Luspatercept to treat beta thalassemia

January 6, 2020 0 By FM

Luspatercept-aamt (Reblozyl) has been granted approval by the US FDA for the treatment of anaemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.

The approval of luspatercept for beta thalassemia, which received a Priority Review designation from the FDA, is based on results from the pivotal, phase 3, randomized, double-blind, placebo-controlled, multicentre BELIEVE trial evaluating the safety and efficacy of luspatercept for the treatment of anaemia in adult patients with beta thalassemia who require regular RBC transfusions (defined as 6-20 RBC units per 24 weeks, with no transfusion-free period greater than 35 days during that period).

All patients were eligible to receive best supportive care, which included RBC transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and/or nutritional support, as needed. The trial achieved a clinically meaningful and statistically significant improvement in the primary endpoint. 

Luspatercept is a first-in-class erythroid maturation agent that promotes late-stage red blood cell maturation in animal models. 

Celgene and Acceleron are jointly developing luspatercept as part of a global collaboration.