PharmD Ana Parvulescu bring the latest news on the treatment of hypercholesterolemia:
The European Medicines Agency (EMA) has recommended authorising Repatha (evolocumab) as treatment to lower high levels of cholesterol in the blood of people who are unable to control their cholesterol despite taking optimal doses of statins or who cannot take statins. It should be used in addition to a healthy diet. Other lipid-lowering therapies (statins and others) should also be used if tolerated. Repatha is also indicated to treat people with homozygous familial hypercholesterolaemia, a rare inherited disorder in which levels of LDL-cholesterol (‘bad cholesterol’) are higher than normal from birth. (Most (60-75%) of the cases of familial hypercholesterolemia are caused by a mutatoion of LDLR gene that is dominant autozomal transmited. There is one case of its heterzygous type in 500 persons and one case of its homozygous type in 1000000 persons in the whole wide world.) It is intended for injection under the skin either once every two weeks, or once a month.
High levels of cholesterol in the blood are common risk factors for heart disease, which is the leading cause of death globally.
Repatha is the first monoclonal antibody (a type of protein) in this therapeutic area and provides a new treatment option for patients who are unable to control their high cholesterol despite taking currently available therapies. Repatha blocks the PCSK9 protein, which would otherwise lower the number of LDL-receptors in the liver and through this, diminishes its ability to remove LDL-cholesterol from the blood.
The efficacy of Repatha as a lipid-lowering agent was assessed in nine trials (about 5,500 people) in patients with hypercholesterolaemia and mixed dyslipidaemia, and in two studies (about 250 people) in patients with homozygous familial hypercholesterolaemia. Repatha reduced LDL-cholesterol for both patient groups. Available evidence does not yet allow the longer term benefits of Repatha for patients in reducing heart disease or death from heart disease to be determined.
The full indication for Repatha approved by the CHMP is as follows:
- Repatha is indicated in adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia, as an adjunct to diet:
- in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin; or
- alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contra-indicated;
- Repatha is indicated in adults and adolescents aged 12 years and over with homozygous familial hypercholesterolaemia in combination with other lipid-lowering therapies;
- The effect of Repatha on cardiovascular morbidity and mortality has not yet been determined.
Once a marketing authorisation has been granted, each Member State will take a decision on price and reimbursement based on the potential role/use of this medicine in the context of its national health system.
On the other hand, FDA announces that the Endocrinologic and Metabolic Drugs Advisory Committee will meet to discuss the safety and efficacy of REPATHA (Evolocumab) for injection, submitted by Amgen Inc., as adjunct to diet to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol (non-HDL-C), TC/HDL-C, ApoB/ApoA1, very low-density lipoprotein cholesterol, triglyceride, and lipoprotein A, and to increase HDL-C and ApoA1, in adults with hyperlipidemia or mixed dyslipidemia, either in combination with a statin or statin with other lipid-lowering therapies (e.g., ezetimibe), or alone, or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or alone or in combination with other lipid-lowering therapies in patients for whom a statin is not considered clinically appropriate. In addition, the committee will discuss the safety and efficacy of evolocumab to reduce LDL-C, TC, ApoB, and non-HDL-C, in combination with other lipid-lowering therapies (e.g. statins, LDL apheresis) in patients at least 12 years of age with homozygous familial hypercholesterolemia.
Also, the Endocrinologic and Metabolic Drugs Advisory Committee will meet to discuss the safety and efficacy of PRALUENT (Alirocumab) for injection, submitted by Sanofi Aventis, U.S., as an adjunct to diet, for long-term treatment of adult patients with primary hypercholesterolemia (non-familial and heterozygous familial) or mixed dyslipidemia including patients with type 2 diabetes mellitus, to reduce low-density lipoprotein cholesterol, total cholesterol, non-high-density lipoprotein cholesterol, apolipoprotein B, tryglyceride, and lipoprotein A, and to increase high-density lipoprotein cholesterol and apolipoprotein A-1 either in combination with a statin or as monotherapy including in patients who cannot tolerate statins.
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