NEW YORK – Gene therapy developer Bluebird Bio has submitted a biologics license application to the US Food and Drug Administration for an investigational sickle cell treatment, the company said Monday.
Lovotibeglogene autotemcel (lovo-cel), which was previously granted orphan drug designation and fast track designation from the FDA, is a potential treatment for patients at least 12 years of age with sickle cell disease and a history of vaso-occlusive events. It's a one-time treatment designed to add functional copies of a modified form of the β-globin gene into a patient's hematopoietic stem cells so that the cells can produce anti-sickling hemoglobin.
Bluebird Bio's application to the FDA includes a request for priority review. The application is based on results from a Phase I/II trial, in which 96 percent of patients treated with lovo-cel experienced resolution of severe vaso-occlusive events, as well as a Phase III study that showed promising results in the two patients treated with the therapy.
If approved, the drug will be Somerville, Massachusetts-based Bluebird Bio's third FDA-approved ex vivo gene therapy for a rare genetic disease.
Earlier this month, Vertex Pharmaceuticals and CRISPR Therapeutics submitted a biologics license application to the FDA — also with a request for priority review — for a competing investigational sickle cell disease treatment called exagamglogene autotemcel (exa-cel), an ex vivo therapy that uses CRISPR-Cas9 gene editing on patients' hematopoietic stem cells.