Eliglustat (Cerdelga®)

What is Cerdelga® and what does it do?

Cerdelga® is an oral capsule for the long-term treatment of adult patients with Gaucher disease type 1. It is approved for use by the European Medicines Agency and available from the NHS.

Cerdelga® can be used by most type 1 Gaucher patients. However, if your specialist thinks this is the right treatment for you, there are a few additional important factors to consider, such as, for example, the need for a specific genetic test (CYP2D6 enzyme status), what other medication you are taking, or whether you are pregnant. Diet is another consideration. There is more information on the ‘CYP2D6’ status below.

Cerdelga® is the brand name for the drug eliglustat tartrate.

How do I take my Cerdelga® capsules?

Cerdelga® is a capsule that needs to be taken regularly once or twice per day as recommended by your specialist. It comes in blister packs of 14 hard capsules.

It does not matter whether you take it with or without food. Do follow the instructions in the capsule box and take the capsule regularly as prescribed. If you miss a dose, just continue again at the next scheduled time. Do not take an extra capsule.

Do I have to follow any special diet?

Unlike previous tablet treatments for Gaucher disease, Cerdelga® does not require you to follow any special diet. However you have to avoid grapefruit and grapefruit juice as this can affect the liver enzymes that break down Cerdelga®.

Are there any side effects of Cerdelga®?

The side effects described are based on clinical trials, and therefore generalised symptoms may not always be directly related to Cerdelga®. The majority of side effects reported in the Cerdelga® clinical trials were mild and short-lived.

The side effects commonly reported are:

• Tiredness
• Dry skin
• Headache
• Gastro-intestinal upset
• Joint pain
• Occasional fainting

How will life be different on Cerdelga®?

Cerdelga® is a first line treatment for Gaucher disease and therefore you are unlikely to need to continue treatment with enzyme replacement therapy, if that is what you have been receiving up to now.

You will still receive medication deliveries from your homecare company but this is likely to be only once every eight weeks. The drug needs to be stored safely in a cupboard at room temperature – no need to store the drug in the fridge. You will still need to attend your specialist centre at least once or twice a year as before, to ensure the treatment is effective for you. You may be advised to carry an alert card notifying healthcare professionals of your Cerdelga® therapy. You will be able to take Cerdelga® away with you when travelling.

What is CYP2D6 status?

CYP2D6 is an enzyme in your liver that helps break down Cerdelga®. The genetically determined activity of this enzyme will help decide what dose of Cerdelga® is right for you. A small number of people have a form of CYP2D6 that breaks down the medication so fast it will not work for them. They are known as “ultra-rapid metabolisers” and currently Cerdelga® is not recommended for them at the moment. Conversely, another small group of people, known as ‘poor metabolisers’ break down the drug so slowly that they need a less frequent dose, and are required to take it just once a day. only once per day, Everyone else will need a twice-daily dose and they are described as either “extensive “or “intermediate metabolisers”.

What medication can I take with Cerdelga®?

Many other drugs are also broken down by the CYP2D6 and other related liver enzymes. This means that not all drugs can be taken whilst receiving treatment with Cerdelga®. There are many complex ways in which drugs can interact with Cerdelga® and you are advised to discuss your current medication. This includes over-the-counter, non-prescription, herbal supplements and complementary therapies before considering starting treatment. Some drugs, including Cerdelga®, potentially affect the electrical activity of the heart. For this reason you should have an ECG (heart tracing) before you start treatment.

Once taking Cerdelga® regularly, it is important to seek medical advice before starting any new medication.

Can I take Cerdelga® if I am pregnant?

There is very little information about the safety of Cerdelga® in pregnancy, therefore it is currently recommended to avoid Cerdelga® during pregnancy. If you are considering pregnancy you should tell your specialist team, particularly when reviewing the treatment options available to you.

Whom I can contact to discuss Cerdelga®?

For further information regarding whether you can receive treatment with Cerdelga® please contact your specialist clinical team.

Lysosomal disorders unit: 01223 274634.

For general information about Eliglustat (Cerdelga®) there is a helpful US website (opens in a new tab). Further UK specific information is currently in development.

Further information

Further information about your capsule can be found in the patient information leaflet found in the capsule box or on the container.

If you have any other questions about your medication, please contact the medicines helpline on 01223 217502.

Cerdelga® is a registered trade mark of Genzyme therapeutics for the drug eliglustat tartrate.