‘BELIEVE’ Study in Adults with TDT β-thalassaemia
Update: 08 January 2021
Long term safety and efficacy results on REBLOZYL were presented at ASH2020 and demonstrated that a higher proportion of luspatercept-treated patients showed decreasing serum ferritin (SF), LIC, and myocardial iron levels during the first 48 wks. Long-term luspatercept treatment led to an increasing proportion of patients with SF < 1,000 μg/L and decreasing iron chelation therapy.
Update: 05 November 2020
Long term safety and efficacy results on REBLOZYL will be presented at the 62nd ASH Meeting in December 2020 focusing on:
- Health-related Quality of Life Outcomes
- Effect on Iron Overload and Iron Chelation Therapy
- Sustained Reductions of RBC Transfusion Burden
Update: 25 August 2020
- Long term safety and efficacy results from the phase III BELIEVE trial were presented at the EHA25 Congress in June, showing
- More than 33% reduction of required transfusions for:
- 2% of β0/β0 patients
- 39% of β0/β+ patients
- 6% of β+/β+ patients
- 7% of HbE/β-thalassaemia patients
- More than 50% reductions of required transfusion for:
- 3% of β0/β0 patients
- 3% of β0/β+ patients
- 2% of β+/β+ patients
- 8% of HbE/β-thalassaemia patients
- More than 33% reduction of required transfusions for:
The European Commission (EC) has approved Reblozyl (luspatercept) for the treatment of adult patients with transfusion-dependent anaemia associated with beta thalassemia.
Update: 31 May 2020
- The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion, recommending the approval of Reblozyl (luspatercept) for the treatment of adult patients with transfusion-dependent anemia associated with beta thalassemia.
- This CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Reblozyl would be the first erythroid maturation agent approved in the EU, representing a new class of therapy for eligible patients.
- Long term safety and efficacy results from the phase III BELIEVE trial will be presented at the EHA25 Congress in June.
Update: 31 March 2020
No update available.
Update: 31 January 2020
Data evaluating Reblozyl® (ex-Luspatercept-aamt) were presented at the 61stAmerican Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida.
- 1% of patients (101/224 patients receiving Luspatercept-aamt in the phase 3 BELIEVE study) achieved at least a 33% reduction in RBC transfusion burden over any 24-week period.
- Among the Luspatercept-aamt patients who had a response, 73.3% (74/101) had at least 2 separate responses, with 59.4% (60/101) having 3 or more responses.
- The median duration of transfusion intervals was 76.3 weeks (24-128.1).
- Iron-related efficacy endpoints were announced:
- Mean change from baseline in serum ferritin was −343.22 μg/L
- Over 96 wks, 6 (20%) Luspatercept-treated pts with baseline cardiac iron ≤ 20 ms had post-baseline results > 20 ms
- Over 96 wks, mean change in LIC among Luspatercept-treated patients overall was −0.5 mg/g.
- Conclusions: Luspatercept treatment resulted in clinically meaningful reductions in serum ferritin levels. Baseline iron overload did not seem to affect response to luspatercept; treatment resulted in clinically meaningful reductions in RBC transfusion burden regardless of baseline serum ferritin level, LIC, or myocardial iron loading.
Update: 25 November 2019
- The U.S. Food and Drug Administration (FDA) has approved REBLOZYL®(ex-Luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassaemia who require regular red blood cell (RBC) transfusions.
- The first and only FDA-approved erythroid maturation agent, representing a new class of therapy which works by regulating late-stage red blood cell maturation to help patients reduce their RBC transfusion burden.
- Published data suggest that the price in the US will be $3,441 per 25 mg vial at list price.
Update: 27 September 2019
No update available
Update: 30 July 2019
- The FDA has accepted the Biologics License Application (BLA) for Luspatercept. The FDA has granted Priority Review to this BLA for the evaluation of the beta-thalassaemia indication and set a Prescription Drug User Fee Act (PDUFA), or target action date of December 4, 2019. This is the date by which the FDA must review the application.
Update: 30 May 2019
- Application for Marketing Authorization to the European Medicines Agency (EMA) for Luspatercept for the treatment of adult patients with beta-thalassaemia-associated anemia who require RBC transfusions has been submitted.
- Submission to the FDA Biologics License Application (BLA) for Luspatercept. A BLA is submitted after an investigational new drug has been approved and constitutes a request for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.
Update: 29 March 2019
Regulatory updates have been announced indicating that Luspatercept Biologics License Application (BLA) will be submitted in April 2019.
A BLA is submitted after an investigational new drug has been approved and constitutes a request for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.
Update: 02 October 2018
Luspatercept is an erythroid maturation agent (EMA) that is believed to regulate late-stage red blood cell maturation.
Results were announced from the Phase 3, randomized, double-blind, multi-centre ‘BELIEVE’ clinical study for the evaluation of efficacy and safety of Luspatercept in adult β-thalassaemia patients.
- Luspatercept achieved a highly statistically significant improvement in the primary endpoint of erythroid response. This was defined as at least a 33 % reduction from baseline in red blood cell (RBC) transfusion burden with a reduction of at least 2 units during the protocol-defined period of 12 consecutive weeks, from week 13 to week 24, compared to placebo. Essentially this means that participants saw more than a 33% reduction in their blood transfusion requirements over a period of 12 consecutive weeks, thus indicating that Luspatercept reduces the need for blood transfusions.
- Luspatercept also met all key secondary endpoints of demonstrating statistically significant improvements in RBC transfusion burden from baseline of at least a 33% reduction during the period from week 37 to week 48, at least a 50% reduction during the period from week 13 to week 24, at least a 50% reduction during the period from week 37 to week 48, and a mean change in transfusion burden from week 13 to week 24.
Thus, the results show a significant reduction in transfusion burden compared to placebo.
Further results with be announced during the Annual ASH Congress.
Regulatory submissions are planned for the FDA and EMA in the first half of 2019, with market access anticipated in early 2020.
Update: 20 December 2018
Results from a pivotal, phase 3 trial (BELIEVE) evaluating the safety and efficacy of Luspatercept for the treatment of adults with transfusion dependent β-thalassaemia were presented at the 60th Annual Meeting of the American Society of Hematology (ASH) in San Diego, held on 01 – 04 December 2018.
- RBC Transfusion Burden (i.e. number of RBC units required) reduced by more than 33%
- The mean change was -1.35 RBC units
- Treatment-emergent adverse events were reported by 29.1% of participants, mainly anaemia (3.1%), increased LIC (2.7%), Hyperuricemia (2.7%) and others.
- Other side effects include bone pain, backache, and hypertension.