bluebird bio, a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, has completed the NIH Recombinant DNA Advisory Committee's (RAC) public review of the HGB-208 pediatric study protocol for bluebird bio's LentiGlobin BB305 product candidate in beta-thalassemia major. The RAC recommendation was to delay initiation of the study in the U.S. for one to two years. This recommendation has no effect on the HGB-207 protocol plan.
"We appreciate the recommendations from the RAC members regarding the HGB-208 pediatric study protocol," said David Davidson, M.D., chief medical officer. "We will take the RAC feedback on the timing of initiating HGB-208 under advisement as we advance the clinical development of our LentiGlobin BB305 product candidate for patients with beta-thalassemia major. The HGB-207 trial protocol did not require further review by the RAC, and we will continue to work closely with the regulatory authorities and our clinical study sites to pursue appropriate accelerated regulatory approval pathways in the U.S. and the EU."
The outcome of RAC review is a series of recommendations and advice. RAC review does not constitute a formal approval of a proposed protocol. The recommendations are captured in a summary letter, which will be sent to the institutional review boards (IRBs) reviewing the protocol in the U.S. The recommendations are non-binding.
In May, bluebird bio announced that it had reached general agreement with the FDA on the design of its planned clinical trials HGB-207 and HGB-208. Based on its discussions with the FDA, bluebird bio believes that data from these trials, together with data from the ongoing beta-thalassemia major clinical studies (Northstar and HGB-205), could form the basis for a Biologics License Application (BLA) submission for LentiGlobin BB305. HGB-207 and HGB-208 share similar trial designs and are differentiated primarily by patient age. HGB-207 will enroll adult and adolescent patients; HGB-208 is planned to enroll pediatric patients.
The RAC had previously notified bluebird bio that only HGB-208 required a public RAC discussion.
bluebird bio also announced in May that it is one of the first companies to participate in the EMA’s Adaptive Pathways (formerly referred to as Adaptive Licensing) pilot program, which is part of the EMA's efforts to improve timely access for patients to new medicines. Based on several discussions involving the EMA, European Health Technology Assessment (HTA) agencies and patient advocacy organizations as part of this program, bluebird bio believes it is possible to seek conditional approval for the treatment of adults and adolescents with beta-thalassemia major on the basis of the totality of clinical data, in particular reduction in transfusion need, from the ongoing Northstar Study and supportive HGB-205 study. Conversion to full approval will be subject to the successful completion of the HGB-207 and HGB-208 clinical trials, supportive long-term follow-up data and "real-life" post-approval monitoring data.
bluebird bio has operations in Cambridge, Mass., Seattle, Washington, and Paris, France.