, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis Pharmaceuticals Inc. (NASDAQ: ISIS), announced today that the Endocrinologic and Metabolic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) voted
9 to 6
that Genzyme had provided sufficient efficacy and safety data to support the marketing of KYNAMRO™ (mipomersen sodium) for the treatment of patients with Homozygous Familial Hypercholesterolemia (HoFH).
Many people with HoFH have aggressive cardiovascular disease beginning in childhood, and even with today’s therapies remain at significant risk of cardiovascular events.
"We are very encouraged by the support for KYNAMRO at today’s Advisory Committee meeting, which marks a significant and positive step in our efforts to bring this important new therapy to patients and families affected by this often unrecognized genetic disorder,” said David Meeker, President and CEO, Genzyme. “There is still a great need for the HoFH patients, who have exhausted conventional medications and still have LDL cholesterol levels 2-4 times above normal. Genzyme looks forward to working with the FDA as it completes its review of the KYNAMRO application.”
The Committee's input will be considered by the FDA in its review of the New Drug Application for KYNAMRO. The FDA is not bound by the Committee's guidance, but takes its advice into consideration when reviewing investigational medicines. Genzyme submitted the NDA on March 29, 2012, and the FDA has set a target action date under the Prescription Drug User Fee Act (PDUFA) of January 29, 2013. An application for marketing approval of KYNAMRO™ is also pending in the European Union.
In considering the benefits and risks associated with KYNAMRO, the Committee reviewed data from one pivotal Phase 3 double-blind, placebo controlled study in HoFH patients, three supportive Phase 3 studies in other high-risk hypercholesterolemia populations and an ongoing long term extension study.