FDA approves first treatment for anemia in beta thalassemia patients

FDA approved on Friday Reblozyl luspatercept-aamt from Celgene and Acceleron as the first treatment for anemia in β thalassemia patients who require red blood cell transfusions. The erythroid maturation agent will be available to patients within a week.

The approval is the first for Acceleron, which was founded in 2003, and will give Bristol-Myers Squibb Co. (NYSE:BMY) the one first-in-class asset among roughly 30 clinical therapies it will gain when it completes its $74 billion acquisition of Celgene Corp. (NASDAQ: CELG) this year. BMS is looking to Reblozyl plus four other late-stage Celgene assets and one of its own, to deliver near-term revenue growth. Celgene has said it expects more than $2 billion in incremental peak sales for Reblozyl (see “Bristol-Myers’ Next Phoenix Act?”).

An Acceleron Pharma Inc. (NASDAQ:XLRN) spokesperson told BioCentury a typical dose is 1 mg/kg and the wholesale acquisition cost (WAC) for a 75 mg vial will be $10,323.53. The companies will also sell Reblozyl in 25 mg vials at a proportionate price.

In the Phase III BELIEVE trial that supported approval, patients were dosed every 21 days for up to 48 weeks. Assuming a mass of 75 kg, or about 166 pounds, the 48-week regimen would cost $165,176.48.

Acceleron will co-promote Reblozyl with Celgene in North America. Celgene is responsible for commercialization in the rest of the world.

Gene therapy Zynteglo from bluebird bio Inc. (NASDAQ:BLUE) has conditional approval in Europe to treat transfusion-dependent β thalassemia. bluebird has previously told investors it expects to submit a BLA with FDA for the autologous CD34+ cell product encoding an engineered β globin (HBB) gene, which has breakthrough status designation, by year end.

A Celgene spokesperson said it is inappropriate to compare Reblozyl with Zynteglo as they have different mechanisms of action and have not been compared in clinical studies. The Celgene and Acceleron representatives did say, though, that Zynteglo requires a myeloablative bone marrow transplant and will be limited to a subset of patients. By contrast, the Acceleron spokesperson said Reblozyl could treat a broad spectrum of β thalassemia patients.

bluebird did not respond in time for comment.

Acceleron gained $2.67 to $43.28 on Friday.

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