CAMBRIDGE, MA, USA I February 2, 2015 I bluebird bio, Inc. (Nasdaq:BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to LentiGlobin® BB305 Drug Product for the treatment of transfusion-dependent patients with beta-thalassemia major.
LentiGlobin BB305 Drug Product aims to treat beta-thalassemia major and severe sickle cell disease by inserting a functional human beta-globin gene into the patient’s own hematopoietic stem cells ex vivo and then returning those modified cells to the patient through an autologous stem cell transplantation.
“The FDA’s Breakthrough designation of LentiGlobin highlights that new therapies are needed for the treatment of patients with beta-thalassemia major, especially treatments with the potential to meaningfully reduce or liberate patients from transfusion dependence,” said David Davidson, M.D., chief medical officer of bluebird bio. “Our early clinical data investigating the use of LentiGlobin in patients with multiple genotypes of beta-thalassemia major, including beta-0/beta-0, the most severe genotype, are very encouraging, and we remain on track to complete enrollment in the Northstar and HGB-205 studies in 2015. In light of the Breakthrough designation, we look forward to working even more closely with the FDA to expedite the development of LentiGlobin for the treatment of beta-thalassemia major.”
The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned for use to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The benefits of Breakthrough Therapy designation include the same benefits as Fast Track designation, plus an organizational commitment involving FDA’s senior managers with more intensive guidance from the FDA. Breakthrough Therapy designation does not however change the standards for approval.
The Breakthrough Therapy designation is supported by data from the ongoing Phase 1/2 Northstar (HGB-204) and HGB-205 studies of LentiGlobin. Findings in eight subjects with beta-thalassemia major were presented at the 56th Annual Meeting of the American Society of Hematology (ASH) in December 2014. In the first four subjects, each of whom had at least three months of follow up, treatment resulted in sufficient hemoglobin production to reduce or eliminate the need for transfusion support among patients with beta-thalassemia major who would otherwise require chronic blood transfusions. These data consisted of the first five subjects treated in bluebird bio’s ongoing Northstar Study and the first three subjects from its HGB-205 study. These included the first beta-thalassemia subjects with the beta-0/beta-0 genotype to be treated with LentiGlobin BB305 drug product. The HGB-205 study also included the first subject with sickle cell disease to be treated with gene therapy.
About bluebird bio, Inc.
With its lentiviral-based gene therapy and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and T cell-based immunotherapy. bluebird bio’s clinical programs include Lenti-D™, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy, and LentiGlobin®, currently in three clinical studies: a global Phase 1/2 study, called the Northstar Study, for the treatment of beta-thalassemia major; a single-center Phase 1/2 study in France (HGB-205) for the treatment of beta-thalassemia major or severe sickle cell disease; and a separate U.S. Phase 1 study for the treatment of sickle cell disease (HGB-206). bluebird bio also has a preclinical CAR T cancer immunotherapy program in collaboration with Celgene Corporation, as well as discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies.
SOURCE: bluebird bio
Post Views: 224
CAMBRIDGE, MA, USA I February 2, 2015 I bluebird bio, Inc. (Nasdaq:BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to LentiGlobin® BB305 Drug Product for the treatment of transfusion-dependent patients with beta-thalassemia major.
LentiGlobin BB305 Drug Product aims to treat beta-thalassemia major and severe sickle cell disease by inserting a functional human beta-globin gene into the patient’s own hematopoietic stem cells ex vivo and then returning those modified cells to the patient through an autologous stem cell transplantation.
“The FDA’s Breakthrough designation of LentiGlobin highlights that new therapies are needed for the treatment of patients with beta-thalassemia major, especially treatments with the potential to meaningfully reduce or liberate patients from transfusion dependence,” said David Davidson, M.D., chief medical officer of bluebird bio. “Our early clinical data investigating the use of LentiGlobin in patients with multiple genotypes of beta-thalassemia major, including beta-0/beta-0, the most severe genotype, are very encouraging, and we remain on track to complete enrollment in the Northstar and HGB-205 studies in 2015. In light of the Breakthrough designation, we look forward to working even more closely with the FDA to expedite the development of LentiGlobin for the treatment of beta-thalassemia major.”
The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned for use to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The benefits of Breakthrough Therapy designation include the same benefits as Fast Track designation, plus an organizational commitment involving FDA’s senior managers with more intensive guidance from the FDA. Breakthrough Therapy designation does not however change the standards for approval.
The Breakthrough Therapy designation is supported by data from the ongoing Phase 1/2 Northstar (HGB-204) and HGB-205 studies of LentiGlobin. Findings in eight subjects with beta-thalassemia major were presented at the 56th Annual Meeting of the American Society of Hematology (ASH) in December 2014. In the first four subjects, each of whom had at least three months of follow up, treatment resulted in sufficient hemoglobin production to reduce or eliminate the need for transfusion support among patients with beta-thalassemia major who would otherwise require chronic blood transfusions. These data consisted of the first five subjects treated in bluebird bio’s ongoing Northstar Study and the first three subjects from its HGB-205 study. These included the first beta-thalassemia subjects with the beta-0/beta-0 genotype to be treated with LentiGlobin BB305 drug product. The HGB-205 study also included the first subject with sickle cell disease to be treated with gene therapy.
About bluebird bio, Inc.
With its lentiviral-based gene therapy and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and T cell-based immunotherapy. bluebird bio’s clinical programs include Lenti-D™, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy, and LentiGlobin®, currently in three clinical studies: a global Phase 1/2 study, called the Northstar Study, for the treatment of beta-thalassemia major; a single-center Phase 1/2 study in France (HGB-205) for the treatment of beta-thalassemia major or severe sickle cell disease; and a separate U.S. Phase 1 study for the treatment of sickle cell disease (HGB-206). bluebird bio also has a preclinical CAR T cancer immunotherapy program in collaboration with Celgene Corporation, as well as discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies.
SOURCE: bluebird bio
Post Views: 224
