NEW YORK -- December 24, 2008 -- The European Commission has approved azacitidine (Vidaza) for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation with:
· Intermediate-2 and high-risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS), or
· Chronic myelomonocytic leukaemia (CMML) with 10% to 29% marrow blasts without myeloproliferative disorder, or
· Acute myeloid leukaemia (AML), with 20% to 30% blasts and multi-lineage dysplasia, according to World Health Organization (WHO) classification
The approval was based upon efficacy and safety data from clinical studies evaluating azacitidine in patients with MDS and refractory anaemia with excess blasts in transformation (RAEB-T), within the AML category as defined by the WHO classification system.
The efficacy and safety data were primarily provided from the international, randomised, controlled, phase 3 azacitidine survival trial (AZA-001), which demonstrated a clinically relevant increase in median survival of 9.4 months (24.4 vs 15 months) when compared with conventional care regimens.
"The unprecedented survival benefit demonstrated by [azacitidine] underscores the urgent need for patients to gain rapid access to this therapy," said lead investigator Pierre Fenaux, MD, Universite of Paris, Paris, France. "I am pleased to have such a meaningful therapy for patients in this underserved population."
In addition to extending overall survival, 45% of patients treated with azacitidine in the AZA-001 study achieved red blood cell transfusion independence. Investigators aimed to treat until disease progression and a median of 9 cycles was delivered.
The drug was generally well-tolerated by patients. The most common major adverse events for patients receiving azacitidine were thrombocytopenia (69.7%), neutropenia (65.7%) and anaemia (51.4%).
SOURCE: Celgene Corporation
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