BlueBird Bio Inc. Drug Might Get Accelerated OK In U.S., Europe

Bluebird Bio Inc.

fluttered up nearly 5 percent Tuesday on news that approvals for its LentiGlobin for treating a rare blood disorder might get accelerated. http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2049713 The Cambridge, Massachusetts company said it reached agreement with U.S. regulators on two 24-month clinical trials that, together with current studies, could form the basis of approval for LentiGlobin as a treatment for beta-thalassemia major, a rare and potentially fatal drug disorder. Beta-thalassemia affects about 15,000 patients in the U.S., but LentiGlobin is also under separate study as a treatment for sickle cell disease, which affects up to about 100,000 Americans. In addition, a pilot program of the European Medical Agency may allow conditional approval of the drug as a beta-thalassemia treatment on the basis of results from current studies, the company said. Protocol for one of the pending U.S. studies is slated for review by regulators June 9. Protocol for the second study doesn't require review or discussion, under a decision by a committee of the National Institutes of Health, Bluebird said. "These discussions have resulted in general agreement from both agencies regarding bluebird bio's development plans, which could potentially result in accelerated approvals," David Davidson, Bluebird's chief medical officer said in a statement Tuesday. LentiGlobin obtained "breakthrough" designation from the Food and Drug Administration in February. The drug treatment entails inserting a functional human blood-related gene into the patient's own stem cells and then transplanting those modified cells into the patient through the bloodstream. Bluebird, which held a conference call with investors about the news earlier, traded recently at $164.05, up $7.65.