In-depth talks with US and EU regulators lead to accelerated approval strategy for gene therapy.

Bluebird Bio have met with the FDA and EMA to establish a clear pathway to regulatory approval for their LentiGlobin BB305 product candidate for the treatment of beta-thalassemia major. It seems that both agencies like Bluebird’s development plans, and may even result in accelerated approvals.

“We are very pleased with the outcome of these recent regulatory interactions,” commented David Davidson, M.D., 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 approval for 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.”

If trials are successful, this would give Bluebird a chance to win the first approval for gene therapy in the USA. With the $1 million price tag set for UniQure’s Glybera (currently the only approved gene therapy in Europe), there would still be some major unanswered questions on how much LentiGlobin is likely to cost.

Bluebird still need to generate clinical evidence for their gene therapy, but in having these productive meetings with regulators they have put themselves in a very strong position.

“We are grateful for the collaborative regulatory feedback from the FDA and EMA on the design of our pivotal studies, as well as feedback from the European HTA agencies and patient advocacy organizations that are participating in our Adaptive Pathways pilot project,” stated Anne-Virginie Eggimann, vice president of regulatory science. “We are looking forward to continuing our engagement with all of these stakeholders in the coming months to support the potential acceleration of the LentiGlobin BB305 program.”

For the breakdown of Bluebird’s next step, take a look at their official release.