FDA accepts NDA filing for KYNAMRO to treat HoFH

May 29 2012

Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis Pharmaceuticals Inc. (NASDAQ: ISIS), announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for KYNAMRO^™ (mipomersen sodium) for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). The NDA filing with the FDA triggers a $25 million milestone payment to Isis from Genzyme.

“The NDA filing with the FDA represents a significant achievement in the development of KYNAMRO™ and our efforts to get this important new drug to the market for patients who are at high-risk of a cardiovascular event”

"The NDA filing with the FDA represents a significant achievement in the development of KYNAMRO^™ and our efforts to get this important new drug to the market for patients who are at high-risk of a cardiovascular event," said David Meeker, M.D., President and CEO, Genzyme. "We look forward to continuing the review process with the U.S. and EU regulatory authorities to bring KYNAMRO^™ to patients in need."

Genzyme submitted an application for U.S. marketing approval of KYNAMRO^™ for the treatment of patients with HoFH in March 2012. The application will be subject to a standard review and will have a Prescription Drug User Fee Act (PDUFA) date of January 29, 2013. In July 2011, Genzyme submitted an application for EU marketing approval of KYNAMRO^TM for the treatment of patients with HoFH and severe heterozygous FH (Severe HeFH).

"We believe that KYNAMRO^™ has the potential to bring real benefit to patients in the United States with HoFH who are unable to adequately control their LDL-C with currently available treatments," said B. Lynne Parshall, Chief Operating Officer and CFO of Isis. "The successes of our joint development efforts for KYNAMRO^™ are evident in the significant progress made in bringing this important new drug to the regulatory agencies for review. We are pleased to have earned the first regulatory milestone payment for KYNAMRO^™ from this collaboration."

The FDA submission for KYNAMRO™ is supported by the largest clinical trial conducted to date in the HoFH patient population. In the randomized, double-blind, placebo controlled, multi-center trial, significant reductions were observed in all atherogenic lipoproteins evaluated (including LDL-C, Apo B and Lp(a)) for patients receiving KYNAMRO™ who are already receiving a regimen of maximally tolerated lipid lowering therapies including statins. Three patients (12 percent) treated with KYNAMRO™ withdrew due to adverse events. Consistent with other studies evaluating KYNAMRO™, commonly observed adverse events included mild to moderate injection site reactions and flu-like symptoms, as well as elevations in liver transaminases.

KYNAMRO™ is the registered trade name submitted to health authorities for investigational agent mipomersen.