Wary Of Surrogates, Payers Seek Outcomes Data For PCSK9 Class
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.
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FDA asked its advisory committee not to discuss accelerated approval for Sanofi/Regeneron's Praluent and Amgen's Repatha; panelists wanted to discuss the idea anyway, however, given uncertainty around the reliability of LDL-cholesterol as a surrogate.
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.
Alirocumab was associated with lower rate of major cardiovascular events compared to placebo in post-hoc, interim Phase III analysis – boosting chances for FDA approval in 2015. The firm is taking no chances on review period, purchasing a priority review voucher from BioMarin.