Celgene's Reblozyl becomes first treatment cleared in US for anaemia in beta thalassaemia

Celgene said Friday that the FDA has approved Reblozyl (luspatercept-aamt) for the treatment of anaemia in adults with beta thalassaemia who require regular red blood cell (RBC) transfusions. According to the company, the decision marks the first erythroid maturation agent, as well as the first treatment for anaemia in beta thalassaemia, cleared in the US.

Reblozyl, also known as ACE-536, is being co-developed with Acceleron Pharma, and works by regulating the late-stage maturation of red blood cells to help patients reduce their RBC transfusion burden. "Today's approval is an important milestone and underscores our continued commitment to patients with haematology disorders," commented Nadim Ahmed, president of global haematology and oncology at Celgene, adding that there are "very limited options" for patients with the condition who depend on long-term RBC transfusions.

A typical dose of Reblozyl consists of 1 mg of the drug per kg of patient's weight, with Acceleron indicating that a 75-mg vial will be available at a wholesale acquisition cost of about $10,324. The drug is expected to be available a week following the approval. Acceleron will co-promote Reblozyl with Celgene in North America, while the latter will handle marketing outside the continent.

The FDA filing, which was assessed via a priority review, was supported by data from the 336-patient Phase III BELIEVE trial. Results showed that the study met its main goal, with 21.4% of Reblozyl-treated patients on best supportive care achieving at least a 33% reduction in transfusions compared to 4.5% for placebo plus best supportive care. Celgene noted that patients treated with Reblozyl will be followed for up to five years.

The FDA is also evaluating Reblozyl as a potential anaemia treatment in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who have ring sideroblasts and require RBC transfusions, and is scheduled to make a decision on this indication by April 4 next year. Meanwhile, Celgene's European filing for the drug to treat anemia in adults with beta thalassaemia or MDS is currently under review.

Celgene is currently in the process of being acquired by Bristol-Myers Squibb for $74 billion, with the transaction expected to close by the end of 2019. Baird analyst Brian Skorney, who expects Reblozyl to generate global sales of $1.7 billion by 2027, said the drug is a nice product, but "won't be anything that has shock and awe value for Bristol-Myers Squibb."

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