Celgene and Acceleron Pharma have a filed an application with the European Medicines Agency (EMA) seeking approval for luspatercept for the treatment of anemia associated with myelodysplastic syndromes (MDS) and beta-thalassemia.
Luspatercept is an erythroid maturation agent (EMA), which has been designed to regulate late-stage red blood cell maturation. The drug is being jointly developed by Acceleron and Celgene under a global collaboration.
The Marketing Authorization Application (MAA) submitted for the drug is for its use in the treatment of very low to intermediate-risk MDS-associated anemia in adults with ring sideroblasts who need red blood cell (RBC) transfusions and have received or are not eligible for erythropoiesis-stimulating agents.
The second indication being targeted for the drug is for the treatment of beta-thalassemia-associated anemia in adult patients who need RBC transfusions.
The MAA for the erythroid maturation agent is backed by data from the phase 3 trials MEDALIST and BELIEVE which assessed the investigational drug’s ability in the effective treatment of anemia associated with MDS and beta-thalassemia, respectively.
Celgene chief medical officer Jay Backstrom said: “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.
“We, along with our partners at Acceleron, now look forward to the regulatory process as we strive to deliver luspatercept to patients in need.”
Earlier this month, Celgene and Acceleron Pharma submitted a Biologics License Application (BLA) for luspatercept for the two indications to the US Food and Drug Administration (FDA).
The erythroid maturation agent, which was discovered by scientists at Acceleron Pharma, is believed to have the ability to repair RBC maturation defect in serious blood disorders and restore and increase production of the red blood cells.
According to Acceleron Pharma, luspatercept is a ligand trap, which is designed to target certain TGF-beta proteins that have a role in late-stage red blood cell maturation.
Under its global collaboration with Celgene, the clinical-stage biopharma company is developing the drug for the treatment of chronic anemia in myelofibrosis as well. The partners are currently evaluating the investigational drug in a phase 3 trial, called COMMANDS, in ESA-naïve, lower-risk MDS patients, the BEYOND phase 2 trial in non-transfusion-dependent beta-thalassemia, and a phase 2 trial in myelofibrosis.