One year after FDA approval, are PCSK9 inhibitors living up to expectations?

It’s been about 1 year since the two currently available PCSK9 inhibitors, evolocumab and alirocumab, received US FDA approval. (Bococizumab is still completing phase 3 studies.) Uptake, as most in the healthcare community know, has been slow, largely due to the cost associated with the two therapies.

The US FDA recently approved a monthly single-dose administration option for evolocumab. The system consists of a hands-free device which is designed to provide 420 mg of evolocumab in a single dose and allows patients the freedom of being able to continue with moderate physical activity such as walking, reaching, and bending. The cost is expected to remain similar to twice weekly dosing, and given how new it is, it remains to be seen what kind of impact on clinical practice it will have.

Researchers have recently claimed that the price for PCSK9 inhibitors would have to fall by more than two-thirds to be cost effective. What this means, according to the study, is that to be cost-effective at $100,000 per QALY, the annual costs of the drugs would need to decrease from $14,350 to $4536 per patient, or less. The researchers, however, did acknowledge their study did not address whether one should prescribe these medications, and evidence does suggest they are highly effective at lowering LDL-C. Additionally, the cost of the drugs in many countries in Europe, where they were introduced at the same time as the United States, is much closer to the cost-effective price identified in the study. Dr. Jennifer Robinson, who recently coauthored an article on nonstatin therapies and PCSK9 inhibitors, explained there are very high-risk patients whose LDL-C remains high who could benefit from PCSK9 inhibitors, namely patients with familial hypercholesterolemia (FH) who have clinical cardiovascular disease. She pointed out that cost-effectiveness analyses for other genetic diseases that could be essentially cured are not done.

Expert analysis on the latest data, perspective on the use of PCSK9 inhibitors in clinical practice, and what the future likely holds in store for them can be heard at the 11th Annual Cardiometabolic Health Congress, being held October 5-8, 2016 at the Sheraton Boston. Some highlights include the symposium “The New Face of Lipid Management for Patients with Hypercholesterolemia and Statin Intolerance,” chaired by Dr. Steven E. Nissen and joined by faculty Michael H. Davidson, MD and Robert H. Eckel, MD; the General Sessions presentation “Lipid-Modifying Therapies 2016: Opportunities and Challenges” by CMHC Chair Christie M. Ballantyne, MD; and the symposium “Patient-focused LDL-C Management and Risk Reduction in Clinical Practice: The Utility of PCSK9 Inhibitors,” also chaired by Dr. Ballantyne and joined by Henry A. Ginsberg, MD, Michael J. Koren, MD and James A. Underberg, MD.

Of further interest, it’s been announced that new data will be presented at the European Society of Cardiology Congress 2016, being held in Rome August 27-31. The 11 abstracts will evaluate evolocumab for the treatment of patients with high cholesterol across ESC/European Atherosclerosis Society cardiovascular risk subgroups, as well as the long-term efficacy and safety of evolocumab in patients with heterozygous FH. Eagerly awaited CV outcomes data of course are not available yet, but results from FOURIER are expected to be announced early next year. Results from ODYSSEY OUTCOMES and SPIRE will most likely not be known until later in 2018.

Share onShare on FacebookShare on Google+Tweet about this on TwitterShare on LinkedIn