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EUROPEAN MEDICINES AGENCY

SCIENCE MEDICINES HEALTH

EMA/504805/2015

EMEA/H/C/003882

EPAR summary for the public

Praluent

alirocumab

This is a summary of the European public assessment report (EPAR) for Praluent. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Praluent.

For practical information about using Praluent, patients should read the package leaflet or contact their doctor or pharmacist.

What is Praluent and what is it used for?

Praluent is a medicine used to treat adults with primary hypercholesterolaemia (high blood cholesterol levels, in particular of the type 'LDL-cholesterol' or 'bad' cholesterol). Primary means that the disease is generally the result of a genetic abnormality. Primary hypercholesterolaemia includes heterozygous familial (when the genetic abnormality is inherited from only one parent) and non-familial disease (when the genetic abnormality arises spontaneously without a family history).

Praluent is also used to treat mixed dyslipidaemia (abnormal levels of fats in blood, including high levels of LDL-cholesterol).

Praluent is to be used together with a low-fat diet, in the following ways:

•    in combination with a statin (other cholesterol-lowering medicines), or a statin plus other fatlowering medicines, in patients who do not adequately respond to the maximum tolerated dose of the statin;

•    alone or in combination with other fat-lowering medicines in patients who cannot tolerate or cannot be given statins.

Praluent contains the active substance alirocumab.

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How is Praluent used?

Before starting treatment with Praluent, other causes of excess cholesterol and abnormal fat levels in the blood should be excluded. The medicine can only be obtained with a prescription.

Praluent is available as a solution for injection in a pre-filled syringe or pre-filled pen (75 and 150 mg). The injection is given under the skin of the abdomen, thigh or upper arm.

The usual starting dose is 75 mg every two weeks but patients requiring bigger reductions of blood fat levels may start with 150 mg every two weeks. The dose of Praluent is adjusted based on the levels of fats in blood and response to the medicine. If the desired response is not achieved after 4 weeks of treatment, the doctor can increase or decrease the dose.

Patients or their carers can inject the medicine once they have been properly trained by a healthcare professional. For more information, see the package leaflet.

How does Praluent work?

The active substance in Praluent, alirocumab, is a monoclonal antibody. A monoclonal antibody is an antibody (a type of protein) that has been designed to recognise and attach to a specific structure (called an antigen) in the body.

Alirocumab has been designed to attach to a protein called 'PCSK9'. This protein attaches to cholesterol receptors on the surface of liver cells and causes these receptors to be absorbed and broken down inside the cells. These receptors control blood levels of cholesterol, especially LDL-cholesterol, by removing it from the bloodstream. By attaching and blocking PCSK9, Praluent prevents the receptors from being broken down inside cells and therefore increases the number of these receptors on the cell surface, where they can attach to LDL-cholesterol and remove it from the bloodstream. This helps to reduce the amount of cholesterol in the blood.

What benefits of Praluent have been shown in studies?

Praluent has been studied in 10 main studies involving over 5,000 adult patients with hypercholesterolaemia (including patients with heterozygous familial disease) and mixed dyslipidaemia. Some studies looked at Praluent taken on its own, while others studied Praluent in combination with other fat-lowering medicines, including patients on the maximum recommended doses of statins. Some studies compared Praluent with placebo (a dummy treatment) and others to another medicine for hypercholesterolaemia (ezetimibe). These studies showed that when Praluent was given on top of a statin it led to a substantial reduction in blood levels of LDL-cholesterol (between 39 and 62% more than placebo) after 6 months of treatment. When given on top of standard treatment or on top of placebo, Praluent produced a 24 to 36% greater reduction in blood levels of LDL-cholesterol than ezetimibe.

What are the risks associated with Praluent?

The most common side effects with Praluent (which may affect up to 1 in 10 people) are injection site reactions such as pain and redness, problems affecting the nose and throat such as colds, and itching. For the full list of side effects and restrictions, see the package leaflet.

Why is Praluent approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Praluent's benefits are greater than its risks and recommended that it be approved for use in the EU. The Committee

Praluent

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noted that across all studies in patients with primary hypercholesterolaemia and mixed dyslipidaemia, including patients on maximum recommended doses of statins or those intolerant to them, Praluent showed an important reduction in LDL-cholesterol levels, which is a known risk factor for cardiovascular (affecting the heart and blood vessels) disease. It is not yet known, however, whether Praluent will reduce cardiovascular disease, as data on long-term cardiovascular outcomes are still pending. Therefore, Praluent has been approved for use in patients who do not adequately respond to the maximum tolerated dose of statins or who cannot be given statins. With regard to safety, the Committee noted an acceptable safety profile.

What measures are being taken to ensure the safe and effective use of Praluent?

A risk management plan has been developed to ensure that Praluent is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Praluent, including the appropriate precautions to be followed by healthcare professionals and patients.

Further information can be found in the summary of the risk management plan.

Other information about Praluent

The European Commission granted a marketing authorisation valid throughout the European Union for Praluent on 23 September 2015.

The full EPAR and risk management plan summary for Praluent can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/European public assessment reports. For more information about treatment with Praluent, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

This summary was last updated in 09-2015.

Praluent

EMA/504805/2015