Celgene wraps up EU, U.S. filings for anemia drug luspatercept

Celgene and the analysts that cover it think the data suggest luspatercept could rack up peak sales of about $2 billion, with myelodysplastic syndromes accounting for upward of two-thirds of those revenues. (European Medicines Agency)

Three weeks after filing luspatercept for FDA approval, Celgene has submitted the anemia drug for consideration by the European Medicines Agency. The applications come four months after the Acceleron-partnered drug reduced the reliance of beta-thalassemia patients on blood transfusions in a phase 3 trial. 

Celgene and Acceleron are seeking U.S. and EU nods for luspatercept to treat patients with anemia linked to beta-thalassemia and myelodysplastic syndromes (MDS), a group of cancers in which immature blood cells in the bone marrow don’t fully develop and become healthy blood cells. Specifically, the partners are looking at patients with either blood disorder that are treated with red blood cell transfusions. Though these transfusions are a life-saving treatment, they can eventually lead to iron overload that can cause organ failure and shorten a patient’s life span. 

“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 Celgene Chief Medical Officer Jay Backstrom, M.D., in a statement. 

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In December, the partners revealed data from a 336-patient phase 3 study showing that about one-fifth of beta-thalassemia patients taking luspatercept every three weeks had a 33% decrease in transfusion burden during weeks 13 to 24 of the study. The figure for the placebo group was 5%. Those data followed results announced in June of a second phase 3 study in patients with very low- to intermediate-risk MDS.

RELATED: Bluebird's gene therapy eliminates blood transfusions for some beta-thalassemia patients  

Celgene and the analysts that cover it think the data suggest luspatercept could rack up peak sales of about $2 billion, with MDS accounting for upward of two-thirds of those revenues. Celgene picked up the rights for luspatercept, then known as ACE-536, for $25 million upfront and up to $217 million in regulatory and commercial milestones in 2011. The drug is an erythroid maturation agent—meaning that it helps immature blood cells to develop into red blood cells to reduce or eliminate the need for blood transfusions.

Bluebird Bio is also working to treat transfusion-dependent beta-thalassemia, but with gene therapy. Last year, the company revealed interim data showing 15 out of 22 patients on its lentiviral-based treatment no longer needed blood transfusions.

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