Cerdelga

Cerdelga is available as capsules (84 mg) to be taken by mouth. Before starting treatment, the patient is tested to see how rapidly their bodies break down the medicine. Patients who break down the medicine at normal speed take 1 capsule twice a day, while those who break down the medicine slowly take 1 capsule once a day.

Patients whose bodies break down this medicine very rapidly (‘ultrarapid metabolisers’) should not take Cerdelga. Patients who have not been tested or whom results of the test are not clear should also not take this medicine.

Cerdelga 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. For more information about using Cerdelga, see the package leaflet or contact your doctor or pharmacist.

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.

Studies show that Cerdelga is effective at treating the disease, including reducing the size of enlarged spleens and livers.

In a study of 40 previously untreated patients with type-1 Gaucher disease, patients taking Cerdelga had a 28% reduction in spleen size, compared with a 2% increase in those taking placebo (a dummy treatment) after 9 months of treatment. Patients who took 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 in red blood cells that carries oxygen).

Another study looked at Cerdelga in 159 patients whose disease was well controlled by enzyme replacement therapy. In this study, the disease remained stable in 85% of patients who switched to Cerdelga for a year and in 94% of patients who continued with enzyme replacement therapy.

The most common side effect seen with Cerdelga is dyspepsia (heartburn) in approximately 6 out of 100 patients. The most common serious side effect is fainting, in 8 out of every 1,000 patients. The majority of side effects are mild and short-lived. For the full list of side effects reported for Cerdelga, see the package leaflet.

Cerdelga must not be taken by some patients with liver problems or those taking certain medicines that can interfere with the ability of the body to break down the medicine. For the full list of restrictions, see the package leaflet.

Studies show that Cerdelga is 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 it was recommended that the long-term safety of the medicine be further investigated.

The European Medicines Agency decided that Cerdelga’s benefits are greater than its risks and it can be authorised for use in the EU.

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 in some patients with liver problems and 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. All healthcare professionals who are expected to prescribe Cerdelga will be provided with a prescriber guide. The company will also keep record of patients treated with Cerdelga to look at the long-term safety of the medicine.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Cerdelga have also been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Cerdelga are continuously monitored. Side effects reported with Cerdelga are carefully evaluated and any necessary action taken to protect patients.

Cerdelga received a marketing authorisation valid throughout the EU on 19 January 2015.