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.

The strong efficacy and sound safety data coming out of the Phase III programs presented at ACC have all eyes on the commercial opportunity for the PCSK9 inhibitor class. Ongoing outcomes trials and market impact of new cholesterol guidelines sparked discussion, and leading sponsors Amgen and Sanofi/Regeneron talked about the potential populations and differences in the devices and dosing of evolocumab and alirocumab.

Part 2: High costs are built into the rare disease business model. Participants at a roundtable discussion led by Scrip at the J.P. Morgan Healthcare Conference talked about how much they are feeling the pressure on pricing.