EMA’s PRIME program designed to optimize development and expedite evaluation of innovative medicines for patients with high unmet need
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.
“Even with recent progress to deliver new medicines for sickle cell disease, there remains a profound unmet need for people living with SCD. SCD is a progressive disease that frequently leads to organ damage and early death, and whose hallmarks are hemolytic anemia, painful vaso-occlusive crises (VOCs) and stroke,” said
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). 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 (ACS), 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 (HSCs). 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. For more information visit: www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov.
LentiGlobin for SCD received orphan medicinal product designation from the
LentiGlobin for SCD is investigational and has not been approved in any geography.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders, including cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia and multiple myeloma, using three gene therapy technologies: gene addition; cell therapy and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in
LentiGlobin and bluebird bio are trademarks of bluebird bio, Inc.
bluebird bio Forward-Looking Statements
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the company’s development and regulatory plans for the LentiGlobin for SCD product candidate. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that the COVID-19 pandemic and resulting impact on our operations and healthcare systems will affect the execution of our development plans or the conduct of our clinical studies; the risk that even if LentiGlobin for SCD addresses ACS and VOC events, that it may not address progressive organ damage experienced by patients with SCD; the risk that the efficacy and safety results observed in the patients treated in our prior and ongoing clinical trials of LentiGlobin for SCD may not persist or be durable; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in when treating additional patients in our ongoing or planned clinical trials; the risk that the HGB-206 and HGB-210 clinical studies as currently contemplated may be insufficient to support regulatory submissions or marketing approval in the
Source: bluebird bio, Inc.