Bristol Myers Squibb announced the withdrawal of the supplemental Biologics License Application (sBLA) for luspatercept-aamt, an erythroid maturation agent, for the treatment of anemia in adults with nontransfusion dependent (NTD) beta thalassemia.
In December 2021, the Food and Drug Administration (FDA) accepted for Priority Review the sBLA for luspatercept-aamt for the treatment of anemia in adults with NTD beta thalassemia. The application included data from the phase 2 BEYOND study (ClinicalTrials.gov Identifier: NCT03342404). The decision to withdraw the application was made after the Company could not appropriately address the agency’s questions about the benefit-risk profile of luspatercept-aamt in this patient population.
Noah Berkowitz, MD, PhD, senior vice president, Hematology Development, Bristol Myers Squibb, commented: “While we will not pursue this indication in the US, we’re continuing to evaluate [luspatercept-aamt] in a broad clinical development program to bring this important therapeutic option to more patients living with the burden of anemia.”
Luspatercept-aamt is currently marketed under the brand name Reblozyl® and is approved for the treatment of anemia in adults with beta thalassemia who require regular red blood cell transfusion. It is also approved for the treatment of anemia in adults with lower-risk myelodysplastic syndromes.
Bristol Myers Squibb withdraws supplemental Biologics License Application (sBLA) for Reblozyl® (luspatercept-aamt) for non-transfusion dependent (NTD) beta thalassemia. News release. Bristol Myers Squibb. Accessed June 6, 2022. https://www.businesswire.com/news/home/20220603005473/en/Bristol-Myers-Squibb-Withdraws-Supplemental-Biologics-License-Application-sBLA-for-Reblozyl%C2%AE-luspatercept-aamt-for-Non-transfusion-Dependent-NTD-Beta-Thalassemia