What is Vidaza?
Vidaza is a powder that is made up into a suspension for injection. The active ingredient contained in it is azacitidine.
What is Vidaza used for?
Vidaza is indicated for the treatment of adult patients with the following diseases who cannot undergo a bone marrow transplant:
- myelodysplastic syndromes, which are a group of diseases in which the bone marrow produces an insufficient number of blood cells. In some cases, these syndromes can lead to the development of acute myeloid leukemia (AML), a type of cancer that affects particular white blood cells, the myeloid cells. Vidaza is used in patients with intermediate to high risk of developing AML or death;
- chronic myelomonocytic leukemia (CML), a type of cancer that affects particular white blood cells, monocytes. Vidaza is used when the bone marrow is made up of 10-29% abnormal cells and does not produce enough white blood cells;
- AML following myelodysplastic syndrome. Vidaza is only used when the bone marrow is made up of 20-30% abnormal cells.
As the number of patients with these diseases is low, these diseases are considered rare and therefore Vidaza was designated an "orphan medicine" (a medicine used in rare diseases) on 6 February 2002 for myelodysplastic syndromes and on 29 November 2007 for AML. . At the time of this designation, LMMC was classified among myelodysplastic syndromes.
The medicine can only be obtained with a prescription.
How is Vidaza used?
Treatment with Vidaza should be initiated and monitored under the supervision of a physician experienced in chemotherapy. Before starting treatment with Vidaza, patients should be given medicines for nausea and vomiting.
The recommended starting dose of Vidaza is 75 mg per square meter of body surface area (calculated based on the patient's weight and height) and is administered by subcutaneous injection in the upper arm, thigh or abdomen each day for one week, followed by three weeks without treatment. This four-week period constitutes a "cycle". Treatment continues for at least six cycles and then as long as the patient continues to benefit from it. Before each
your liver, kidneys and blood should be checked. If the blood counts drop too low or if the patient develops kidney problems, the next treatment will be postponed or conducted at a reduced dose. Patients with severe liver problems should be carefully monitored for side effects, however Vidaza should not be used in patients with advanced liver cancer.
For all details, see the Summary of Product Characteristics included in the EPAR.
How does Vidaza work?
The active substance in Vidaza, azacitidine, is a drug of the 'antimetabolite' class. Azacitidine is an analogue of cytidine; this means that it is incorporated into the genetic material of the cells (RNA and DNA). It is thought to work by changing the way the cell turns genes on and off and also by interfering with the production of new RNA and DNA. Such interventions are believed to correct the problems with the maturation and growth of new blood cells in the bone marrow that cause myelodysplastic syndromes and that kill cancer cells in cases of leukemia.
How has Vidaza been studied?
The effects of Vidaza were first tested in experimental models before being studied in humans.
Vidaza was the subject of a single main study involving 358 adults with intermediate to high risk myelodysplastic syndrome, CML or AML who were unlikely to have a bone marrow transplant. The bone marrow of these patients contained 10-29% of abnormal cells, with not very high white blood cell counts. The study compared Vidaza to conventional therapies (treatment chosen individually based on local practices and the patient's disease). All patients also received "best supportive care" (ie drugs or treatments that help the patient, such as antibiotics, pain relievers, and transfusions); some patients have also been treated with other anticancer drugs, such as cytarabine with or without anthracycline. The main measure of effectiveness was how long the patients lived. The study lasted 44 months.
What benefit has Vidaza shown during the studies?
Vidaza has been shown to be more effective than traditional treatments in prolonging survival. Patients treated with Vidaza survived for an average of 24.5 months, compared with 15 months for patients treated with conventional care. The effects of Vidaza are similar for all three diseases.
What is the risk associated with Vidaza?
The most common side effects of Vidaza (seen in over 60% of treated patients) are blood reactions including thrombocytopenia (low platelet counts), neutropenia (low levels of neutrophils, a type of white blood cell) and leukopenia (low platelet counts) of white blood cells), side effects affecting the stomach and intestines, including nausea and vomiting, and injection site reactions. For the full list of side effects reported with Vidaza, see the package leaflet.
Vidaza should not be used in patients who may be hypersensitive (allergic) to azacitidine or any of the other ingredients. Vidaza cannot be used in patients with advanced liver cancer or in women who are breastfeeding.
Why has Vidaza been approved?
The Committee for Medicinal Products for Human Use (CHMP) decided that Vidaza's benefits are greater than its risks for the treatment of adult patients ineligible for haematopoietic stem cell transplantation with intermediate 2 and high risk myelodysplastic syndromes, MMC with 10 -29% abnormal blasts without myeloproliferative disorder or AML with 20-30% blasts and multilinear dysplasia. The committee recommended the granting of a marketing authorization for Vidaza.
Other information about Vidaza:
On December 17, 2008, the European Commission granted Celgene Europe Ltd a "Marketing Authorization" for Vidaza, valid throughout the European Union.
For the registration of the designation of Vidaza as an orphan drug here (myelodysplastic syndromes) e here (LMA).
For the full version of the Vidaza EPAR, click here.
Last update of this summary: 11-2008.
The information on Vidaza - azacitidine published on this page may be out of date or incomplete. For a correct use of this information, see the Disclaimer and useful information page.
