- Based on meetings with regulatory authorities in the U.S. and Europe, bluebird bio (NASDAQ:BLUE) clarifies its regulatory strategy for the approval of LentiGlobin BB305 for the treatment of beta thalassemia major.
- In the U.S., the Biologics License Application (BLA) will be supported by the results from four clinical trials: Northstar and HGB-205, both of which are in process, and HGB-207 (adults and adolescents) and HGB-208 (pediatric), both of which are planned. The latter two trials will each enroll 15 patients with a follow-up period of 24 months. The primary endpoint will be 12 months of transfusion independence. The FDA designated LentiGlobin BB305 a Breakthrough Therapy in February. This provides for more intensive guidance from the agency on an approval path, the involvement of more senior personnel and a rolling review of the BLA.
- In Europe, approval will be pursued via the European Medicines Agency's Adaptive Pathways pilot program which will allow conditional approval based, in large part, on the results from the Northstar and HGB-205 studies. Full approval will be based on the results from HGB-207 and HGB-208, supportive long-term follow-up data and "real-life" post-approval monitoring data.
- The company will host a conference call this morning at 8:00 am ET for investors.