Genzyme and Isis file Kynamro NDA in USA

Biotech firm Genzyme, a subsidiary of French drug major Sanofi (Euronext: SAN), has submitted a New Drug Application to the US Food and Drug Administration seeking approval for Kynamro (mipomersen sodium) for the treatment of patients with homozygous familial hypercholesterolemia (HoFH), a rare cholesterol disorder.

The drug is partnered with US firm Isis Pharmaceuticals (Nasdaq: ISIS), which 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.

“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, chief of the sections of Cardiology and Cardiovascular Research, and Professor of Medicine and Genetics, Baylor College of Medicine, USA. “Currently available therapies work on clearance whereas Kynamro uniquely targets Apo B production and production of all Apo B-containing, atherogenic lipoproteins, including VLDL, LDL, and Lp(a). Current options reserved for these patients are mechanical procedures such as LDL-aphereis or liver transplant,” Dr Ballantyne added.