US FDA Approves Reblozyl (Luspatercept-aamt) To Rare Blood Disorder: Beta Thalassemia
The U.S. Food and Drug Administration has granted approval for Reblozyl
) to be used for the treatment of anemia (lack of red blood cells) in adult patients with beta thalassemia
who require regular red blood cell (RBC) transfusions.
Dr Richard Pazdur, M.D., director of the US FDA
's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the US FDA
's Center for Drug Evaluation and Research told Thailand Medical
News via a phone interview, "When patients receive multiple blood transfusions, there is a risk for iron overload, which can affect many organs. 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 blood disorder Beta thalassemia
, also called "Cooley's anemia," is an inherited blood disorder that reduces the 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.
The US FDA 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
Typically, 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 Reblozy
l. 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 US FDA
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 US FDA
granted this application Fast Track designation. R
also received Orphan Drug designation
, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted approval of Reblozyl