A drug for a type of blood cancer will face scrutiny by a panel of outside experts later this month as it goes before the Food and Drug Administration for review, the manufacturers said Tuesday.
Cambridge, Massachusetts-based Acceleron Pharma and New York-based Bristol-Myers Squibb said their application for the drug Reblozyl (luspatercent-aamt), in anemia associated with low- to intermediate-risk myelodysplastic syndrome patients with ring sideroblasts and who require red blood cell transfusions, would undergo review by the Oncologic Drugs Advisory Committee at the panel’s Dec. 18 meeting. Acceleron had developed the drug in partnership with Celgene, which BMS acquired earlier this year for $74 billion.
Reblozyl – which the FDA approved on Nov. 8 as the first drug to treat patients with the inherited blood disorder beta-thalassemia – is an erythroid maturation agent that the company noted represents a new class of therapy. The FDA is expected to rule on whether or not to approve the drug for MDS by April 4, 2020. Acceleron and Celgene submitted their applications for Reblozyl in MDS and beta-thalassemia in April of this year. The agency usually convenes advisory committees in cases when a drug represents a new class of therapy requiring additional, outside expertise.
Current treatments for low- or intermediate-risk MDS include blood transfusions, erythropoiesis-stimulating agents and – in the case of those who carry a mutation called deletion-5q – Celgene’s drug Revlimid (lenalidomide).
Data from the Phase III MEDALIST study presented at the American Society of Hematology’s 2018 annual meeting showed a statistically significant increase in red blood cell transfusion independence among patients receiving Reblozyl compared with those receiving placebo. Among 153 patients receiving Reblozyl, 38 percent achieved RBC transfusion independence for at least eight weeks during the first 24 weeks of study, and 28.1 percent achieved at least 12 weeks. Meanwhile, 33.3 percent achieved at least 12 weeks of independence in the first 48 weeks of study. Treatment-emergent adverse events of Grade 3-4 were reported in 42.5 percent of patients on Reblozyl and 44.7 percent among those on placebo. Four patients on the drug progressed to acute myeloid leukemia, compared with one on placebo.
Photo: FDA, Flickr (free of copyright protection)
