Bristol-Myers Squibb Company and Acceleron Pharma Inc. announced that the New England Journal of Medicine (NEJM) has published results from MEDALIST, the pivotal phase III study evaluating the use of Reblozyl (luspatercept-aamt) to treat anemia in patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who have ring sideroblasts (RS+) and require red blood cell transfusions, and who had failed, were intolerant to, or ineligible for/unlikely to respond to treatment with erythropoiesis-stimulating agents (ESAs).
MEDALIST achieved a statistically significant improvement in the primary endpoint of red blood cell transfusion independence (RBC-TI) for 8 or more weeks during the first 24 weeks of the study. The study also met the key secondary endpoint of RBC-TI for 12 or more weeks during the first 24 or 48 weeks of the study, as well as the additional secondary endpoint of hematologic improvement-erythroid (HI-E) for 8 or more weeks as assessed by International Working Group 2006 criteria. The most common treatment-emergent adverse events (TEAEs) of any grade in greater than 10% of patients in either the treatment or placebo arm were fatigue, diarrhea, asthenia, nausea, dizziness, and back pain. TEAEs of Grade 3 or 4 were reported in 42.5% (65/153) of patients receiving luspatercept-aamt and 44.7% (34/76) of patients receiving placebo. Progression to acute myeloid leukemia (AML) occurred in three patients (2.0%) receiving luspatercept-aamt and one patient (1.3%) receiving placebo. Five patients receiving luspatercept-aamt (3.3%) and four patients receiving placebo (5.3%) experienced one or more TEAE that resulted in death.
The FDA is evaluating Reblozyl for the treatment of anemia in adults with very low- to intermediate-risk MDS who have ring sideroblasts and require red blood cell transfusions, and has set a Prescription Drug User Fee Act (PDUFA), or target action, date of
4 April 2020 for this indication. In Europe, Bristol-Myers Squibb’s Marketing Authorization Application for the treatment of anemia in adults with beta thalassemia and MDS is currently under review.
See- "Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes"- Pierre Fenaux, M.D., Ph.D., Uwe Platzbecker, M.D., Ghulam J. Mufti, F.R.C.P., Guillermo Garcia-Manero, M.D., Rena Buckstein, M.D., ., et al. January 9, 2020N Engl J Med 2020; 382:140-151 DOI: 10.1056/NEJMoa1908892.