The Food and Drug Administration (FDA) expanded the approval of Reblozyl (luspatercept-aamt) to include the treatment of anemia (low red blood cell count) in patients with very low- to intermediate-risk myelodysplastic syndrome (MDS) who may require red blood cell transfusions and have not previously received any erythropoiesis-stimulating agents (ESAs), according to Bristol Myers Squibb, the manufacturer of the drug.
“For patients with lower-risk MDS, current standard therapies, including ESAs, have provided limited benefit in controlling anemia, with only one in three patients responding for a duration of six to 18 months,” Dr. Guillermo Garcia-Manero, lead investigator and Chief of the Section of Myelodysplastic Syndromes at The University of Texas MD Anderson Cancer Center, said in a press release issued by Bristol Myers Squibb.
The approval is based on findings from the phase 3 COMMANDS clinical trial, which showed that Reblozyl was better at achieving transfusion independence (when patients are not reliant on receiving transfusions of red blood cells) of 12 weeks or longer, compared with epoetin alfa, an injectable drug that is commonly used to treat anemia.
The trial included adults with very low- to intermediate-risk MDS who required red blood cell transfusions: half the patients were randomly assigned to receive Reblozyl, while the other half received epoetin alfa. The main goal of the study was red blood cell transfusion independence for at least 12 weeks with a concurrent mean hemoglobin increase of at least 1.5 g/dL (weeks 1-24). Study findings showed that this endpoint was reached in 58.5% of patients in the Reblozyl group compared with 31.2% in the epoetin alfa group.
Within the first 24 weeks of treatment, red blood cell transfusion independence lasting at least 24 weeks was achieved by 47.6% of patients taking Reblozyl, compared with 29.2% of epoetin alfa patients. Additionally, a total of 66.7% and 46.1% of patients in the Reblozyl and epoetin alfa groups, respectively, achieved red blood cell transfusion independence.
“Results from the COMMANDS study showed nearly twice as many patients treated with Reblozyl achieved transfusion independence of at least 12 weeks and concurrent hemoglobin increase compared to epoetin alfa. Today’s approval represents an important advancement for patients with lower-risk MDS,” Garcia-Manero said.
Of note, Reblozyl was initially approved in the MDS space in 2020, to treat anemia in patients who previously received an erythropoiesis stimulating agent.
Common moderate to severe side effects (grade 3 or 4) that occurred in 3% or more of patients taking Reblozyl included high blood pressure, anemia, difficulty breathing, neutropenia, thrombocytopenia, pneumonia, COVID-19 and fainting. The most common all-grade side effects from the agent were fatigue, weakness/lack of energy, peripheral edema (leg swelling), nausea and difficulty breathing.
“The majority of patients with MDS experience chronic anemia and require (red blood cell) transfusions,” said Tracey Iraca, executive director, MDS Foundation, in the press release. “The approval of Reblozyl in the first-line treatment of anemia for patients with lower-risk MDS represents a crucial step in making transfusion independence possible for more patients.”
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