Bluebird's LentiGlobin gene therapy is granted PRIME status by EMA for sickle cell disease

Bluebird's LentiGlobinTM for sickle cell disease (SCD) gene therapy (bb1111) was granted eligibility to the Priority Medicines (PRIME) program by the European Medicines Agency (EMA). LentiGlobinTM is an investigational therapy. PRIME provides early and enhanced scientific and regulatory support to medicines that have the potential to significantly address patients’ unmet medical needs. Clinical data from the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study and ongoing long-term safety and efficacy follow-up study LTF-303 supported the PRIME application for LentiGlobinTM for SCD. The U.S. FDA granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobinTM for SCD. LentiGlobinTM for SCD also received orphan medicinal product designation from the European Commission for the treatment of SCD. For more information, see here.