BMS Withdraws sBLA for Reblozyl for Rare Blood Disorder

Bristol Myers Squibb has withdrawn a supplemental biologics license application (sBLA) for Reblozyl (luspatercept-aamt) for the treatment of anemia in adults with non-transfusion dependent (NTD) beta thalassemia. Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin.

The company said in a press release that it could not appropriately address the FDA’s questions about the benefit-risk profile of Reblozyl in this patient population based on the current dataset from the phase 2 BEYOND trial.

Noah Berkowitz, M.D., Ph.D.

“While we will not pursue this indication in the United States, we’re continuing to evaluate Reblozyl in a broad clinical development program to bring this important therapeutic option to more patients living with the burden of anemia,” Noah Berkowitz, M.D., Ph.D., senior vice president, hematology development, at Bristol Myers Squibb, said in a press release.

The agency had accepted the sBLA in December 2021, but in March 2022, the FDA extended the review of this application. A written response to the FDA’s information request was determined required additional time to review.

Reblozyl, a first-in-class therapeutic option, is currently approved in the United States, European Union and Canada to address transfusion-dependent anemia-associated beta thalassemia and lower-risk myelodysplastic syndromes. It is being developed and commercialized through a collaboration between Bristol Myers Squibb and Merck, following Merck’s acquisition of Acceleron Pharma in November 2021.

The application was based on data from BEYOND trial, which were presented in June 2021 at European Hematology Association’s Virtual Congress. Results demonstrated that 77.7% of patients treated with Reblozyl achieved a hemoglobin increase. In the placebo group, no patients achieved a hemoglobin increase. Changes in patient-reported outcomes also correlated with increases in hemoglobin. In the study, 89.6% of patients treated with Reblozyl remained transfusion free versus 67.3% of patients in the placebo arm at weeks 1 to 24.

Beta thalassemia is caused by mutations in the HBB gene. It is relatively rare in the United States, but is one of the most common autosomal recessive disorders. The disease results in the production of fewer and less healthy red blood cells, often leading to severe anemia—a condition that can be debilitating and can lead to other complications for patients—as well as other serious health issues.

The disorder is prevalent in the Mediterranean, Middle East, Africa, central Asia, the Indian subcontinent, and the Far East, according to the National Organization for Rare Disorders. While beta thalassemia remains a rare disease, its prevalence has increased in the United States is about 7.5% over the last 50 years.