bluebird bio’s Thalassemia gene therapy granted accelerated assessment by the EMA

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Bluebird bio have announced that its transfusion dependent β-Thalassemia (TDT) treatment, the investigational gene therapy LentiGlobin, has been granted an accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the EMA, for its upcoming marketing authorization application (MAA).

Bluebird bio have announced that its transfusion dependent β-Thalassemia (TDT) treatment, the investigational gene therapy LentiGlobin, has been granted an accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the EMA, for its upcoming marketing authorization application (MAA).

LentiGlobin is an investigational gene therapy developed for treating adult and adolescent patients suffering with TDT and a non-β0/β0 genotype. The company aim to file an MAA for LentiGlobin with the EMA later in 2018. Accelerated assessments can reduce the active review time of an MAA from 210 days to 150 days, pending validation by the EMA. They are granted to products which the CHMP consider to be of major interest for public health and are representative of therapeutic innovation.

LentiGlobin’s accelerated assessment approval is supported by data from clinical studies, which includes 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.

Previously, LentiGlobin has been granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation, and is part of the EMA’s Adaptive Pathways pilot program, aims to improve timely access for patients to new medicines. The FDA have also granted LentiGlobin Orphan Drug status and Breakthrough Therapy designation for the treatment 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.”

Source: bluebird bio’s LentiGlobin™ Gene Therapy Granted Accelerated Assessment by European Medicines Agency for the Treatment of Transfusion-Dependent β-Thalassemia Press Release