Non-Transfusion Dependent Thalassaemia
Transfusion Dependent Thalassaemia
Update: 30 June 2023
- Data presented at the 28th EHA Annual Congress (9 – 11 June 2023) in Frankfurt (Germany) showed that patients on luspatercept during the BEYOND trial showed sustained improvement in haemoglobin levels and long-term erythroid response thus requiring fewer transfusions.
Update: 31 March 2023
The European Commission (EC) has granted full Marketing Authorization for Reblozyl® (luspatercept), a first-in-class therapeutic option, for treatment in adult patients of anemia associated with non-transfusion-dependent (NTD) beta thalassemia.
Reblozyl is currently approved in the European Union (EU), United States and Canada to address anemia associated with transfusion-dependent beta thalassemia and 8 transfusion-dependent lower-risk myelodysplastic syndromes. The centralized Marketing Authorization approves use of Reblozyl in all EU member states, as well as Norway, Iceland and Liechtenstein.
The EC approval of Reblozyl was based on results from the Phase 2 BEYOND study, evaluating the efficacy and safety of Reblozyl versus placebo in 145 adults with NTD beta thalassemia. See study results below.
BEYOND Study Results
BEYOND (NCT03342404) is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion-dependent beta thalassemia. The study is divided into the Screening Period, Double-blind Treatment Period (DBTP), Open-label Phase (OLP), and Post-Treatment Follow-up Period (PTFP) and randomized 145 subjects at a 2:1 ratio of Reblozyl versus placebo. All patients were eligible to receive best supportive care, which included red blood cell transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and/or nutritional support, as needed. The primary endpoint of the study is the proportion of subjects who have an increase from baseline ≥1.0 g/dL in mean of hemoglobin values over a continuous 12-week interval from Week 13 to Week 24 of treatment in the absence of transfusions. Key secondary endpoints include mean change in non-transfusion-dependent beta thalassemia-patient reported outcome (NTDT-PRO) Tiredness and Weakness (TW) domain score and baseline hemoglobin (Hb).
Results demonstrated 74 of 96 (77.1%) patients in the Reblozyl treatment arm achieved the study’s primary endpoint, ≥1.0 g/dL mean Hb increase from baseline, versus 0 of 49 (0%) patients in the placebo arm (P<0.0001).
In a key secondary endpoint of the study, 47 of 96 patients (49.0%) treated with Reblozyl achieved mean Hb increase of ≥1.5 g/dL compared to baseline from Week 37-48 in the absence of transfusions versus 0 patients (0%) in the placebo arm. In the Reblozyl arm, 89.6% of patients remained transfusion free at weeks 1-24 versus 67.3% of patients in the placebo arm. Improvements in patient reported quality of life outcomes (tiredness and weakness) were also observed to correlate with Hb increases.
Serious adverse reactions occurred in 11.5% of patients (n=11) who received Reblozyl and in 25% of patients (n=24) who received placebo. The most common adverse reactions occurring in ≥10% of patients treated with Reblozyl were bone pain (36%), headache (30%), arthralgia (29%), back pain (28%), prehypertension (23%), hypertension (20%), cough (18%), diarrhea (17%), influenza-like illness (17%), asthenia (13%), influenza (13%), insomnia (11%) and nausea (10%).
Update: 30 June 2023
- Data presented at the 28th EHA Annual Congress (9 – 11 June 2023) in Frankfurt (Germany) showed that patients on luspatercept during the BELIEVE trial maintained stable bone health and did not significantly worsen despite reduced transfusions.
Update: 31 March 2023
- The Egyptian Ministry of Health and Population (MoHP), the local healthcare authorities including the Egyptian Drug Authority (EDA) and the Egyptian Authority for Unified Procurement (UPA) have approved the commercialisation of Reblozyl® (luspatercept) in the country.
Reblozyl® (luspatercept) is available in other key markets of the Middle East including Saudi Arabia, UAE, Kuwait and Oman with others in the pipeline.