The European Medicines Agency will undertake an accelerated review of bluebird bio’s LentiGlobin, a gene therapy for the treatment of transfusion-dependent beta-thalassemia.
TDBT is a rare inherited blood disorder caused by one of over 200 mutations in the beta-globin gene, characterised by failure to grow and gain weight, infection, and life-threatening anemia, which occur within the first two years of life.
The only curative approach thus far is allogeneic haematopoietic stem cell transplantation, which is linked with significant transplant-related morbidity and mortality.
LentiGlobin is a one-time gene therapy being studied as a potential treatment to address the underlying genetic cause of TDT, which could eliminate or reduce the need for blood transfusions.
“People living with transfusion-dependent beta-thalassemia require frequent blood transfusions that are life-saving but may lead to complications, including organ failure due to iron overload,” said David Davidson, chief medical officer, bluebird bio.
“The acceptance of our marketing authorisation 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.”
The news came as the European Medicines Agency also assigned Orchard Therapeutics’ hematopoietic stem cell gene therapy PRIME designation, giving the firm a pass to closer engagement with the regulator to optimise development and speed up the review timeline.