USA — Bluebird Bio, the gene therapy company, disclosed that its plan to seek FDA approval for the one-time treatment lovotibeglogene autotemcel (lovo-cel) to treat sickle-cell disease, has been delayed.
Last year, after the US regulator lifted a partial clinical hold on the therapy, allowing young patients to participate in the study, bluebird bio indicated that it would file for approval in the first quarter of 2023.
However, in an update on the program provided along with the fourth-quarter results, the company noted that the filing would be delayed.
Consequently, shares of Bluebird bio fell by as much as 27% on Wednesday indicating that investors were disappointed with the news.
The company stated that it responded earlier this month to recent feedback from the FDA on vector and drug product analytical comparability evaluations completed last December.
According to CEO Andrew Obenshain, bluebird bio may be able to file the application in a few weeks, depending on when the FDA provides new feedback about the company’s drug manufacturing process.
He also said, “As soon as we get that, we’ll move quickly to expedite the application, pending the resolution of these FDA comparability questions.”
The delay in the filing for FDA approval has raised concerns about Bluebird bio’s ability to compete in the gene therapy market.
However, Obenshain reassured investors that the company is committed to providing life-changing treatments for people with rare diseases and will continue to work closely with the FDA to expedite the approval process.
Eyeing commercial launch in early 2024
According to an update given alongside its fourth-quarter results, bluebird bio plans to request priority review for lovo-cel in patients aged 12 and older with a history of vaso-occlusive events, and anticipates a commercial launch in early 2024, pending approval.
The company’s CEO, Andrew Obenshain, suggested that the additional back-and-forth with the FDA may lead to a smoother review process.
Although data from the pivotal Group C cohort of the HGB-206 trial showed promising results, with 96% of patients treated with lovo-cel experiencing complete resolution of severe vaso-occlusive events, the gene therapy still faces concerns over potential risks.
Specifically, three patients given the gene therapy in studies developed illnesses that looked like cancer, which raised concerns about its safety.
However, Bluebird Bio later determined that only two of those cases were actually cancerous and were not related to the vector.
Unfortunately, the company also announced that another patient who had received lovo-cel in a sickle-cell study was recently diagnosed with myelodysplastic syndrome (MDS).
Despite this, Bluebird Bio notes that the trial’s independent data monitoring committee disagreed with the physician’s diagnosis and concluded that the case did not meet their criteria for the condition.
Moreover, the company adds that there is “no evidence of insertional oncogenesis” tied to the vehicle that delivers the therapy.
The patient in question is reported to be clinically stable and is not undergoing treatment for MDS.
In 2022, Bluebird Bio was faced with significant financial challenges due to a series of safety concerns associated with lovo-cel, which resulted in a delay of more than a year in the company’s efforts to seek approval.
Despite these obstacles, Bluebird Bio was ultimately able to obtain two critical approvals from the FDA. The first was for Zynteglo (betibeglogene autotemcel), which is intended to treat β-thalassemia in patients who require regular red blood cell transfusions.
The second approval was for Skysona (elivaldogene autotemcel), which is used to slow the progression of neurologic dysfunction in patients with cerebral adrenoleukodystrophy.
Looking to the future, Vertex Pharmaceuticals and CRISPR Therapeutics have announced that they are on track to finish an application for a competing therapy to treat sickle-cell disease by the end of this month.
As the race to develop effective treatments for this debilitating condition continues, the market is likely to remain highly competitive, with many companies vying for a slice of the pie.
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