Luspatercept-aamt, marketed under the brand name REBLOZYL® (Celgene and Acceleron Pharma Inc.), has received a Priority Review designation from the U.S. Food and Drug Administration (FDA) for the treatment of anaemia in beta-thalassemia (see FDA press release here). This is exciting news for transfusion-dependent beta-thalassaemia patients as REBLOZYL®, for the first time, will help decrease the number of blood transfusions and consequent iron-induced dysfunction by decreasing the iron-loading burden. The approval for REBLOZYL® is based on the results of the BELIEVE trial, which showed that 21.4% patients treated with REBLOZYL® achieved at least 33% reduction in transfusions compared to 4.5% of 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 of the treatment included headache, bone pain, arthralgia (joint pain), fatigue, cough, abdominal pain, diarrhea and dizziness. REBLOZYL® 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. See Celgene press release here.