bluebird bio plots regulatory path for LentiGlobin

US clinical-stage biotech firm bluebird bio (Nasdaq: BLUE) says that it has met with regulatory authorities in Europe and the USA to discuss potential approval pathways for its LentiGlobin BB305 product candidate for the treatment of beta-thalassemia major.

These discussions have resulted in general agreement from both agencies regarding bluebird bio’s development plans, which could potentially result in accelerated approvals.

“We are very pleased with the outcome of these recent regulatory interactions,” commented David Davidson, chief medical officer. “We look forward to advancing our beta-thalassemia major program based on data both from our ongoing studies as well as two planned open-label studies with a sample size of 15 patients each. The EMA Adaptive Pathways pilot program will allow us to pursue conditional approvalfor the treatment of beta-thalassemia major on the basis of clinical data from our ongoing HGB-204 and HGB-205 studies. This feedback brings us closer to achieving our vision of delivering one-time, potentially transformative gene therapy to patients,” he added.