A novel class of cholesterol-lowering drug could be available to Irish patients by the end of the year, following the approval of the first of the new agents by the European Medicines Agency (EMA).
The EMA has recommended to the European Commission that it issue a EU-wide marketing authorisation for evolocumab as a treatment to lower high blood levels of cholesterol in cases where the standard treatment with statin drugs is not effective.
The new drug, which will be marketed under the trade name Repatha and is manufactured by Amgen, is also indicated for people who cannot take statins and for those with a rare inherited form of familial high cholesterol in which levels of LDL cholesterol (“bad cholesterol”) are higher than normal from birth.
Unlike statins, which are taken orally, the new class of monoclonal antibodies are injected either monthly or once every two weeks.
Evolocumab, and other drugs in the class, block a protein called PCSK9, the effect of which is to increase the number of LDL- receptors in the liver, thereby enhancing the body’s ability to remove the harmful form of cholesterol from the blood.
LDL cholesterol causes “furring” of the arteries that supply blood to the heart and the brain, which puts those affected at greater risk of heart attack and stroke.
Evolocumab and another agent alirocumab received preliminary approval from the US Food and Drug Administration (FDA) last week even though research data proving their efficacy in reducing cardiovascular disease is not yet complete. This fact led a minority of experts on the FDA panel to vote against approval.
However other experts say that trial results showing a 40 to 65 per cent reduction in LDL levels among participants is highly significant.
Dr Jim Crowley, medical director of CROí, the west of Ireland cardiology foundation, said it is reasonable to use LDL reduction as a surrogate for reducing cardiac events based on current knowledge.
Both Dr Crowley and Dr Angie Brown, medical director of the Irish Heart Foundation, emphasised a need to establish the long -term safety profile of the new class of agents.
“It is very important to see the results of long term outcome trials before these agents are used more widely,” Dr Brown said. She predicted the new agents are likely to complement statins in the prevention of heart disease rather than replace them.
While the exact cost of the drug in Europe is not yet known, US sources have predicted a cost per patient per year of around $10,000.
Once marketing authorisation has been granted, the HSE medicines management programme will take a decision on reimbursement based on the potential role of the monoclonal agent and its cost effectiveness.
