Advisors to the US Food and Drug Administration have recommended approval of Sanofi and Isis Pharmaceuticals' Kynamro for the treatment of a rare genetic form of high cholesterol.

The FDA's Endocrinologic and Metabolic Drugs Advisory Committee has voted 9 to 6 that Sanofi unit Genzyme had provided sufficient efficacy and safety data to support Kynamro (mipomersen sodium) for homozygous familial hypercholesterolaemia (HoFH). The recommendation comes after the panel reviewed data from a Phase III study in HoFH patients, three other late-stage trials in other high-risk hypercholesterolaemia populations and an ongoing long-term extension study.

Genzyme chief executive David Meeker said the committee meeting "marks a significant and positive step in our efforts to bring this important new therapy to patients and families affected by this often unrecognised genetic disorder". He added that "there is still a great need for the HoFH patients, who have exhausted conventional medications and still have low-density lipoprotein cholesterol levels two-four times above normal".

The panel had expressed concerns about side-effects, including liver problems and a potentially higher risk for cancer, but the committee members have taken into account the lack of treatments for HoFH patients. Earlier this week, the panel also recommended approval of Aegerion Pharmaceuticals' HoFH drug lomitapide, voting 13-2 in favour.