Last update: February 2019
*Studies submitted for marketing authorisation
Ten phase III trials from the global phase III programme were submitted for marketing authorisation (Figure 2). Primary efficacy endpoint was mean percent reduction change in LDL-C from baseline to week 24 as compared to placebo or ezetimibe. All studies met their primary efficacy endpoint.
Figure 2: Studies submitted for marketing authorisation1
The 10 studies from the global programme included more than 5000 patients, more than a quarter of patients had heterozygous familial hypercholesterolaemia (HeFH) and 97% of patients had high cardiovascular risk (CV) (Figure 3).
Figure 3. Profile of randomised patients2
- Praluent Summary of Product Characteristics. Available at https://www.medicines.org.uk/emc/medicine/30956. Accessed August 2018.
- European Medicines Agency (2015). European Public Assessment Report (EPAR) Praluent EMA/CHMP/392430/2015. Available at http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/003882/WC500194524.pdf. Accessed August 2018.
- Kastelein et al. ODYSSEY FH I and FH II: 78 week results with alirocumab treatment in 735 patients with heterozygous familial hypercholesterolaemia. Eur Heart J 2015; 36:2996–3003
- Ginsberg HN et al. ODYSSEY HIGH FH: Data presented at the AHA, Chicago, USA November 2014
- Kereiakes et al. Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 inhibitor alirocumab among high cardiovascular risk patients on maximally tolerated statin therapy: The ODYSSEY COMBO I study. Am Heart J. 2015; 169:906–91
- Cannon CP et al. Efficacy and safety of alirocumab in high cardiovascular risk patients with inadequately controlled hypercholesterolaemia on maximally tolerated doses of statins: the ODYSSEY COMBO II randomized controlled trial. Eur Heart J 2015; 36:1186–94.
- Robinson J et al. Efficacy and safety of alirocumab in reducing lipids and cardiovascular events. NEJM 2015; 372:1489–99.
- Moriarty PM et al. Efficacy and safety of alirocumab vs ezetimibe in statin-intolerant patients, with a statin rechallenge arm: The ODYSSEY ALTERNATIVE randomized trial. J Clin Lipidol. 2015; 9:758–69.
- Roth et al. Monotherapy with the PCSK9 inhibitor alirocumab versus ezetimibe in patients with hypercholesterolemia: Results of a 24 week, double-blind, randomized Phase 3 trial. Int J Cardiol. 2014; 176:55–61.
- Bays et al. Alirocumab as Add-On to Atorvastatin Versus Other Lipid Treatment Strategies: ODYSSEY OPTIONS I Randomized Trial. J Clin Endocrinol Metab 2015; 100: 3140–48.
- Farnier et al. Efficacy and safety of adding alirocumab to rosuvastatin versus adding ezetimibe or doubling the rosuvastatin dose in high cardiovascular risk patients: The ODYSSEY OPTIONS II randomized trial. Atherosclerosis. 2016; 244:138–46.