On 8 November 2019, the US Food and Drug Administration (FDA) approved luspatercept-aamt (REBLOZYL, Celgene Corp.) for treatment of anaemia in adult patients with beta thalassemia who require regular red blood cell transfusions.
Efficacy was evaluated in the BELIEVE trial (NCT02604433), a multicentre, randomised, double-blind, placebo-controlled trial enrolling 336 adult patients with beta thalassemia requiring regular red blood cell (RBC) transfusions. Patients were randomised 2:1 to luspatercept-aamt (224) or placebo (112). Luspatercept-aamt was administered subcutaneously once every 3 weeks as long as a reduction in transfusion requirement was observed or until unacceptable toxicity.
The primary efficacy outcome measure was the proportion of patients achieving RBC transfusion burden reduction from baseline of at least 33%, with a reduction of at least 2 units from week 13 to week 24. Of the patients who received luspatercept-aamt, 21.4% achieved the primary endpoint compared with 4.5% of those who received placebo (risk difference 17.0; 95% CI 10.4, 23.6; p < 0.0001).
The most common adverse reactions (>10%) in patients with beta thalassemia were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhoea, and dizziness.
The recommended starting dose is 1 mg/kg once every 3 weeks by subcutaneous injection.
Full prescribing information for REBLOZYL is available here.
FDA granted this application fast track and orphan drug designations.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System.