The approval was based on the findings from the Phase 3 COMMANDS trial in which 58.5% of patients administered with Reblozyl against 31.2% of patients treated with epoetin alfa met the primary endpoint of RBC-TI of at least 12 weeks
A product image of Reblozyl. (Credit: Bristol Myers Squibb)
American pharmaceutical company Bristol Myers Squibb has received expanded approval from the US Food and Drug Administration (FDA) for Reblozyl (luspatercept-aamt) to treat certain types of anaemia patients.
Reblozyl is a first-in-class therapeutic option that encourages late-stage red blood cell maturation in animal models.
The FDA has approved Reblozyl as the first line of treatment against anaemia without previous erythropoiesis-stimulating agent use (ESA-naïve) in adult patients who have very low- to intermediate-risk myelodysplastic syndromes (MDS) and who need regular red blood cell (RBC) transfusions.
The approval was based on the findings from the Phase 3 COMMANDS trial.
In the trial, the therapy demonstrated superior efficacy of concurrent RBC transfusion independence (RBC-TI) and haemoglobin (Hb) increase compared to epoetin alfa, an ESA, regardless of ring sideroblast status.
The late-stage trial showed that 58.5% of patients administered with Reblozyl against 31.2% of patients treated with epoetin alfa met the primary endpoint of RBC-TI of at least 12 weeks with a mean Hb growth of at least 1.5 g/dL during the first 24 weeks.
According to the pharmaceutical firm, these findings highlight Reblozyl’s capacity to treat chronic anaemia earlier during therapy in a larger patient population.
Bristol Myers Squibb US Hematology and Cell Therapy SVP and GM Wendy Short-Bartie said: “Today’s expanded approval of Reblozyl marks an important milestone in our commitment to MDS patients with anaemia by providing a durable and more effective treatment option, with more convenient and less frequent administration.
“We remain dedicated to addressing hard-to-treat diseases with a significant burden to patients and look forward to bringing this important option earlier in the treatment process.”
In a separate development, the company announced new long-term follow-up results from two Phase 3 trials of CAMZYOS (mavacamten) in adult patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
The results from the 56-week analysis of the VALOR-HCM long-term extension (LTE) study showed that with longer follow-up, CAMZYOS continued to reduce eligibility for invasive septal reduction therapy (SRT).
Additionally, the findings from the cumulative 120-week analysis of the EXPLORER cohort of the MAVA-LTE study demonstrated sustained improvements in LVOT obstruction, symptoms, and N-terminal pro-brain natriuretic peptide (NT-proBNP) levels with no signs of new safety signals.