Bluebird Bio Inc. (NASDAQ:BLUE) has announced that it has received eligibility from the European Medicines Agency to the Priority Medicines program (PRIME) for its experimental sickle cell disease treatment, LentiGlobin™ for sickle cell gene therapy.
PRIME eligibility to help Bluebird accelerate LentiGlobin development
This is a huge milestone because the PRIME program offers more support and enhanced interaction between the company and the agency. The aim is to optimize development plans and accelerate the regulatory process to potentially deliver novel treatments or therapies to patients as soon as possible. For a company to receive PRIME eligibility, a therapy they are advancing has to show potential benefits through clinical data for patients with an unmet medical need. Bluebird Bio’s PRIME application for the sickle cell disease gene therapy was supported with Phase 1/2 HGB-205 clinical study data, the ongoing efficacy and safety follow-up LTF-303 study, and the continuing Phase 1/2 HGB-206 study.
Anne-Virginie Eggimann, the company’s SVP Regulatory Science, stated that there is still a profound unmet medical need for sickle cell disease patients, even with recent advances to deliver new treatments. Eggimann explained that SCD is a progressive disease that mostly results in organ damage and even early death.
Designation clears a regulatory pathway for expedited development
Based on Bluebird Bio’s results from clinical trials, LentiGlobin’s treatment for SCD has demonstrated the potential of offering significant outcomes to patients having SCD. Eggimann affirmed that the PRIME designation grant is evidence that the EMA acknowledges the significance of delivering novel medicine to SCD patients efficiently. She added that the designation will allow the company to work closely with EMA in helping accelerate the development and review process of LentiGlobin™ for SCD patients.
SCD is a hereditary disease that results from mutation of the β-globin gene, leading to the production of unusual sickle hemoglobin and thus affects red blood cells. It makes red blood cells sickled and fragile, leading to hemolytic anemia, painful VOCs, and vasculopathy. LentiGlobin has been designed to add more functional copies of the mutated β-globin gene into the hematopoietic stem cells.