A U.S. Food and Drug Administration panel of outside experts recommend the Sanofi SA and Isis Pharmaceutical Inc. drug, Kynamro, for treatment of a rare genetic disorder that causes unusually high cholesterol and life-threatening cardiovascular disease.
In a 9-6 vote, the FDA advisory committee concludes that company-provided research showed the 200mg injection to be safe and effective enough for the one in a million Americans who have homozygous familial hypercholesterolemia, or HoFH, and are already on a cholesterol treatment regimen that includes lipid-lowering medication.
The panel's recommendation, reached despite misgivings about side-effects, including liver problems and a potentially higher risk for cancer, will now be considered by FDA regulators as they decide by the end of January whether to grant final approval for the drug known generically as mipomersen sodium.
The recommendation came in the same week as FDA advisers recommended approval for Aegerion Pharmaceuticals Inc's HoFH drug, lomitapide, with a decision by the regulatory agency expected before the end of December.
The FDA would impose risk mitigation strategies to control the use of both drugs because of safety concerns, including a buildup of fatty tissues in the liver, a condition that can lead to serious liver damage and cardiovascular disease over time.
FDA officials said they would seek to ensure maximum benefits for HoFH patients, whose clogged arteries pose a lingering danger of heart attack and early death.
Sanofi's Genzyme unit said it would also create a registry to monitor patients and collect data on the drug's effects.
Excerpted from Reuters. Click here for the full article.