Genzyme and Isis have announced the FDA approval of Kynamro (mipomersen sodium) injection indicated for homozygous familial hypercholesterolemia (HoFH).
The regulatory agency approved Kynamro 200mg subcutaneous injection per week as an added therapy to lipid-lowering medications and diet in HoFH patients.
Kynamro will reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B, total cholesterol, and non-high density lipoprotein-cholesterol in HoFH patients.
Genzyme president and CEO David Meeker said, "Today's FDA approval of KYNAMRO is great news for patients with HoFH who are in need of additional treatment options for this rare, and often under-diagnosed disease."
With FDA approval of the antisense drug, Isis will receive a $25m milestone payment from Genzyme.
Isis CEO and board chairman Stanley Crooke said, "As evidenced by our robust pipeline, our antisense drug discovery technology is applicable to many different diseases, including the treatment of a chronic and rare disease, like HoFH."
The approval is based on randomized, double-blind trial conducted in 51 HoFH patients, which established the efficacy of Kynamro in reducing LDL-C levels and all measured endpoints for atherogenic particles.
Safety data of the drug candidate was based on collective results from four Phase 3, randomized, placebo-controlled trials, according to the company.
