Voxelotor, marketed under the brand name Oxbryta™ (Global Blood Therapeutics Inc., GBT), has received Accelerated Approval from the U.S. Food and Drug Administration (FDA) for the treatment of adults and pediatric patients aged 12 years and older with sickle cell disease (SCD). Oxbryta™ is a once-daily pill that works by directly inhibiting sickle haemoglobin (Hb) polymerization, the main cause of SCD. The drug’s approval came three months earlier than expected and is based on results from the randomized, global Phase 3 HOPE trial (NCT03036813). Trial findings were published in The New England Journal of Medicine, showing clinically meaningful and statistically significant improvements in Hb levels, accompanied by reductions in red blood cell destruction (hemolysis). Briefly, 51.1% of patients receiving Oxbryta™ for 24 weeks at 1500 mg daily, its now approved dose, achieved a greater than 1 g/dL increase in Hb compared with 6.5% receiving placebo (n=274, P<0.001). Common side effects for patients taking Oxbryta™ were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever). In spite FDA Fast Tract designation, further clinical trials are required to verify and describe the drug's clinical benefit. Oxbryta™ also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. For more information, read GBT press release here and FDA press release here

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 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

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.