Sanofi and partner Isis Pharmaceuticals have filed Kynamro for a rare genetic form of high cholesterol with regulators in the USA.
The French drugmaker's Genzyme unit and Isis have submitted a New Drug Application to the US Food and Drug Administration seeking approval for Kynamro (mipomersen) for the treatment of patients with homozygous familial hypercholesterolaemia (HoFH). The first-in-class apo-B synthesis inhibitor was filed in Europe in July last year.
The FDA submission is supported by "the largest clinical trial conducted to date in the HoFH patient population", Genzyme says, noting that significant reductions were observed in all atherogenic lipoproteins evaluated for patients receiving Kynamro who are already receiving lipid-lowering therapies including statins.
Isis will receive a $25 million milestone payment following FDA acceptance of the NDA submission. The agency has granted mipomersen orphan drug designation.