FDA approves first treatment of patients with rare blood diseases

recently, the U.S. Food and Drug Administration approved luspatercept-aamt treatment for adults who require routine red blood cell infusions β
“ When a patient receives multiple blood transfusions, there is a risk of iron overload, which affects many organs," said Dr. Richard Pazdur, director of the FDA Center of Excellence in Oncology and acting director of the U.S. Food and Drug Administration's Office of Oncology at the Center for Drug Evaluation and Research. This approval is an example of our continued progress in rare diseases and the early provision of important new drugs to patients. β Thalassemia, also known as Curley's anemia, is an inherited blood disorder that reduces the production of hemoglobin, an iron-containing protein in red blood cells that delivers oxygen to cells throughout the body. In people with β thalassemia, low levels of hemoglobin can lead to hypoxia and anemia in many parts of the body, which can lead to pale, weak, fatigue and more serious complications. Support β patients with thalassemia usually includes lifelong chronic blood transfusions to survive and treatment for iron overload caused by blood transfusions. People β with thalassemia also have an increased risk of abnormal blood clots.
Reblozyl's approval was based on clinical trial results in 336 patients with β thalassemia who required red blood cell infusion, 112 of whom received a placebo. Twenty-one percent of patients treated with Reblozyl had at least a 33 percent reduction in blood transfusions, compared with a 4.5 percent decrease in placebo patients. The reduction in blood transfusions means that patients need less blood transfusions during the 12 consecutive weeks of taking Reblozyl.
Common side effects of patients taking Ribozi include headache, bone pain, joint pain (joint pain), fatigue, cough, abdominal pain, diarrhea and dizziness. Patients may experience symptoms of high blood pressure when using Reblozyl. Health care professionals are advised to monitor the patient's blood pressure during treatment and, if necessary, to begin blood pressure reduction treatment. Patients treated with Reblozyl should be monitored for thrombosis (blood clots). The U.S. Food and Drug Administration recommends that health care professionals tell women of childbearing age to use effective methods of contraception during reboz treatment. Pregnant or lactating women should not take Reboz because it can cause harm to a developing fetus or newborn.The Food and Drug Administration approved a fast-track grant for the application.
Reblozyl was also awarded the title of Orphan Drug and provided incentives to assist and encourage drug development for rare diseases.
U.S. Food and Drug Administration approved Searle Genetics' new drug, Reblozyl. （cyy123.com）