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.

The Cooley’s Anemia Foundation is accepting applications for medical research grants and fellowships in areas related to thalassaemia. The awards are in 3 categories:

1. Support for Ongoing Clinical Research in Thalassaemia (Deadline: December 20, 2019 for a letter of intent and February 3, 2020 for invited full applications).

2. Clinical Trials in Thalassaemia Cell and Gene Therapy Deadline: December 20, 2019 for a letter of intent and February 3, 2020 for invited full applications).

3. Research Fellowships  (Deadline: February 3, 2020).

For a description of each award category and further information on the criteria for each, please click here.

The European reference network for rare hematological disorders (ERN-EuroBloodNet) recently published its second report on all the activities performed and results achieved in its second year of implementation, also putting forward a number of suggestions for the future actions of the network. Deliverables submitted to the European Commission are available here.

The European Commission granted conditional marketing authorization to Bluebird Bio's Lentiglobin BB305 gene therapy (ZYNTEGLOTM) for transfusion-dependent β-thalassaemia (TDT). ZYNTEGLOTM is a one-time gene therapy (autologous CD34+ cells encoding βA-T87Q-globin gene) for a specific genotype of TDT, cleared for use in patients aged 12 and older who are able to receive a stem cell transplant but have no matched donor. These are exciting news as ZYNTEGLOTM is the first gene therapy to gain regulatory approval for TDT in the European Union, offering for the first time the possibility of a transfusion-free future and a better quality of life to severely-affected patients. The company will offer the therapy first in Germany, followed by Italy, the U.K. and France, and hopes to launch ZYNTEGLOTM in the U.S. next year.

More information: Bluebird Bio press releaseEMA Medicine Report

A marketing authorization application (MAA) has been submitted to the European Medicines Agency (EMA) for luspatercept for the treatment of anaemia related to myelodysplastic syndromes and β-thalassaemia. Luspatercept (Celgene and  Acceleron Pharma Inc.) is a first-in-class erythroid maturation agent that is designed to regulate late-stage red blood cell maturation. The MAA for the agent is for adult patients with β-thalassemia-associated anaemia who require blood transfusions. In the phase 3 BELIEVE clinical trial, adult β-thalassaemia patients randomized to the luspatercept arm had a statistically and clinically meaningful reduction in transfusion burden during any 12 or 24 weeks in the study period with minor side effects, according to data presented at ASH 2018. A Phase 2 trial in non-transfusion-dependent β-thalassemia (BEYOND) is ongoing. See Celgene's Press Release here.