bluebird bio has reported its financial results and business highlights for the second quarter of 2022, in which the company revealed the anticipated launch of both betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel) gene therapies in the fourth quarter of the year.
The news comes after both therapies were endorsed by the US Food and Drug Administration (FDA) Cell Tissue & Gene Therapy Advisory Committee (CTGTAC) in June 2022.
Beti-cel is under priority review for the treatment of people with transfusion-dependent beta-thalassaemia (beta-thal), a rare genetic blood disease caused by a gene defect that impairs the ability of red blood cells to produce haemoglobin. Patients with the most severe form of beta-thal develop life-threatening anaemia and have to undergo regular blood transfusions, a lengthy process typically needed every two to five weeks.
Eli-cel is under priority review for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients under the age of 18 who do not have an available and willing matched sibling donor. CALD is a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death.
Beti-cel and eli-cel have Prescription Drug User Act Fee (PDUFA) goal dates of 19 August 2022 and 16 September 2022, respectively. If approved, the company anticipates that both therapies will be availabile in the fourth quarter of 2022.
Andrew Obenshain, chief executive officer, bluebird bio, said: “The second quarter marked significant progress for bluebird bio and a precedent-setting moment for the field of gene therapy.
“With the FDA advisory committee’s unanimous support for beti-cel and eli-cel for their target indications, we are now laser-focused on commercial readiness and, if approved, we anticipate launching both therapies in the fourth quarter of this year.”
bluebird bio also reported that it remains on track to submit a Biologics Licensing Application (BLA) to the FDA for lovotibeglogene autotemcel (lovo-cel) for sickle cell disease in the first quarter of 2023.
The company reported that it ended the quarter with $218m in restricted cash, cash and cash equivalents and marketable securities, having raised approximated $24.7m in gross proceeds through its At-the-Market (ATM) equity facility.
The company is exploring additional financing opportunities, including public or private equity financings and monetising any priority review vouchers that may be issued upon approval of beti-cel or eli-cel.