US FDA Approves Reblozyl (Luspatercept-aamt) To Rare Blood Disorder: Beta Thalassemia
Source: Thailand Medical News Nov 09, 2019 5 years, 1 week, 5 days, 17 hours, 34 minutes ago
The U.S. Food and Drug Administration has granted approval for
Reblozyl (
luspatercept-aamt) 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
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
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
eblozyl 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 to
Celegene Corporation.
.
