The FDA today approved luspatercept-aamt to treat anemia in adults with beta-thalassemia who undergo regular red blood cell transfusions.
Beta-thalassemia — a rare, inherited blood disorder — decreases the production of hemoglobin, which can lead to a lack of oxygen in different parts of the body, anemia and abnormal blood clotting.
Standard treatment for beta-thalassemia includes regular blood transfusions. Patients are often then treated for iron overload because of the transfusions.
“When patients receive multiple blood transfusions, there is a risk for iron overload, which can affect many organs,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the office of oncologic diseases in the FDA’s Center for Drug Evaluation and Research, said in an agency-issued press release. “Today’s approval provides patients with a therapy that, for the first time, will help decrease the number of blood transfusions. This approval is an example of our continued progress for rare diseases and providing important new drugs to patients earlier.”
The FDA based this approval, in part, on a study that showed luspatercept-aamt (Reblozyl; Acceleron Pharma, Celgene) reduced the number of transfusions patients needed over 12 consecutive weeks.
Researchers randomly assigned 336 patients with beta-thalassemia who needed regular transfusions to receive luspatercept-aamt (n = 224) or placebo (n = 112).
As HemOnc Today previously reported, results showed that 21% of patients in the luspatercept-aamt group achieved at least a 33% reduction in transfusions over weeks 13 to 24 compared with 4.5% patients in the placebo group.
The study also met its secondary endpoints of a 33% or greater reduction in red blood cell transfusion burden at weeks 37 to 48 (19.6% vs. 3.6%; P < .0001), and a 50% or greater reduction in transfusion burden at weeks 13 to 24 (7.6% vs. 1.8%; P = .0303) and at weeks 37 to 48 (10.3% vs. 0.9%; P = .0017).
Common adverse events associated with luspatercept-aamt included headache, bone pain, joint pain, fatigue, cough, abdominal pain, diarrhea and dizziness.
“Today’s approval is an important milestone and underscores our continued commitment to patients with hematology disorders,” Nadim Ahmed, president of global hematology and oncology for Celgene, said in a company-issued press release. “There are very limited options for patients living with anemia 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 anemia, a significant clinical complication of beta-thalassemia.”