FDA approves Kanuma for the treatment of patients with lysosomal acid lipase deficiency

Kanuma is the first approved treatment in the US for patients suffering from LAL-D, a life-threatening and ultra-rare metabolic disorder.

“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,” said David Hallal, CEO of Alexion. “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. This approval also strengthens Alexion’s global leadership in rare diseases as we broaden our product portfolio to transform the lives of more patients with severe and life-threatening disorders. We look forward to bringing Kanuma to patients with LAL-D and their physicians in the US.”

“I am delighted that patients with LAL-D now have the first approved therapy that treats the underlying cause of the disease,” said 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. “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. In clinical studies, 67% 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 morbidity and premature mortality. It is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million of the general population.1 Patients with LAL-D can experience a rapid onset of life-threatening disease manifestations, and without treatment, the youngest patients with LAL-D face rapid disease progression that is typically fatal within a matter of months. In addition, similar to other liver diseases, many patients may be asymptomatic until they experience a severe consequence of the disease. LAL-D is caused by genetic mutations that result in a marked decrease or loss in LAL enzyme activity in the lysosomes across multiple body tissues, leading to the chronic build-up of cholesteryl esters and triglycerides in the liver, blood vessel walls, and other organs.

“Patients with LAL-D often suffer for years from a delayed diagnosis, only to be further devastated once properly diagnosed because there have been no approved treatments for this disease,” said Brett Billmeyer, Board of Directors, LAL Solace. “Today, we are thrilled to welcome the FDA approval of Kanuma, providing an approved and effective treatment to patients with LAL-D and their families for the first time and, with it, bringing much-needed awareness to this often overlooked and devastating disease.”

Alexion is preparing to serve patients in the US with Kanuma and expects that Kanuma will become available commercially during the first week of January 2016. The company’s expanded access program will remain open to enable patients with LAL-D in the US to access Kanuma until commercial product is available.

The FDA approved Kanuma under Priority Review, and had previously granted Breakthrough Therapy Designation for Kanuma for LAL-D presenting in infants. With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases. 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.