Research & Development
European Medicines Grants Priority Medicines Designation to bluebird bio's LentiGlobin for Sickle Cell Disease Gene Therapy
23 September 2020 - - US-based gene therapy company bluebird bio, Inc's (NASDAQ: BLUE) investigational treatment for sickle cell disease, LentiGlobin for SCD gene therapy (bb1111), was granted eligibility to the Priority Medicines (PRIME) program by the European Medicines Agency, the company said.

The EMA's PRIME initiative provides enhanced support and increased interaction to companies, with the goal of optimizing development plans and speeding regulatory evaluations to potentially bring innovative medicines to patients more quickly.

To be accepted for PRIME, a therapy must demonstrate potential to benefit patients with unmet medical need through early clinical data.

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 LentiGlobin for SCD.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the β-globin gene that leads to the production of abnormal sickle hemoglobin.

HbS causes red blood cells to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and unpredictable, painful VOCs.

For adults and children living with SCD, this means painful crises and other life altering or life-threatening acute complications--such as acute chest syndrome, stroke and infections. If patients survive the acute complications, vasculopathy and end-organ damage, resulting complications can lead to pulmonary hypertension, renal failure and early death.

LentiGlobin for SCD was designed to add functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient's own hematopoietic (blood) stem cells.

Once patients have the βA-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin, HbAT87Q, which decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

bluebird bio's clinical development program for LentiGlobin for SCD includes the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel for β-thalassemia or LentiGlobin for SCD.

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD.

The US FDA granted orphan drug designation, fast track designation, regenerative medicine advanced therapy designation and rare pediatric disease designation for LentiGlobin for SCD.

LentiGlobin for SCD is investigational and has not been approved in any geography.


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