SAN DIEGO — The benefit for patients treated by an experimental blood disease drug from Acceleron Pharma (XLRN) and licensed to Celgene (CELG) was further clarified and debated by experts Saturday at a press conference during the American Society of Hematology annual meeting.
The Acceleron drug, called luspatercept, is widely expected to secure approval late next year based on results from two separate Phase 3 clinical trials demonstrating statistically significant reductions in the need for blood transfusions. The patients enrolled in these two studies were diagnosed with myelodysplastic syndrome (MDS), a cancer-like disease of the bone marrow, and beta-thalassemia, a rare inherited disorder that reduces the production of oxygen-carrying hemoglobin.
But was the luspatercept benefit seen among MDS patients, in particular, clinically meaningful and strong enough to drive adoption?
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