The EC approval was based on results from Phase 2 BEYOND study and allows BMS to commercialise Reblozyl in all EU member states, in addition to Norway, Iceland, and Liechtenstein
Bristol-Myers Squibb facility in Wirral, England. (Credit: Rept0n1x/Wikipedia)
Bristol Myers Squibb (BMS) has received the European Commission (EC) full marketing authorisation for Reblozyl (luspatercept) to treat anaemia in adults with non-transfusion-dependent (NTD) beta thalassemia.
Luspatercept is a recombinant fusion protein derived from human activin receptor type IIb (ActRIIb) that is linked to a protein derived from immunoglobulin G.
The US-based pharmaceutical company is developing the drug under a global collaboration with Merck, which started after the acquisition of Acceleron Pharma, in 2021.
Reblozyl is currently approved in the European Union (EU), the US, and Canada to treat anaemia related to transfusion-dependent beta thalassemia and low-risk myelodysplastic syndromes.
The current marketing authorisation allows the commercialisation of Reblozyl in all EU member states, along with Norway, Iceland, and Liechtenstein.
The drug is not recommended for use as a substitute for RBC transfusions in patients who require immediate correction of anaemia, BMS said.
Bristol Myers Squibb Haematology development senior vice president Noah Berkowitz said: “Beta thalassemia is an inherited blood disorder that puts patients at significant risk for long-term clinical complications due to anaemia, leaving a substantial need for treatment options, regardless of a patient’s dependence on blood transfusions.
“This announcement is welcome news for patients with non-transfusion-dependent beta thalassemia-associated anaemia across the EU who are seeking newer treatment options to reduce these burdens.
“Today’s approval represents the third indication for Reblozyl in Europe, and we look forward to continuing to evaluate this first-in-class therapeutic option across multiple diseases impacted by the burden of anaemia in a broad clinical development programme.”
The EC approval was based on results from the Phase 2 BEYOND study, which evaluated the efficacy and safety of Reblozyl compared to placebo in 145 adults with NTD beta thalassemia.
The study period was divided into the Screening Period, Double-blind Treatment Period (DBTP) and Post-Treatment Follow-up Period (PTFP).
Its results showed that 77.1% of patients receiving the Reblozyl achieved the primary endpoint of ≥1.0 g/dL mean Hb increase from baseline, compared to placebo.
In addition, 49% of patients treated with Reblozyl achieved a mean Hb increase of ≥1.5 g/dL in the absence of transfusions, compared to placebo, a key secondary endpoint of the study.
The most common adverse reactions include bone pain, headache, arthralgia, back pain, prehypertension, hypertension, cough, diarrhoea, influenza-like illness, asthenia, influenza, insomnia, and nausea.