October 18th, 2012
FDA Reviewers Recommend Approval of Lomitapide for Homozygous Familial Hypercholesterolemia
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 13-2 on Wednesday to recommend approval of Aegerion Pharmaceuticals’ cholesterol-lowering drug lomitapide for use in patients with homozygous familial hypercholesterolemia (FH).
The lopsided vote does not completely reflect the views of many of the panel members, who expressed considerable concern that the drug might be used in lower-risk populations, in particular, patients with heterozygous FH. The committee also expressed concern about the use of lomitapide in children with homozygous FH, since they were not included in clinical trials, but might be considered candidates for therapy in clinical practice.
Panel members appeared to largely agree with one panel member, who explained that his yes vote was “specific for this condition” (homozygous FH) only. He said he could accept the “trade-off between a near-certain early demise versus the possibility of liver disease.”
Aegerion estimates that there are about 6000 homozygous FH patients in the U.S. and Europe.
The committee expressed support for the FDA’s proposed Risk Evaluation and Mitigation Strategy (REMS) that would limit access to the drug to medically appropriate patients and to provide education to prescribers about how to use lomitapide, how to prevent liver damage, and how to monitor patients during treatment. The REMS would require special certification for health care professionals and pharmacies that prescribe and dispense the drug.
On Thursday, the same committee will meet to discuss a similar indication for Genzyme’s mipomersen injection. Following the release of a highly critical FDA review, many observers believe the mipomersen panel will be much more contentious.