Celgene International Sarl announced that its cancer drug, Vidaza (azacitidine), has been granted full marketing authorization by the European Commission (EC) for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation with - intermediate-2 and high-risk MDS according to the IPSS, or chronic myelomonocytic leukaemia (CMML) with 10-29 per cent marrow blasts without myeloproliferative disorder, or AML with 20-30 per cent blasts and multi-lineage dysplasia, according to WHO classification.
"The EC approval of Vidaza is recognition of the significant survival benefit afforded by this therapy for critical haematologic malignancies," said Philippe Van Holle, president of Celgene Europe. "We will now begin working with local regulatory authorities on a country-by-country basis for reimbursement and distribution for all European Union member states."
The approval was based upon efficacy and safety data from clinical studies evaluating Vidaza in MDS and RAEB-T patients within the AML category as defined by the WHO classification system. These pivotal efficacy and safety data were primarily provided from the Vidaza survival trial (AZA-001), the largest, international randomized phase-III controlled study ever conducted in higher-risk MDS and WHO AML patients, demonstrating a clinically relevant increase in median survival of 9.4 months (24.4 vs. 15 months) as compared to conventional care regimens.
"The unprecedented survival benefit demonstrated by Vidaza underscores the urgent need for patients to gain rapid access to this therapy," said Pierre Fenaux, lead investigator of the AZA-001 survival trial. "I am pleased to have such a meaningful therapy for patients in this underserved population."
In addition to extending overall survival, 45 per cent of Vidaza treated patients achieved red blood cell transfusion independence in the AZA-001 study. It was also well-tolerated by patients. Investigators aimed to treat until disease progression and a median of nine cycles was delivered.
In the AZA-001 survival study, the most commonly occurring major adverse events for patients receiving Vidaza were thrombocytopenia (69.7%), neutropenia (65.7%) and anaemia (51.4%).
Vidaza has received orphan drug designation for the treatment of MDS and AML in the European Union.
In August 2008, Vidaza became the first and only drug approved by the FDA to demonstrate a significant extension of overall survival compared to conventional care regimens, for patients with Intermediate-2 and high-risk MDS and AML. The FDA approved Vidaza, the first in a new class of drugs called hypomethylating agents, for treatment of all five French, American, British (FAB) MDS subtypes, which includes both low-risk and high-risk patients.
These subtypes include: refractory anaemia (RA) or refractory anaemia with ringed sideroblasts (RARS) if accompanied by neutropenia or thrombocytopenia or requiring transfusions; refractory anaemia with excess blasts (RAEB), refractory anaemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukaemia (CMML). The more recent WHO classification system incorporates RAEB-T patients within the AML category. Vidaza has received orphan drug designation in several markets including the European Union, the US and Japan.
Celgene International Sarl, located in Boudry, in the Canton of Neuchatel, Switzerland, is a wholly owned subsidiary and international headquarters of Celgene Corporation.