Celgene has submitted a biologics licence application (BLA) to the US Food and Drug Administration (FDA) seeking approval for luspatercept to treat patients suffering from anaemia.
The BLA covers use of the drug for adults with very low to intermediate risk anaemia associated with myelodysplastic syndromes (MDS), who have ring sideroblasts and require red blood cell (RBC) transfusions.
In addition, the company is seeking approval in adults with beta-thalassemia-associated anaemia and require RBC transfusions.
Discovered by Acceleron Pharma , luspatercept is an erythroid (red blood cell) maturation agent (EMA). Acceleron is developing the product in alliance with Celgene.
The drug candidate is meant to treat patients with serious blood disorders characterised by a late-stage defect in the RBC maturation. It is designed to repair the maturation defect and restore RBC production.
Celgene chief medical officer Jay Backstrom said: “There remains a high unmet medical need for patients with MDS or beta-thalassemia who suffer from the effects of their disease-related anaemia. The primary treatment option for these patients currently is chronic transfusion of red blood cells which can be associated with complications such as iron overload.
“New treatment options are urgently needed for these patients. With this submission, we look forward to working with the agency to deliver luspatercept to patients with these serious blood diseases.”
The BLA includes safety and efficacy data form the pivotal Phase III MEDALIST and BELIEVE clinical trials.
MEDALIST assessed luspatercept in a total of 229 adult patients with very low-, low- or intermediate-risk myelodysplastic syndromes (MDS) at 65 sites across 11 countries.
BELIEVE evaluated luspatercept in conjunction with best supportive care (BSC), compared to placebo plus BSC, in a total of 336 beta-thalassemia patients. This trial was performed at 65 sites in 15 countries.
Celgene and Acceleron plan to seek the European regulatory approval in the second quarter of this year.