The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months. Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free! STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.
Bluebird Bio has moved a step closer to receiving European approval for the first gene therapy designed to treat beta-thalassemia. The company's treatment, Lentiglobin, has been recommended for approval by the the European Medicines Agency's Committee for Medicinal Products for Human Use, the drug-reviewing arm of EU. Lentiglobin works by using an inactivated virus to insert a healthy copy of the beta-globin gene into a patient’s bone marrow, producing red blood cells and reducing the need for transfusions. A final decision on whether to approve the treatment is expected within the next three months.