European Commission green light for luspatercept for transfusion-dependent beta-thalassemia-related anemia

European Commission green light for luspatercept for the treatment of adults with anemia associated with transfusion-dependent beta-thalassemia (NTD). The European Commission approval is based on the results of a phase 2 extracorporeal study evaluating the efficacy and safety of luspatercept and placebo in 145 adults with non-transfused beta-thalassemia.

08 March – The European Commission has approved Bristol Myers Squibb’s luspatercept as a first-in-class treatment option for the treatment of adult patients with anemia associated with transfusion-dependent beta-thalassemia (NTD).

Luspadercept is currently approved in the European Union, the United States, and Canada for the treatment of anemia associated with transfusion-dependent beta-thalassemia and very low-, low-, and intermediate-risk transfusion-dependent myelodysplastic syndromes.

A centralized marketing authorization authorizes the use of luspatercept in all EU member states, as well as in Norway, Iceland and Liechtenstein.

“Beta-thalassemia is an inherited blood disorder that puts patients at high risk for long-term medical complications from anemia, confirming the substantial need for treatment options regardless of transfusion dependency. Today’s announcement is a new treatment to reduce these complications in patients with anemia associated with non-transfusion-dependent beta-thalassemia in Europe. “The important message is to wait for treatment options,” he says. Noah Berkowitz, Senior Vice President, Hematology Development, Bristol Myers Skip. “Today’s approval marks the third indication of luspatercept in Europe, and we are evaluating this first-in-class treatment option in a large clinical development program in several anemia-related diseases.”

The European Commission approval is based on the results of a phase 2 extracorporeal study evaluating the efficacy and safety of luspatercept and placebo in 145 adults with non-transfused beta-thalassemia. Patients deserve better care, including transfusions of red blood cells and iron chelating agents.

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BEYOND (NCT03342404) is a phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept-aamt (ACE-536) versus placebo in adults with untransfused beta-thalassemia.

The study was divided into a screening period, a double-blind treatment period (DBTP) and a post-treatment follow-up period (PTFP) and randomized 145 patients in a 2:1 ratio to luspatercept and placebo. All patients deserve best supportive care, including red blood cell transfusions; iron chelating agents; Use of antibiotics, antiviral and antifungal therapy; and/or nutritional support, as needed.

The primary outcome of the study was the proportion of patients experiencing an increase in mean hemoglobin concentration ≥1.0 g/dL from baseline over a 12-week interval from treatment weeks 13 to 24, in the absence of transfusion. Key secondary endpoints included mean change in patient-reported outcomes with transfusion-dependent beta-thalassemia in the fatigue and weakness (TW) domain of the questionnaire (NTDT-PRO) and baseline hemoglobin (Hb).

74 of 96 patients (77.1%) in the luspatercept treatment arm met the study’s primary endpoint, an increase from baseline in mean hemoglobin concentration of ≥1.0 g/dL, compared to 0 (0%) of 49 patients in the placebo arm (P<0.0001).

At the study’s key secondary endpoint, 47 of 96 patients (49.0%) treated with luspadercept had a baseline hemoglobin ≥1.5 g/dL at weeks 37 to 48 compared with 0 patients without transfusion. ) in the placebo arm (P<0.0001).

In the luspatercept arm, 89.6% of patients remained transfusion-free for weeks 1 to 24, compared with 67.3% of patients in the placebo arm (P=0.0013). Improvements in patients’ quality of life (fatigue and weakness) were observed associated with increases in hemoglobin.

Serious adverse reactions with lucifercept occurred in 11.5% of patients (n=11). The most common adverse reactions were bone pain (36%), headache (30%), arthralgia (29%), back pain (28%), hypertension (23%), and hypertension in ≥10% of patients treated with luspatercept. (20%), cough (18%), diarrhea (17%), influenza-like illness (17%), asthenia (13%), fever (13%), insomnia (11%) and nausea (10%).

08 March 2023
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