BRIEF—Celgene submits MAA for EU approval of luspatercept

US biotech Celgene has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for luspatercept.

The company is co-developing the erythroid maturation agent with Acceleron Pharma, and the application is for the treatment of adult patients with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia who have ring sideroblasts, require red blood cell (RBC) transfusions and have received or are not eligible for erythropoiesis-stimulating agents, and for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions.

The safety and efficacy results provided in the application are from the pivotal Phase III studies MEDALIST and BELIEVE, evaluating the ability of luspatercept to effectively address anemia associated with MDS and beta-thalassemia, respectively.

“As a first-in-class erythroid maturation agent, luspatercept has the potential to become an important therapeutic option for patients with these serious diseases by treating the associated anemia and reducing the burden of transfusions,” said Dr Jay Backstrom, chief medical officer for Celgene. “We, along with our partners at Acceleron, now look forward to the regulatory process as we strive to deliver luspatercept to patients in need.”

Luspatercept is an investigational therapy that is not approved for any use in any country.