Amgen PCSK9 drug gets FDA staff rec, but panelists express concerns
- An advisory panel to the FDA on Wednesday recommended that the agency approve Amgen's Repatha (evolocumab), part of a new class of potential blockbuster PCSK9 cholesterol-fighting drugs.
- The panel recommended Sanofi/Regeneron's competing Praluent (alirocumab) for approval on Tuesday.
- The advisory committee's vote was 11-4 in favor of recommending Repatha's approval in high-risk cardiovascular disease patients (other than homozygous familial hypercholesterolemia [HoFH] patients) whose cholesterol cannot be maintained with statins alone, and 15-0 in favor of approving it for HoFH. Several panel members expressed concerns about the Amgen drug's dosing and raised the specter of doctors reducing their patients' statin intake, while others said that longer and more detailed studies should be conducted on the drug to determine actual CVD outcomes such as reductions in heart attacks.
As BioPharma Dive has previously reported, it seems that Praluent's more versatile dosing regimen may give it a marketing and regulatory advantage. Repatha is available in biweekly 140 mg injections or monthly 420 mg injections, whereas Praluent has 75 mg and 150 mg biweekly injections. That also gives Sanofi a pricing advantage by allowing the firm to set two distinct price points at more affordable levels.
Several advisory committee members also raised concerns that Repatha may lower bad LDL cholesterol too much. While there is no compelling data showing that excessively low LDL-C levels are bad, per se, there is also a lack of safety data on particularly low levels of that cholesterol. More than one panelist said that longer-term and larger-population data will be necessary to judge the true efficacy of Repatha and PCSK9 medications.
Both Praluent and Repatha have been shown to lower LDL-C by as much as 60%. The question is: Which population of patients would this benefit? And is simply lowering this type of cholesterol even an effective therapeutic regimen?
Amgen has already enrolled a far larger and longer-term ongoing CVD outcomes trial to guage whether the drug can, in fact, deter adverse events. Results from that study won't be released until 2017.