Familial Hypercholesterolaemia Sanofi Booklet
Inherited Cholesterol Guide

Download our easy to read pictorial guide to Familial Hypercholesterolemia. Find out more about inherited high cholesterol.  

Reducing cholesterol

Making changes to the food you eat and being more active can help lower your cholesterol to normal levels.

In some cases, particularly if you are older or at greater risk, you may also need to take a cholesterol-lowering medicine like a statin. Statins are very effective, safe and well tolerated and have been shown to reduce heart attacks.

The basics of cholesterol

Find out more about the basics of cholesterol in our health and high cholesterol section.

Cholesterol level concerns?
Cholesterol level concerns?

Concerned about your cholesterol levels?  Is there is a history of heart disease in your family? Contact us or download our cholesterol fact sheets.

Heart of the Family - combined

Watch this film to hear GP, Dr Sarah Jarvis, and patients, talk about inherited high cholesterol

What’s your heart age?

Heart Age is a sign of how healthy your heart is. This clever calculator works it out from a few simple questions.


What is a PCSK9 inhibitor?

"PCSK9 inhibitor" is a general term used to describe a new class of cholesterol lowering treatments. These drugs help the liver to lower cholesterol levels in the blood. 

How do they work?

Several PCSK9 inhibitors are being developed and whilst they don’t all work in the same way they are based on the same principals. These drugs all act on the PCSK9 protein, which is produced by liver cells.  

They have been developed as a result of studying people that are genetically programmed to have lots of the PCSK9 protein and some that have very little. When the protein is more abundant and active it leads to life-long high cholesterol and early heart disease.  However when there is very little PCSK9 protein this leads to life-long low cholesterol and big reductions in cardiovascular disease.

Why?  Well because the PCSK9 protein is responsible for breaking down cholesterol receptors which sit on the surface of healthy liver cells. Normally these receptors catch hold of LDL-cholesterol particles in the blood and help to remove them. The fewer receptors there are, the less able the liver is to keep our blood cholesterol low.
So PCSK9 inhibitors target the PCSK9 protein making it less effective at breaking down LDL receptors. The result is more working receptors on the surface of liver cells, and more cholesterol can be removed from the blood.
The first PCSK9 inhibitors that are being developed are a kind of drug called a monoclonal antibody or MAB.  All MABs are made in a laboratory under strict controls.  They can recognise and attach to specific proteins produced by cells in our bodies. Each monoclonal antibody recognises just one particular protein. They work in different ways depending on which protein they target. 

Who might benefit from taking a PCSK9 inhibitor?

Doctors who look after people with blood fat conditions believe that these new drugs could be very helpful for certain groups of people with high levels of cholesterol in their blood who are at high risk of early and avoidable heart disease.  These include:        

  • People with familial hypercholesterolaemia (FH) – an inherited problem which results in very high levels of cholesterol in the blood 
  • People who cannot tolerate other cholesterol lowering treatments like statins
  • People who already have heart disease but cannot lower their LDL-cholesterol enough despite being on maximum therapy

How often do people need to take them? 

Monoclonal antibodies (MABs) cannot be taken orally as tablets, capsules or liquids. They have to be injected directly under the skin. But the good news is that PCSK9 inhibitors only need to be taken once every 2 to 4 weeks dependent upon which drug is used and its strength. And one recent study suggested that once every 8 weeks might be enough. Doctors and nurses will be able to train most of the people that need PCSK9 inhibitors so that they can give their own injections all by themselves. So most people won’t need to attend a hospital or GP surgery to receive the treatment.  

Are there any side effects  

All drugs have side effects but the good news is that PCSK9 inhibitors appear to be well tolerated. We know this from the drug trials that have been carried out. People in the two groups that took the real drug and the dummy drug reported similar levels of side effects. The main side effect that doctors are aware of from these studies is a mild soreness or itchiness at the site of injections. So far the research suggests that these drugs can be used to control life-long high cholesterol conditions such as Familial Hypercholesterolaemia (FH) 

How effective are they?

PCSK9 Inhibitors seem to be very effective. Clinical studies have shown that they can cut LDL cholesterol levels by more than half in a wide variety of patient types. They are also effective alongside other cholesterol lowering treatments such as statins. Early data also suggests that there are fewer heart attacks and strokes amongst people that have taken these drugs compared to people on a placebo drug.  

Are these drugs available yet?

You can recognise the names of all monoclonal antibodies (MABs) because they end in the letters “mab”.   There are two PCSK9 inhibitors which are already licenced for use in the UK.  These are Evolocumab and Alirocumab.  

Previously these drugs were only available for patients on clinical trials and for private prescriptions. On May 5th 2016, Following HEART UK’s #Yes2PCSK9 campaign, the National Institute for Health and Care Excellence (NICE) published a final draft guidance recommending Evolocumab and Alirocumab for some people who have conditions that put them at extremely high risk of heart attacks or strokes.

The draft guidance recommends Alirocumab (Praluent, Sanofi) and Evolocumab (Repatha, Amgen) for adults with primary hypercholesterolaemia/mixed dyslipidaemia and those with heterozygous familial hypercholesterolaemia (HeFH) ​to help reduce their risk of cardiovascular disease.

Furthermore the drugs are recommended for people whose cholesterol levels are not controlled adequately using other drugs such as statins, or who can’t tolerate statins because of their side effects or have another condition which means they can’t take them.

Until final guidance is issued, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations across the country.

Find out more here