deficiency (LAL-D)
LAL-D is a genetic, chronic and progressive ultra-rare metabolic disease associated with devastating morbidities and premature mortality. In patients with LAL-D, genetic mutations result in decreased activity of the LAL enzyme. This leads to marked accumulation of cholesteryl esters and triglycerides in vital organs, blood vessels, and other tissues, resulting in progressive and multi-organ damage including fibrosis, cirrhosis, liver failure, accelerated atherosclerosis, cardiovascular disease, and other devastating consequences.1,2
LAL-D affects patients of all ages with clinical manifestations from infancy through adulthood and may have sudden and unpredictable clinical complications. Infants experience profound growth failure, liver fibrosis, and cirrhosis with a median age of death at 3.7 months.3 In an observational study, approximately 50% of children and adults with LAL-D progressed to fibrosis, cirrhosis, or liver transplant in 3 years.4 The median age of onset of LAL-D is 5.8 years and the disease can be diagnosed with a simple blood test.5,6
About Kanuma (sebelipase alfa)
Kanuma (sebelipase alfa) is an innovative enzyme replacement therapy designed to address the underlying cause of lysosomal acid lipase deficiency (LAL-D) by aiming to reduce substrate accumulation in the lysosomes of cells throughout the body, including the liver, to prevent vital organ damage and premature death.