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Celgene and Acceleron's Reblozyl secures FDA approval for anaemia in beta thalassaemia

Published on 11/11/19 at 10:36am

The FDA has moved to authorise Celgene and Acceleron Pharma’s Reblozyl (luspatercept-aamt) in the treatment of anaemia associated with the rare hereditary blood disorder beta thalassaemia, it has emerged.

The decision relates specifically to adult patients who require regular red blood cell (RBC) transfusions and makes Reblozyl the first and only erythroid maturation agent approved by the US regulator to treat the condition. However, the authorisation does not cover the therapy’s use as a substitute for RBC transfusions in patients who require immediate correction of anaemia.

This regulatory approval was issued under Priority Review and was based on findings that showed that 21.4% of Reblozyl patients achieved a 33% or greater reduction from baseline in RBC transfusion burden after 13 to 24 weeks of treatment compared to just 4.5% of patients receiving placebo, meeting the primary endpoint of the trial.

“Today’s approval is an important milestone and underscores our continued commitment to patients with haematology disorders,” commented Nadim Ahmed, President, Global Hematology and Oncology at Celgene. “There are very limited options for patients living with anaemia due to beta thalassemia who are dependent on long term red blood cell transfusions. We are pleased to make Reblozyl available as a new therapy for these patients to help address their anaemia, a significant clinical complication of beta thalassemia.”

Habib Dable, President and Chief Executive Officer at Acceleron, also added: “We’re thrilled that Acceleron’s first approved medicine is one with the potential to help patients with beta thalassemia, who have been in need of new treatments for this lifelong disease. We are enormously grateful to the patients, families and caregivers who participated in and supported our research. Their contributions have been essential in helping to ensure that Reblozyl would emerge successfully from our longstanding collaboration with Celgene.”

Reblozyl will become available to patients one week following the FDA decision, it was confirmed.

Matt Fellows

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