European Commission Grants Conditional Marketing Authorization for Zynteglo

June 6, 2019

Gene therapy development company, bluebird bio, has revealed that the European Commission has granted conditional marketing authorization for Zynteglo.

Gene therapy development company, bluebird bio, has revealed that the European Commission (EC) has granted conditional marketing authorization for Zynteglo, its gene therapy for patients aged 12 years and older with transfusion-dependent β-thalassemia (TDT).

Zynteglo is a one-time gene therapy indicated for patients of 12 years and older with TDT that do not have a β0genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. Its conditional authorization decision was supported by efficacy, safety, and durability data from the Phase I/II HGB-205 study and the completed Phase I/II Northstar (HGB-204) study, in addition to ongoing Phase III studies and long-term follow-up study.

“EC approval of Zynteglo is a milestone that represents the dedication and commitment of clinical investigators, healthcare providers, patients and their families, and our employees, all of whom have helped advance this treatment from concept to an approved therapy,” said Nick Leschly, CEO of bluebird, in a press release. “Our first product approval is a humbling moment for all of us at bluebird, and we look forward to continuing our work with the TDT community and health systems to bring this important treatment to patients.”

“As one of the investigators in the clinical studies of Zynteglo, I have witnessed firsthand the hope this gene therapy can provide to patients and their families who have often been managing this disease and transfusions for years, often for decades,” added Franco Locatelli, MD, PhD, Professor of Pediatrics, Sapienza University of Rome, Italy, and director, Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy. “This approval by the EC means we now have a gene therapy for certain patients with TDT that has the potential to transform lives by offering the possibility of a transfusion-free future.”

Zynteglo was reviewed as part of the European Medicines Agency’s Priority Medicines program. The gene therapy is the first to gain regulatory approval for TDT and is the first approved gene therapy for bluebird bio.

Source: bluebird bio