The U.S. Food and Drug Administration has approved Reblozyl (luspatercept–aamt) for the cure of anemia (deficiency of red blood cells) in adult patients with beta-thalassemia. These patients need regular transfusions of red blood cells (RBCs).
Richard Pazdur is an acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. Moreover, he is also working as an M.D. and director of the FDA’s Oncology Center of Excellence.
He said that when a patient receives multiple blood transfusions, there are chances of iron overload. That in return can affect many organs of the body. FDA’s approval provides patients with the first therapy, that will help in reducing the number of blood transfusions.
This approval shows continued progress in the field of rare diseases like beta-thalassemia and ensures the earlier provision of important new drugs to patients.
Beta thalassemia, also known as “Cooley’s anemia,” is one of the genetic blood disorders. It causes an overall reduction in the process of hemoglobin (Hb) synthesis. That is an iron-containing protein in RBCs responsible for carrying oxygen to cells to the whole body.
The patients of beta-thalassemia experience low levels of Hb which decreases the oxygen supply for major parts of the body, this way it leads to anemia. Anemia is a disese in which a patient feels extreme weakness, fatigue, paleness of skin, and other serious complications.
Supportive therapy for patients with beta-thalassemia often comprises of lifelong regimens of chronic blood transfusions. That is necessary for the survival and treatment of iron overload due to the transfusions.
Additionally, patients suffering from beta-thalassemia have also an increased risk of developing abnormal blood clots.
The Reblozyl approval by FDA is based on the findings of a clinical trial comprising of 336 people with beta-thalassemia who required RBC transfusions. Among these people, 112 were receiving a placebo.
21% of the patients who received Reblozyl showed at least a 33% reduction in transfusions compared to 4.5 percent of the patients who received a placebo. This transfusion reduction means that the patients required fewer transfusions during 12 consecutive weeks while taking Reblozyl.
However, some side effects were also observed among patients taking Reblozyl. Some of the common side effects were fatigue, headache, cough, abdominal pain, diarrhea, bone pain, arthralgia (joint pain), and dizziness.
Patients may also experience hypertension while they use Reblozyl. Health care providers are suggested to monitor a patient’s blood pressure during therapy and to begin anti-hypertensive treatment if required.
Moreover, patients taking Reblozyl should also be checked for thrombosis (blood clots). The FDA suggests health care providers advise females of reproductive age to use effective contraception while receiving Reblozyl.
Also, women who are breastfeeding or pregnant should not take Reblozyl because it may harm the developing fetus or newborn baby.
This application has received Fast Track designation by FDA. Reblozyl has also received Orphan Drug designation, which offers incentives to encourage and assist the development of drugs for rare diseases. The FDA has approved this therapy to Celgene Corporation.