What is Cerdelga - eliglustat used for?
Cerdelga is a medicine indicated for the long-term treatment of adult patients with type 1 Gaucher disease. Gaucher disease is a rare inherited disorder in which there is a deficiency of an enzyme called glucocerebrosidase (also called beta-glucosidase acid), which normally metabolizes a fat called glucosylceramide (or glucocerebroside). In the absence of the enzyme, fat is deposited in the body, usually in the liver, spleen and bones. This results in the typical symptoms of the disease: anemia (low number of red blood cells), fatigue, tendency to ecchymosis and bleeding, enlargement of the spleen and liver, bone pain and fractures. Cerdelga is used in patients with type 1 Gaucher disease, which is the form that normally affects the liver, spleen and bones. It is used in subjects that metabolize it at normal speed (ie in the so-called "intermediate metabolizers" or "extensive") or at slow speed ("slow metabolizers"). 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 ingredient eliglustat.
How is Cerdelga - eliglustat used?
Cerdelga is available as capsules (84 mg) to be taken by mouth. The medicine can only be obtained with a prescription and treatment should be started and followed by a doctor experienced in the management of Gaucher disease. Before starting the treatment with Cerdelga it is necessary to carry out a test to determine the rapidity of metabolization of the medicine in the patient's body (ie to determine if it is a question of slow, intermediate or extensive metabolisers). The medicine should not be administered to patients who metabolize it at a very high rate (so-called "ultra-fast metabolisers") or to subjects whose ability to metabolize the drug is not known or in whom the rate of metabolism has not been determined with a special test. The recommended dose of Cerdelga is one capsule twice a day in patients with normal metabolizing activity (intermediate or extensive metabolisers). In patients whose body metabolizes the medicine slowly (slow metabolisers), the recommended dose is one capsule once a day. For more information, see the package leaflet.
How does Cerdelga - eliglustat work?
The active substance in Cerdelga, eliglustat, works by blocking the action of an enzyme involved in the production of glucosylceramide. Since the accumulation of this fatty substance in organs such as the spleen, liver and bones is responsible for the symptoms of type 1 Gaucher disease, reducing its production helps to limit its accumulation in such organs, which therefore work better.
What benefit has Cerdelga - eliglustat shown during the studies?
Cerdelga has been shown to be effective in the treatment of Gaucher disease in two main studies. 40 patients with previously untreated type 1 Gaucher disease participated in the first study, which mainly examined the reduction in spleen size. Subjects treated with eliglustat showed an average reduction in the size of the spleen by 28% compared to the 2% increase observed in patients treated with placebo (a dummy treatment) nine months after the treatment. Patients treated with Cerdelga have also shown an improvement in other signs of the disease, including a reduction in the size of the liver and an increase in hemoglobin levels (the protein in red blood cells that carries oxygen in the body). The efficacy of Cerdelga was also demonstrated in another study, involving 160 patients with type 1 Gaucher disease, previously treated with an absent enzyme replacement therapy and in whom the symptoms of the disease were under control. Some patients were treated with Cerdelga, while others were treated with enzyme replacement therapy. From this study it was found that, after a one-year treatment, the disease remained stable in 85% of subjects treated with Cerdelga compared to 94% of patients who continued with enzyme replacement therapy.
What is the risk associated with Cerdelga - eliglustat?
The most common side effect of Cerdelga (which may affect more than 1 in 10 people) is diarrhea, which affects about 6 out of 100 patients. Most side effects are mild and transient. For the full list of all side effects reported with Cerdelga, see the package leaflet. Cerdelga should not be taken in combination with certain medicines that can interfere with the body's ability to degrade it, as this interference can affect the levels of Cerdelga in the blood. For the full list of limitations, see the package leaflet.
Why has Cerdelga - eliglustat been approved?
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 has been shown to be effective in improving the symptoms of the disease in a majority of previously untreated type 1 Gaucher disease patients, as well as in keeping the disease stable in most patients previously treated with enzyme replacement therapy. However, a minority of patients (about 15%) who switched from an enzyme replacement therapy to Cerdelga did not respond optimally after a one-year treatment. For these patients, other treatment options should be considered. Disease progression should be periodically monitored in all patients switching from an enzyme replacement therapy to Cerdelga. Regarding safety, although the undesirable effects were mostly mild and transient, the CHMP recommended further studying the long-term safety of the medicine.
What measures are being taken to ensure the safe and effective use of Cerdelga - eliglustat?
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 information materials to ensure that only people with type 1 Gaucher disease are treated with Cerdelga and that the medicine is not used in combination with other drugs that could significantly alter the blood levels. All patients who will be prescribed Cerdelga will receive an alert card. The company will also keep a register of patients treated with Cerdelga to assess the long-term safety of the medicine. Further information is available in the summary of the risk management plan.
More information on Cerdelga - eliglustat
On 19 January 2015, the European Commission issued a marketing authorization for Cerdelga, valid throughout the European Union. 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 related to Cerdelga is available on the Agency's website: ema.Europa.eu/Find medicine / Human medicines / Rare disease designation. Last update of this summary: 01-2015.
