o

EUROPEAN MEDICINES AGENCY

SCIENCE MEDICINES HEALTH

EMA/39261/2015

EMEA/H/C/003724

EPAR summary for the public

Cerdelga

eliglustat

This is a summary of the European public assessment report (EPAR) for Cerdelga. 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 Cerdelga.

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

What is Cerdelga and what is it used for?

Cerdelga is a medicine used for the long-term treatment of adult patients with type-1 Gaucher disease. Gaucher disease is a rare inherited disorder, in which people do not have enough of an enzyme called glucocerebrosidase (also known as acid beta-glucosidase). This enzyme normally breaks down a fat called glucosylceramide (or glucocerebroside), and without it, the fat builds up in the body, typically in the liver, spleen and bone. This produces the symptoms of the disease: anaemia (low red blood cell counts), tiredness, easy bruising and a tendency to bleed, an enlarged spleen and liver, and bone pain and breaks.

Cerdelga is used in patients who have type-1 Gaucher disease, which is the type that usually affects the liver, spleen and bones. Cerdelga is used in patients whose body breaks down this medicine at normal speed (known as 'intermediate' or 'extensive metabolisers') or at slow speed ('poor metabolisers').

Because the number of patients with Gaucher disease is low, the disease is considered 'rare', and Cerdelga was designated an 'orphan medicine' (a medicine used in rare diseases) on 4 December 2007.

Cerdelga contains the active substance eliglustat.

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact

© European Medicines Agency, 2015. Reproduction is authorised provided the source is acknowledged.

Cerdelga is available as capsules (84 mg) to be taken by mouth. It can only be obtained with a prescription and treatment should be started and supervised by a doctor who has experience in the management of Gaucher disease. Before starting treatment with Cerdelga, a test should be carried out to find out how rapidly the medicine is broken down in the patients' bodies (i.e. to determine if they are poor, intermediate, or extensive metabolisers). This medicine should not be given to patients whose body breaks down this medicine at very fast speed (known as 'ultrarapid metabolisers') or to those for whom the ability to break down the medicine is unknown or for whom no test has been done.

The recommended dose of Cerdelga is 1 capsule taken twice a day in patients whose body breaks down the medicine at normal speed (intermediate or extensive metabolisers). For patients whose body breaks down the medicine slowly (poor metabolisers), the recommended dose is 1 capsule once a day. For more information, see the package leaflet.

How does Cerdelga work?

The active substance in Cerdelga, eliglustat, works by blocking the action of an enzyme involved in the production of the glucosylceramide fat. Because the build-up of this fat in organs like the spleen, liver and bones is responsible for the symptoms of type-1 Gaucher disease, reducing its production helps prevent its build-up, and thereby helps the affected organs to function better.

What benefits of Cerdelga have been shown in studies?

Cerdelga has been shown to be effective in treating Gaucher disease in two main studies.

The first study involved 40 previously untreated patients with type-1 Gaucher disease, and it looked mainly at the reduction in the size of patients' spleens. Patients who were given eliglustat showed an average reduction of 28% in spleen size, compared with a 2% increase in those given placebo (a dummy treatment) after 9 months of treatment. Patients who received Cerdelga also showed an improvement of other signs of the disease such as a reduction in liver size and increased levels of haemoglobin (the protein found in red blood cells that carries oxygen around the body).

Cerdelga was also shown to be effective in another study involving 160 patients with type-1 Gaucher disease, who had been previously treated with a therapy to replace the missing enzyme and whose disease symptoms were under control. Some of the patients were treated with Cerdelga, while others were treated with enzyme replacement therapy. This study found that, after treatment for a year, the disease remained stable in 85% of patients treated with Cerdelga compared with 94% of patients who continued with the enzyme replacement therapy.

What are the risks associated with Cerdelga?

The most common side effect seen with Cerdelga (which may affect more than 1 in 10 people) is diarrhoea, in approximately 6 out of 100 patients. The majority of side effects are mild and transient. For the full list of all side effects reported for Cerdelga, see the package leaflet.

Cerdelga must not be taken together with certain medicines that can interfere with the ability of the body to break down the medicine, which may affect the levels of Cerdelga in blood. For the full list of restrictions, see the package leaflet.

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Cerdelga's benefits are greater than its risks and recommended that it be approved for use in the EU. Cerdelga was shown to be effective in improving the symptoms of the disease for a majority of previously untreated patients with type-1 Gaucher disease and in keeping the disease stable in most patients previously treated by enzyme replacement. However, a minority of patients (about 15%) who were switched from enzyme replacement to Cerdelga, did not respond optimally after treatment for one year. For these patients other treatment options should be considered. All patients switching from enzyme replacement should be regularly monitored for disease progression.

Regarding safety, side effects were mostly mild and transient, but the CHMP recommended that the long-term safety of the medicine be further investigated.

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

A risk management plan has been developed to ensure that Cerdelga 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 Cerdelga, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company that markets Cerdelga will provide doctors and patients with educational materials to ensure that only patients with type-1 Gaucher disease are treated with Cerdelga and that Cerdelga is not used together with other medicines which can significantly alter its blood levels. All patients who are prescribed Cerdelga will be provided with a patient alert card. The company will also keep record of patients treated with Cerdelga to look at the long-term safety of the medicine.

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

Other information about Cerdelga

The European Commission granted a marketing authorisation valid throughout the European Union for Cerdelga on 19 January 2015.

The full EPAR and risk management plan summary for Cerdelga 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 Cerdelga, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

The summary of the opinion of the Committee for Orphan Medicinal Products for Cerdelga can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/Rare disease designation.

This summary was last updated in 01-2015.

Cerdelga

EMA/39261/2015

Page 3/3