bluebird bio has released an update on its first two gene therapies – beti-cel for β-thalassaemia and eli-cel for cerebral adrenoleukodystrophy – which are now under review by the US Food and Drug Administration (FDA).
The company also shared an update on its lovotibeglogene autotemcel (lovo-cel) gene therapy clinical programme, which was previously placed under the FDA’s partial clinical hold. The programme is for patients under the age of 18 with sickle cell disease (SCD).
In January 2022, bluebird bio received feedback from the FDA related to the partial clinical hold. Following a review of the FDA’s feedback, coupled with an evaluation of the timeline for manufacturing drug product lots and collecting analytical comparability data in the HGB-210 study, the company is reinforcing its plans to submit the Biologics License Application (BLA) for lovo-cel in Q1 2023.
bluebird bio also announced the completion of the final patient infusion in the HGB-206 study – an ongoing phase 1/2 open-label study developed to evaluate the efficacy and safety of lovo-cel for sickle cell disease. In total, 45 patients have been treated with lovo-cel in the HGB-206 study across three treatment groups: Group A, B and C. Select data from Group C of the HGB-206 study has been published in The New England Journal of Medicine (NEJM).
Regarding bluebird bio’s betibeglogene autotemcel (beti-cel) gene therapy, in November 2021, the company announced its BLA had been accepted by the FDA for the treatment. The potentially curative gene therapy is specifically developed for adult, adolescent and paediatric patients with β-thalassaemia across all genotypes who require regular red blood cell (RBC) transfusions. Since then, the FDA extended the review period for the BLA and decided to change the Prescription Drug User Fee Act (PDUFA) goal date to 19 August 2022.
Under priority review, bluebird bio would be able to receive a priority review voucher if the approval of beti-cel is granted later in 2022.
At the 63rd ASH Annual Meeting, bluebird bio revealed new data demonstrating that adult and paediatric patients diagnosed with β-thalassaemia, who require regular RBC transfusions, can produce normal or near-normal levels of total haemoglobin.
The data showed patients in these groups could continue to remain transfusion-free and achieve stable iron markers, through up to seven years of follow-up in the ongoing long-term follow-up study (LTF-303) of beti-cel.
For the company’s gene therapy programme, elivaldogene autotemcel (eli-cel), developed for cerebral adrenoleukodystrophy patients less than 18 years of age, the FDA has notified the company that the clinical hold will remain in place. It also requested more information about safety events and monitoring in the eli-cel clinical programme.
In January 2022, the FDA extended the review period for the BLA and revised the PDUFA goal date to 16 September 2022.