The FDA has accepted for filing the NDA by Genzyme, a Sanofi company, and Isis Pharmaceuticals for Kynamro 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,” said David Meeker, M.D., president and chief executive officer, 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 the NDA in March 2012. The application will be subject to a standard review and will have a PDUFA date of January 29, 2013. In July 2011, Genzyme submitted an application for EU marketing approval of Kynamro 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 U.S. with HoFH who are unable to adequately control their LDL-C with currently available treatments,” said B. Lynne Parshall, chief operating officer and chief financial officer 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.”
Kynamro's NDA is supported by the largest clinical trial conducted to date in the HoFH patient population, according to Genzyme. 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.