Two drugmakers have submitted an approval application to the Food and Drug Administration for a drug to treat a rare blood disorder and a type of blood cancer.
Cambridge, Massachusetts-based Acceleron Pharma and Summit, New Jersey-based Celgene said Friday that they had submitted a Biologics License Application to the FDA for luspatercept, for patients with the inherited blood disorder beta-thalassemia who require red blood cell transfusions. They also are seeking the drug’s approval for anemia in patients with low- to immediate-risk myelodysplastic syndrome (MDS), a type of malignant blood disease, who have ring sideroblasts and require red blood cell transfusions.
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In a note to clients Sunday, Cowen analyst Yaron Werber wrote that the filing was in line with expectations, and approval is expected in April 2020. While Cowen anticipates a regular review, there is also a “good probability” that the FDA will give the drug a priority review designation, a question on which Celgene should hear from the agency by June 4. That would move the FDA’s decision date to December of this year.
The application is based on two Phase III trials, MEDALIST, in MDS, and BELIEVE, in beta-thalassemia. Results of MEDALIST presented at the American Society of Hematology meeting in December showed that a statistically significantly higher percentage of patients receiving luspatercept became transfusion-dependent than those receiving placebo. The BELIEVE results, also presented at the ASH meeting, showed statistically significant improvements in transfusion dependence by 33 percent or more and 50 percent or more.
The submission came just a week after the European Medicines Agency’s Committee for Human Medicinal Products gave a preliminary nod to another therapy for beta-thalassemia, bluebird bio’s gene therapy Zynteglo, meaning that therapy is now awaiting conditional approval by the European Commission. Celgene and Acceleron plan to seek EMA approval for luspatercept in the second quarter of this year. Beta-thalassemia is more common in Europe, particularly around the Mediterranean – than in the US. Celgene has a partnership with bluebird focused on bb2121, a CAR-T therapy for the blood cancer multiple myeloma.
Photo: MilosJokic, Getty Images
