Physicians' Academy for Cardiovascular Education

Patients with heterozygous familial hypercholesterolemia benefit from PCSK9-antibody

Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 monoclonal antibody alirocumab vs placebo with heterozygous familial hypercholesterolemia

Literature - Kastelein JJP, Hovingh GH, Langslet G et al. - J Clin Lipidol, 2017, DOI: http://dx.doi.org/10.1016/j.jacl.2016.12.004

Background

Only approximately 20% of patients with heterozygous familial hypercholesterolemia (HeFH) reach their LDL-C goal of ≤100 mg/dL when treated with statins, ezetimibe, and/or bile acid sequestrants [1,2]. On the other hand, the PCSK9 inhibitor alirocumab resulted in significant LDL-C reductions, both as monotherapy and in combination with statin therapy, in a variety of patient populations in the phase III ODYSSEY program [3-5].

In this analysis of four 78-week placebo-controlled ODYSSEY studies (FH I, FH II, long term and high FH), the LDL-C-lowering efficacy and safety of alirocumab Q2W were evaluated in 1257 HeFH patients on maximally tolerated dose of statins and other lipid-lowering therapies. Patients of the FH I and FH II trials received 75 mg Q2W or 150 mg Q2W and at week 12, some patients increased their dose from 75 to 150 mg Q2W if LDL-c was still ≥70 mg/dL at week 8. Patients of the long term and high FH received 150 mg Q2W from the start.

Main results

Conclusion

In four studies with HeFH patients, alirocumab resulted in significant LDL-C-lowering in most patients who were receiving the maximally tolerated dose of a statin, with or without other lipid-lowering therapies.

References

Show references

Find this article online at J Clin Lipidol