According to a story from BioPharma Reporter, the company bluebird bio has recently submitted its Biologics License Application (BLA) to the US Food and Drug Administration (FDA). This application is for lovotibeglogene autotemcel (lovo-cel), which is a gene therapy intended to treat sickle cell disease, a rare disease. The BLA is directed for patients aged 12 or older with a history of vaso-occlusive events.
About Sickle Cell Disease
Sickle cell disease is a genetic, inherited disorder of the blood. This disorder is characterized by an abnormality affecting hemoglobin, which is found in red blood cells and is responsible for carrying oxygen. The abnormality causes blood cells to lose their typical circular shape and instead take on an elongated, sickle-like appearance. This is caused by a genetic mutation that may have arisen as a defense against malaria, although this benefit only occurs in people with sickle cell trait, not the disease. Symptoms begin to appear at around six months old and include swelling of the hands and feet, stroke, bacterial infections, and acute episodes of severe pain termed sickle cell crisis. Severity of disease varies, but these attacks can result in serious declines in health and organ damage. Treatment is mostly symptomatic, but bone marrow transplant has been curative in children. The disease most frequently affects people of African ancestry. Life expectancy is between 40 and 60. To learn more about sickle cell disease, click here.
This application also includes a request for Priority Review. If granted the agency will complete the review in six months instead of the typical 10 months. Gene therapies for genetic disorders are slowly making their way on to the market and have the potential to be breakthrough treatments that need to only be administered once to have lasting, potentially lifelong effects.
Lovo-cell is designed to deliver modified yet functional copies of the gene that is affected by sickle cell disease into the patient’s stem cells. The gene therapy has received Orphan Drug, Fast Track, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy designation from the FDA. If approved, this will be the third gene therapy from the company to enter the market, following two approvals for beta thalassemia and cerebral adrenoleukodystrophy, respectively.