Cholesterol Endpoints Draw Scrutiny At HoFH Panels
The Metabolic and Endocrinologic Advisory Committee accepts LDL lowering as endpoint for homozygous hypercholesterolemia drugs, backing approval of Aegerion’s lomitapide by 13-2 and Genzyme’s mipomersen by 9-6, but hones in on the need for better surrogates for cardiovascular morbidity and mortality.
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FDA advisors reviewing the PCSK9 inhibitors say reliability of LDL-cholesterol-lowering to predict clinical benefit depends on patient population; they backed the Sanofi/Regeneron and Amgen drugs for patients with genetic cholesterol disorders and very high CV risk, but said approval for lower-risk populations should await outcomes data.
Regeneron/Sanofi and Amgen stress the strengths of the Phase III PCSK9 development programs at the American College of Cardiology annual meeting. The mechanism of action holds appeal, but development is taking place at a time of skepticism about surrogate CV markers, following failed trials of other drugs.
Sponsor will institute a registry to monitor patients taking the LDL cholesterol-lowering drug for homozygous familial hypercholesterolemia for 10 years, as part of an approval that is a likely template for Genzyme’s Kynamro.