Celgene nabs FDA approval for blockbuster hopeful anemia med Reblozyl

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Celgene's Reblozyl nabbed an FDA approval to treat anemia Friday. (Celgene)

Bristol-Myers Squibb's $74 billion pending pickup of Celgene has notched a win already with an FDA approval for JAK med Inrebic. Now, the consummation has grabbed another victory with an approval for a Celgene blood disease candidate––and it could spell blockbuster sales ahead.

The FDA Friday approved Celgene's Reblozyl (luspatercept), a novel erythroid maturation agent used to treat anemia in patients with beta thalassemia who require regular red blood cell transfusions. Though these transfusions are a lifesaving treatment, they can eventually lead to iron overload that can cause organ failure and shorten a patient’s life span. 

Reblozyl, co-developed with Cambridge, Massachusetts-based biotech Acceleron, is now the first FDA-approved treatment for anemia in beta thalassemia. The drug is expected to be ready for sale in one week, Celgene said. 

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The FDA's nod was based on phase 3 data showing Reblozyl significantly increased the number of patients who required fewer blood transfusions over placebo in a 24-week period.

Celgene's drug will come with label warnings for thrombosis, or blood clots, hypertension and embryo-fetal toxicity, the company said. Serious adverse effects, including cerebrovascular accident and deep vein thrombosis, were reported in 1% of trial patients, and one patient reportedly died during clinical testing due to an unconfirmed case of acute myeloid leukemia, Celgene said.

RELATED: In a much-needed positive for BMS, Celgene gains FDA review for blockbuster hopeful

Reblozyl comes with blockbuster hopes, with Jefferies analysts forecasting $2 billion in peak annual sales for the drug if it gets a positive nod from the FDA in April to treat myelodysplastic syndromes.

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