FDA Approves Reblozyl First-in-Class Treatment for Beta Thalassemia

The US Food and Drug Administration (FDA) has approved Celgene and Acceleron’s Reblozyl (luspatercept-aamt) for the treatment of anemia in patients with beta thalassemia. This is the first of its kind to treat patients with the rare blood disorder.

According to Genetics Home Reference, beta thalassemia is an inherited blood disorder that reduces the production of hemoglobin, which is the iron-containing protein in red blood cells that allows the cell to carry oxygen around the body. In affected people, low levels of hemoglobin are present in the blood which leads to a lack of oxygen in many parts of the body. This shortage causes pale skin, weakness, fatigue and many more serious complications.

One traditional way of helping those with beta thalassemia is by providing them with lifelong regimens of blood transfusions as treatment. However, one must consider that people with beta thalassemia have an increased risk of developing abnormal blood clots as well as iron overload as a result.

“When patients receive multiple blood transfusions, there is a risk for iron overload, which can affect many organs,” said Dr. Richard Pazdur, 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.

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Reblozyl is a first-in-class erythroid maturation agent (EMA) for the treatment of transfusion dependent beta thalassemia associated anemia. According to a press release, the approval was based on 336 patients in a clinical trial. These patients have beta thalassemia and require RBC transfusions. 112 of those patients received placebo. When the results were gathered, they recognized that 21 percent of patients that received Reblozyl achieved at least 33 percent reduction in transfusions compared to 4.5 percent of patients who received the placebo. This means that the patients with reduced transfusions required less transfusions over 12 consecutive weeks while taking Reblozyl.

“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,” said Dr. Richard Pazdur.

Reblozyl is going to be available one week following the FDA approval. However, like most drugs, there are a few common side effects that one should be cautious of. These side effects include, headaches, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea and dizziness. The press release states that “health care professionals are advised to monitor a patient’s blood pressure during treatment and to initiate anti-hypertensive treatment if necessary. Patients who receive Reblozyl should be monitored for thrombosis (blood clots).”