FDA grants fast track status to luspatercept for beta-thalassemia
The FDA granted fast track status to luspatercept for patients with transfusion- dependent beta-thalassemia and nontransfusion-dependent beta-thalassemia, according to a press release from the drug’s manufacturer.
Luspatercept (Acceleron and Celgene) — a modified activin receptor type IIB fusion protein — acts as a ligand trap for members of the Transforming Growth Factor-Beta superfamily involved in the late stages of red blood cell production.
Unlike erythropoietin, which stimulates the proliferation of early-stage erythrocyte precursor cells, luspatercept regulates late-stage red blood cell precursor cell differentiation and maturation, according to the press release.
Luspatercept is currently being evaluated in a phase 2 trial in patients with beta-thalassemia and myelodysplastic syndromes.
“The FDA’s Fast Track designation for the luspatercept development program recognizes the serious unmet medical needs of patients with beta-thalassemia and the potential for luspatercept in this area,” Jacqualyn A. Fouse, PhD, president of hematology/oncology at Celgene, said in a press release. “Celgene and Acceleron are working diligently to initiate a phase 3 clinical program in 2015 to treat patients with beta-thalassemia and we look forward to continuing to work closely with health authorities and other important stakeholders to advance this program.”