FDA approves first treatment for anemia in thalessemia patients

The U.S. Food and Drugs Administration has granted approval to Reblozyl (luspatercept–aamt) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.

REBLOZYL is a first-in-class erythroid maturation agent that promotes late-stage red blood cell maturation in animal models.REBLOZYL is indicated for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.

The Food and Drugs Administration has granted approval of Reblozyl to Celegene Corporation.

Beta thalassemia, an inherited blood disorder is also known as “Cooley’s anemia”.In thalessemia there is reduced production of hemoglobin, an iron-containing protein in red blood cells that carries oxygen to cells throughout the body. In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body and anemia, which can cause pale skin, weakness, fatigue and more serious complications. Supportive treatment for people with beta thalassemia often consists of lifelong regimens of chronic blood transfusions for survival and treatment for iron overload due to the transfusions. People with beta thalassemia are also at an increased risk of developing abnormal blood clots.

“When patients receive multiple blood transfusions, there is a risk for iron overload, which can affect many organs,” said Richard Pazdur, M.D., 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. “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 approval of Reblozyl was based on the results of a clinical trial of 336 patients with beta thalassemia who required RBC transfusions, of which 112 received a placebo. Twenty-one percent of the patients who received Reblozyl achieved at least a 33% reduction in transfusions compared to 4.5% of the patients who received a placebo. The transfusion reduction meant that the patient needed fewer transfusions over 12 consecutive weeks while taking Reblozyl.

Common side effects for patients taking Reblozyl were headache, bone pain, arthralgia (joint pain), fatigue, cough, abdominal pain, diarrhea and dizziness. Patients may experience hypertension while using Reblozyl. 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). The Food and Drugs Administration advises health care professionals to tell females of reproductive age to use effective contraception during treatment with Reblozyl. Women who are pregnant or breastfeeding should not take Reblozyl because it may cause harm to a developing fetus or newborn baby.

The FDA granted this application Fast Track designation. Reblozyl also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.