The US Food and Drug Administration (FDA) has accepted bluebird bio’s application for priority review for its sickle cell gene therapy lovotibeglogene autotemcel (lovo-cel). The decision was made based on efficacy data from 36 patients and safety results from 50. Sickle cell disease (SCD) is an inherited blood disorder caused by a mutation in the gene encoding for hemoglobin that sticks together inside red blood cells.
According to Emily Kimber, “Following treatment, a patient’s red blood cells can produce anti-sickling haemoglobin that decreases the proportion of sickle haemoglobin, with the goal of reducing sickled red blood cells, haemolysis and other complications. The company’s US submission is backed by efficacy results from 36 patients with 32 months of follow-up data and two patients with 18 months of follow-up. Safety data from 50 patients, including six patients with six or more years of follow-up is also included in the application.”
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(Source: PM Live, June 22nd, 2023)