Genzyme, a Sanofi company (SNY), and Isis Pharmaceuticals Inc. (ISIS) announced that Genzyme has submitted a New Drug Application or NDA to the U.S. Food and Drug Administration or FDA seeking approval for Kynamro (mipomersen sodium) for the treatment of patients with homozygous familial hypercholesterolemia or HoFH.
"HoFH patients have aggressive, life-threatening cardiovascular disease starting at birth because of genetic mutations which severely impair LDL clearance and also trigger an overproduction of all atherogenic lipoproteins," said Christie Ballantyne, M.D., Chief of the Sections of Cardiology and Cardiovascular Research, and Professor of Medicine and Genetics, Baylor College of Medicine.
Genzyme stated that Isis will receive a $25 million milestone payment from Genzyme following FDA acceptance of the NDA submission. Provided the necessary approvals are granted, mipomersen would be marketed under the brand name Kynamro, the name that has been submitted to health authorities for the investigational agent.
The FDA has granted mipomersen Orphan Drug designation for the treatment of patients with HoFH.
Genzyme noted that 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 for patients receiving Kynamro who are already receiving a regimen of maximally tolerated lipid-lowering therapies including statins.
The company stated that 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.
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