On 5 October, the European Medicines Agency (EMA) accepted bluebird bio's market authorization application (MAA) for its gene therapy LentiGlobin™ for the treatment of adolescents and adults with transfusion-dependent ß-thalassaemia. The manufacturing partner will be apceth Biopharma GmbH from Ottobrunn/Munich.
Transfusion-dependent ß-thalassemia is a hereditary blood disease caused by a mutation in the beta-globin gene. Patients suffer from ineffective red blood cell production and the resulting anaemia. The symptomatic treatment of people with TDT consists of lifelong blood transfusions. However, transfusion therapy and the increased uptake of iron caused by the disease lead to iron deposition in various organs, which can lead to life-threatening complications. Therefore, the administration of iron-binding medication is also part of the treatment.
“We congratulate our partner bluebird bio on this recent success”, commented Dr Christine Guenther, CEO of apceth Biopharma. “These are exciting times also for apceth, as we are now in the position to become one of the very few companies worldwide to manufacture a cell-based gene therapy for commercial use.” apceth has supported and will continue to support bluebird bio for obtaining the market authorization for LentiGlobin™.
“People living with transfusion-dependent β-thalassemia require frequent blood transfusions that are life-saving but may lead to complications, including organ failure due to iron overload,” said David Davidson, M.D., chief medical officer, bluebird bio. “The acceptance of our marketing authorization application for LentiGlobin is a milestone that advances us toward our goal of providing to patients the first one-time gene therapy that addresses the underlying genetic cause of TDT. We share this important milestone with the patients, families and healthcare providers who made it possible through their participation in our pioneering clinical studies of LentiGlobin.”
Already in 2016, apceth Biopharma and bluebird bio had signed a manufacturing agreement for commercial products. apceth Biopharma is the clinical and commercial manufacturing partner of bluebird bio in Europe for LentiGlobin™. As a pioneer in the field of cell therapy and regenerative medicine, apceth Biopharma is a certified partner for the development and production of cell and gene therapies for various customers. The company also develops its own drug candidates for the treatment of inflammation, autoimmune diseases and solid tumors.
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