Cheshire-based Alexion Pharmaceuticals Inc. on Tuesday said the U.S. Food and Drug Administration has approved Kanuma for the treatment of patients with lysosomal acid lipase deficiency (LAL-D), a genetic and progressive ultra-rare metabolic disease that causes multi-organ damage and premature death.
Kanuma, an enzyme replacement therapy (ERT), is the first therapy approved in the U.S. for the treatment of patients with LAL-D, according to a news release from Alexion.
“In the absence of treatment, LAL-D is nearly always fatal in infants and puts pediatric and adult patients at high risk of vital organ damage and premature mortality,” Dr. Barbara K. Burton, lead clinical trial investigator, professor of pediatrics at the Northwestern University Feinberg School of Medicine and attending physician at the Ann and Robert H. Lurie Children’s Hospital of Chicago, said in the release. “In clinical studies, 67 percent of infants who received enzyme replacement therapy survived beyond 12 months of age, and children and adults had meaningful improvements in multiple disease-related liver and lipid abnormalities.”
LAL-D is a genetic, chronic, and progressive metabolic disease associated with significant premature mortality. It affects fewer than 20 patients per 1 million of the general population. Patients with LAL-D can experience a rapid onset of life-threatening disease, and without treatment, the youngest patients with LAL-D face rapid disease progression that is typically fatal within a matter of months, the release said.
Alexion is preparing to serve patients in the U.S. with Kanuma and expects that Kanuma will become available commercially during the first week of January.
Kanuma is also approved in the European Union, and a new drug application for Kanuma has been submitted to Japan’s Ministry of Health, Labour and Welfare.
