Amgen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for an extension to the marketing authorization of a new 420 mg single-dose delivery option for Repatha (evolocumab). The new automated mini doser (AMD) with prefilled cartridge is a hands-free device designed to provide 420 mg of Repatha in a single injection per administration. Repatha is a human monoclonal antibody that blocks a protein called proprotein convertase subtilisin/kexin type 9 (PCSK9), which inhibits the body's natural system for eliminating "bad" cholesterol (low-density lipoprotein cholesterol or LDL-C) from the blood.
If approved by the European Commission (EC), Repatha would be the first PCSK9 inhibitor in Europe with the option of a single monthly injection. Repatha was the first PCSK9 inhibitor to gain marketing authorization in Europe as an every-two-week or monthly dosing regimen on July 17, 2015. Repatha AMD will be available in Europe during 2017 depending on reimbursement requirements.
"We are pleased to advance our new dosing administration option and look forward to continuing our work with regulators to bring it to patients in Europe," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "The approval of this delivery system will provide an alternative for patients who need the additional LDL lowering that Repatha can provide and are seeking less frequent administration."
In Europe, Repatha is approved as an adjunct to diet for the treatment of high cholesterol in combination with statins or other lipid-lowering therapies in patients unable to control their LDL-C with maximum tolerated statin doses, or alone or in combination with other lipid-lowering therapies in patients who are statin intolerant or for whom a statin is contraindicated. Repatha is also approved in combination with other lipid-lowering agents in patients with homozygous familial hypercholesterolemia (age 12 and over). The effect of Repatha on cardiovascular morbidity and mortality has not yet been determined. Top-line results from the approximately 27,500-patient event-driven FOURIER study are anticipated in first quarter of 2017.
The CHMP positive opinion will now be reviewed by the EC and if granted, the marketing authorization will be extended to include the 420 mg single injection in the 28 member countries of the European Union (EU), as well as Iceland, Lichtenstein and Norway.
Today's CHMP positive opinion follows the US Food and Drug Administration's approval of the single 420 mg monthly injection option on July 11, 2016, as the Repatha Pushtronex system (on-body infusor with prefilled cartridge).
More than 60 per cent of high-risk patients in Europe are still unable to adequately lower their LDL-C levels with statins or other currently approved lipid-lowering agents. Among very high-risk patients, the percentage is increased to more than 80 per cent. The health care cost of cardiovascular disease (CVD) in the EU is approximately €106 billion per year.
Repatha (evolocumab) is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9). Repatha binds to PCSK9 and inhibits circulating PCSK9 from binding to the low-density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, Repatha increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.
The FOURIER outcomes trial is designed to evaluate whether treatment with Repatha in combination with statin therapy, compared to placebo plus statin therapy, reduces the risk of cardiovascular events in patients with high cholesterol and clinically evident cardiovascular disease, and completed patient enrollment in June 2015. Top-line results from the approximately 27,500-patient event-driven FOURIER study are anticipated in first quarter of 2017.
Repatha is approved in more than 40 countries, including the US, Japan, Canada and in all 28 countries that are members of the EU. Applications in other countries are pending.