Bluebird gene therapy granted EMA accelerated assessment status
LentiGlobin is an autologous gene therapy candidate being developed by Cambridge, Massachusetts-based Bluebird Bio for the treatment of patients with transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease.
The firm announced last week the therapy has been accepted on the European Medicines Agency (EMA) PRIME programme, an accelerated assessment initiative.
PRIME was developed last year to offer companies support for candidates that show potential to benefit patients with unmet medical needs based on early clinical data, and benefits include the drug company having a point of contact within the agency dedicated to their candidate, and access to scientific advice at key development milestones.
“PRIME designation will allow bluebird bio to further improve our communication with European regulators as we continue to refine our evidence generation plan in the context of adaptive biomedical innovation,” said Bluebird’s chief medical officer David Davidson.
“Earlier this year we completed enrollment in the Northstar (HGB-204) global clinical study of LentiGlobin drug product in patients with TDT, which along with the supporting HGB-205 study, will form the basis of our eventual application for conditional approval in the EU under the Adaptive Pathways Pilot program.”
According to Bluebird, the candidate works by inserting a functional human beta-globin gene into a patient’s own hematopoietic stem cells ex vivo, before being infused back into the patient.
As with Bluebird’s other autologous gene therapy candidate Lenti-D (in development to stabilise or prevent progression of cerebral adrenoleukodystrophy), clinical batches of LentiGlobin is being made by Lonza.
The two firms struck a strategic partnership in June for Lonza to produce the gene therapies on a commercial scale, with the contract manufacturing organisation (CMO) providing capabilities from a production site in Houston, Texas, currently under construction.
