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FDA grants breakthrough therapy designation to LentiGlobin for beta-thalassemia major
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The FDA granted breakthrough therapy designation to the LentiGlobin gene therapy product for the treatment of transfusion-dependent patients with beta-thalassemia major, according to a press release from the therapy’s manufacturer.
LentiGlobin (BB305, bluebird bio) — designed to treat beta-thalassemia major and severe sickle cell disease — inserts a functional human beta-globin gene into the patients’ hematopoietic cells ex vivo. The patient then receives the modified cells through an autologous stem cell transplantation.
The FDA based its decision in part on data from two ongoing phase 1/phase 2 trials designed to evaluate LentiGlobin in patients with beta-thalassemia major and sickle cell disease. The first four patients treated — all of whom required chronic blood transfusions at baseline —demonstrated sufficient hemoglobin production to reduce or eliminate the need for transfusion support after at least 3 months of follow-up after receiving the therapy.
"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," David Davidson, MD, chief medical officer of bluebird bio, said in a press release. "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."