Posted by Michael Wonder on 27 Jul 2018
bluebird bio’s LentiGlobin gene therapy granted accelerated assessment by EMA for the treatment of transfusion-dependent β-thalassemia
26 July 2018 - Company on track to submit marketing authorisation application in European Union in 2018.
bluebird bio today announced that its investigational LentiGlobin™ gene therapy for the treatment of adolescent and adult patients with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype, was granted an accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), for its upcoming marketing authorization application (MAA). LentiGlobin is a potential one-time gene therapy that may address the underlying genetic cause of TDT.
“Transfusion-dependent β-thalassemia is a severe genetic disease that requires a lifetime of chronic blood transfusions for survival, and while these transfusions are life-saving, they are also associated with serious medical complications such as organ failure from iron overload,” said David Davidson, M.D., chief medical officer, bluebird bio. “Receiving accelerated assessment for LentiGlobin helps support our goal of delivering the first gene therapy to patients with TDT. We look forward to working in collaboration with the regulatory authorities on this potentially transformative treatment option.”
bluebird bio intends to file an MAA for LentiGlobin in TDT with the EMA in 2018. Accelerated assessments can reduce the active review time of an MAA from 210 days to 150 days once it has been validated by the EMA. An accelerated assessment is granted to products deemed by the CHMP to be of major interest for public health and represent therapeutic innovation.
The accelerated assessment for LentiGlobin is supported by data from clinical studies, including the completed Phase 1/2 Northstar (HGB-204) study, the ongoing Phase 1/2 HGB-205 study as well as available data from the Phase 3 Northstar-2 (HGB-207) study and the long-term follow-up study LTF-303.
The EMA previously granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT. LentiGlobin is also part of the EMA’s Adaptive Pathways pilot program, which is part of the EMA’s effort to improve timely access for patients to new medicines.
The U.S. Food and Drug Administration (FDA) also granted LentiGlobin Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.
