Praluent - Alirocumab

What is Praluent - Alirocumab used for and what is it used for?

Praluent is a medicine indicated for adult patients with primary hypercholesterolemia (high levels of cholesterol in the blood, in particular "LDL cholesterol" or "bad" cholesterol). Primary means that the disease is generally due to a genetic abnormality. Primary hypercholesterolemia includes heterozygous familial hypercholesterolemia (when the genetic anomaly is inherited from a single parent) and unfamiliar hypercholesterolemia (when the genetic anomaly appears spontaneously unprecedented in the family). Praluent is also used to treat mixed dyslipidemia (abnormal levels of fat in the blood, including high levels of LDL cholesterol).

Praluent should be used together with a low fat diet in the following cases:

in association with a statin (another medicine used for lowering cholesterol) or with a statin and other medicines that reduce cholesterol in patients who do not adequately respond to the maximum tolerated statin dose;
alone or in combination with other cholesterol-lowering medicines in patients who do not tolerate or cannot take statins.

Praluent contains the active substance alirocumab.

How is Praluent - Alirocumab used?

Before starting treatment with Praluent, secondary causes of hypercholesterolemia and abnormal levels of fat in the blood must 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 in a pre-filled pen (75 and 150 mg). The injection is made under the skin of the abdomen, thigh or upper arm.

The usual starting dose is 75 mg every two weeks, but patients who need a greater reduction in blood fat levels can start with a dose of 150 mg every two weeks. The Praluent dose is adequate based on blood fat levels and response to therapy. If the desired response has not been obtained after 4 weeks of treatment, the doctor may increase or decrease the dose.

Patients can self-administer the medicine or the injection can be given by a person who takes care of them after a healthcare professional has given appropriate instructions. For more information, see the package leaflet.

How does Praluent - Alirocumab work?

The active substance in Praluent, alirocumab, is a monoclonal antibody. A monoclonal antibody is an antibody (a type of protein) designed to recognize a specific structure (called an antigen) in the body and bind to it.

Alirocumab was designed to bind to a protein called "PCSK9". This protein binds to cholesterol receptors on the surface of liver cells and causes these receptors to be absorbed and split within the cells. These receptors control blood cholesterol levels, especially LDL cholesterol, removing it from the bloodstream. By binding to PCSK9 and blocking this protein, Praluent prevents the cleavage of receptors within cells and thus increases the number of receptors on the cell surface, where they can bind to LDL cholesterol and remove it from the bloodstream. This helps reduce blood cholesterol levels.

What benefit has Praluent - Alirocumab shown during the studies?

Praluent has been studied in 10 main studies involving over 5, 000 adult patients with hypercholesterolemia (including patients with heterozygous familial hypercholesterolemia) and mixed dyslipidemia. In some studies, Praluent was administered alone while in others it was used in combination with other cholesterol-lowering medicines, even in patients taking the maximum recommended statin doses. In some studies, Praluent was compared with placebo (a dummy treatment) while in others it was compared to another medicine (ezetimibe). From these studies it was found that, when administered in addition to a statin, Praluent induced a substantial reduction in blood levels of LDL cholesterol (39-62% higher than placebo) after 6 months of treatment. Furthermore, when administered as an adjunct to standard or placebo therapy, Praluent induced a reduction in blood LDL cholesterol levels 24-36% higher than in ezetimibe

What is the risk associated with Praluent - Alirocumab?

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

Why has Praluent - Alirocumab been 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 noted that in all studies conducted on patients with primary hypercholesterolemia and mixed dyslipidemia, including patients taking the maximum recommended statin doses or who were intolerant to them, Praluent showed a significant reduction in LDL cholesterol levels, which is a known risk factor for cardiovascular diseases (affecting the heart and blood vessels). It is not yet certain, however, if Praluent can determine a reduction in cardiovascular pathologies, since data on long-term cardiovascular outcomes are still being processed. Therefore, the use of Praluent has been approved in subjects who do not respond adequately to the maximum tolerated dose of statins or who cannot be subjected to a statin-based treatment. As for safety, the committee noted that the safety profile of the medicine is acceptable.

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

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.