bluebird bio’s first gene therapy gains regulatory authorisation

New insight into microRNA function has been published by scientists at the University of Eastern Finland and the University of Oxford.

bluebird bio’s first gene therapy gains regulatory authorisation. bluebird bio, Inc has announced that the European Commission (EC) has granted conditional marketing authorisation for Zynteglo™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β00 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. Payer agencies will now begin the country-by-country reimbursement process to help support access to the therapy for appropriate patients.

TDT is a severe genetic condition caused by mutations in the β-globin gene that result in reduced or absent haemoglobin. In order to survive, people with TDT maintain haemoglobin levels through life-long chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload. This one-time gene therapy addresses the underlying genetic cause of TDT and offers patients 12 years and older who do not have a β00 genotype the potential to become transfusion independent, which is expected to be life-long once achieved.

The conditional marketing authorisation is supported by efficacy, safety and durability data from the completed Phase 1/2 HGB-205 study and Phase 1/2 Northstar (HGB-204) study as well as available data from the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies, and the long-term follow-up study LTF-303, as of the data cut-off of 13 December 2018.

The treatment continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies and the long-term follow-up study LTF-303.

In addition to Priority Medicines (PRIME) designation, this therapy received an Orphan Medicinal Product designation from the EC for the treatment of β-thalassaemia intermedia and major, which includes TDT. The evaluation took place via the European Medicines Agency’s (EMA) Priority Medicines (PRIME) and Adaptive Pathways programmes, which support medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. The PRIME and Adaptive Pathway programmes allowed for early and enhanced dialogue and accelerated assessment, which was completed on the shortest timetable for an advanced therapy medicinal product (ATMP) by the EMA to date.

The conditional marketing authorisation is valid in all 28-member states of the EU, as well as Iceland, Liechtenstein and Norway.