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FDA Grants Bluebird Bio Priority Review of BLA for SCD Treatment
Bluebird Bio gets FDA priority review for SCD treatment in patients 12+ with vaso-occlusive history.
On June 21, 2023, Bluebird Bio issued a press release announcing that the FDA had granted its biological license application (BLA) for a new sickle cell disease (SCD) treatment priority review designation. The treatment is intended for SCD patients 12 and older with a history of vaso-occlusive events.
SCD is a genetic disease characterized by pain, anemia, organ damage, and, in some cases, early death. The condition causes high concentrations of sickled hemoglobin in red blood cells, which leads to a shorter RBC lifespan.
“The burden that people living with SCD and their families live with today is staggering. Beyond extreme pain crises that send patients to the hospital, SCD progression is associated with grave long-term consequences,” said Andrew Obenshain, chief executive officer of the company, in its press release.
As a result of these molecular changes, patients present with hemolytic anemia, vasculopathy, and vaso-occlusion. Eventually, 50–60% of patients with SCD get end organ damage. Roughly 24% are impacted by multi-organ injury.
The announcement revealed that the drug is called lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy for SCD treatment. This therapy is supposed to add copies of a modified β-globin gene (βA-T87Q-globin gene) into hematopoietic stem cells.
The genetic treatment allows patients to generate anti-sickling hemoglobin, reducing the number of sickled blood cells.
The BLA includes data from multiple different studies. One study assessed data from 36 patients across a median of 32 months. Additionally, two patients were evaluated over 18 months. Beyond data on the efficacy of the drug, there was also safety data from 50 patients submitted.
“The FDA’s acceptance of our BLA for lovo-cel moves us one step closer in bringing a potentially transformative therapy to the sickle cell disease community that is long overdue, and we are grateful to the patients, caregivers, researchers, clinicians, and community leaders who have enabled this exciting milestone. We look forward to working with the agency on its review,” added Obenshain in the release.
