Policy & Regulation
FDA Advisory Committee Will Review Reblozyl for Use in Patients with Myelodysplastic Syndromes
5 December 2019 - - The US Food and Drug Administration's Oncologic Drugs Advisory Committee will hold a review of US-based drugmaker Bristol-Myers Squibb Company's (NYSE: BMY) supplemental Biologics License Application for the use of Reblozyl (luspatercept-aamt) in patients with myelodysplastic syndromes at its meeting on December 18, 2019, Bristol-Myers Squibb and Acceleron Pharma Inc. (NASDAQ: XLRN) said.

Bristol-Myers Squibb is seeking approval of Reblozyl, an erythroid maturation agent representing a new class of therapy, for the treatment of adult patients with very low- to intermediate-risk MDS-associated anemia who have ring sideroblasts and require red blood cell transfusions.

Reblozyl is currently being reviewed by the FDA for an indication in patients with MDS, and the agency has set a Prescription Drug User Fee Act (PDUFA), or target action, date of April 4, 2020 for completion of the review.

The agency recently granted approval of Reblozyl for the treatment of anemia in adult patients with beta thalassemia who require regular RBC transfusions. Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.

Reblozyl is not approved for the treatment of MDS in any country.

Myelodysplastic syndromes are a group of closely related, but diverse blood cancers in which mutations prevent the bone marrow stem cells from making healthy blood cells. Most patients experience severe chronic anemia due to the lack of mature RBCs.

Anemia associated with MDS remains a significant area of unmet need for these patients, as current treatment options are limited, consisting primarily of medicines that stimulate the production of erythropoietin, and regular RBC transfusions.

Regular RBC transfusions can disrupt and diminish a patient's quality of life and are associated with an increased risk of iron overload, transfusion reactions and infections.

Reblozyl is a first-in-class erythroid maturation agent that promotes late-stage RBC maturation in animal models. Bristol-Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration.

It is currently approved in the US for the treatment of anemia in adult patients with beta thalassemia who require regular RBC transfusions. 

Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia. A Phase 3 trial (COMMANDS) in erythroid stimulating agent-naïve, lower-risk MDS patients, the BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta thalassemia, and a Phase 2 trial in myelofibrosis patients are ongoing.
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