What is Cerdelga - eliglustat and what is it 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 a deficiency in an enzyme called glucocerebrosidase (also called beta-glucosidase acid) is observed. , which under normal conditions 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. The typical symptoms of the disease result: anemia (low number of red blood cells), fatigue, tendency to bruising 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 people who metabolize it at a normal rate (ie in so-called 'intermediate' or 'extensive' metabolisers) or at a slower rate ('slow 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.
How is Cerdelga used - eliglustat?
Cerdelga is available in capsule form (84 mg) to be taken by mouth. The medicine can only be obtained with a prescription and treatment should be started and supervised by a doctor experienced in the management of Gaucher disease. Before starting treatment with Cerdelga, a test should be performed to determine how quickly the medicine is metabolized in the patients' bodies (ie to determine whether they are poor, intermediate or extensive metabolisers). The medicine must not be given to patients who metabolize it at a very high rate (so-called "ultra-rapid metabolisers") or to individuals whose ability to metabolise the medicine is not known or in whom the rate of metabolism has not been determined in a specific test. The recommended dose of Cerdelga is of one capsule twice daily in patients with "metabolic activity within the normal range" (intermediate or extensive metabolisers). In patients whose body metabolizes the medicine slowly (poor 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 these organs, which therefore function better.
What benefit has Cerdelga - eliglustat shown during the studies?
Cerdelga has been shown to be effective in treating Gaucher disease in two main studies. 40 previously untreated patients with type 1 Gaucher disease participated in the first study, which mainly looked at reduction in the size of the spleen. treated with eliglustat had an average reduction in spleen size of 28% compared with the 2% increase seen in patients treated with placebo (a dummy treatment) nine months after treatment. Patients treated with Cerdelga also showed 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 around the body). The efficacy of Cerdelga was also demonstrated in another study involving 160 patients with type 1 Gaucher disease who had previously been treated with absent enzyme replacement therapy and whose disease symptoms were under Some patients were treated with Cerdelga, while others were treated with enzyme replacement therapy. This study found that, after one year of treatment, the disease remained stable in 85% of subjects treated with Cerdelga compared 94% of patients who continued enzyme replacement therapy.
What is the risk associated with Cerdelga - eliglustat?
The most common side effect with Cerdelga (which may affect more than 1 in 10 people) is diarrhea, which affects approximately 6 in 100 patients. Most side effects are mild and transient. For the full list of side effects detected with Cerdelga, see package leaflet. Cerdelga must not be taken in combination with certain medicines that may interfere with the body's ability to break it down, as this interference may affect the blood levels of Cerdelga. For the full list of restrictions, 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 patients with type 1 Gaucher disease, as well as in maintaining stable disease in most patients previously treated with enzyme replacement therapy. However, a minority of patients (approximately 15%) who are patients who switched from enzyme replacement therapy to Cerdelga did not respond optimally after one year of treatment. Other treatment options should be considered for these patients. Disease progression should be monitored periodically in all patients who switch from therapy. enzyme replacement in Cerdelga Regarding safety, although side effects were of mostly mild and transient in nature, the CHMP recommended further investigating the medicine's long-term safety.
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 added to the summary of product characteristics and 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 people with type 1 Gaucher disease are treated with Cerdelga and that the medicine is not used in combination with other medicines that may significantly alter their blood levels. All patients who are 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 can be found in the summary of the risk management plan.
More information about Cerdelga - eliglustat
On 19 January 2015, the European Commission granted a "Marketing Authorization" for Cerdelga, valid throughout the European Union. For more information on Cerdelga therapy, read the package leaflet (included with the EPAR) or consult your doctor or the pharmacist. The summary of the opinion of the Committee for Orphan Medicinal Products for 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.
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