FDA filings for bluebird bio's LentiGlobin, Lenti-D pushed back until mid-2021

Bluebird bio indicated Thursday that regulatory applications for both LentiGlobin and Lenti-D in the US will be hit by the global COVID-19 pandemic, with filings for the two therapies pushed back until mid-2021. "The COVID-19 pandemic has created new challenges for bluebird, the broader biotech community, and society as a whole," remarked CEO Nick Leschly.

The company said it "remains committed to maintaining its development plans," although the "rapidly evolving global environment" is prompting it to shift the timing of enrollment and completion of clinical studies by at least three months, with specific timing shifts expected to vary by clinical trial and programme.

In regards to the rolling submission for its LentiGlobin drug product in beta-thalassaemia, bluebird said it has been in discussions with the FDA about requirements and timing of certain information related to release assays (for related analysis, see ViewPoints: Zynteglo delay highlights US/EU regulatory unity- and stringency). According to the company, while the assays are advancing through development and validation for use in the commercial setting, they "are likely to be impacted by the COVID-19 situation."

Bluebird also explained that it has not been able to come to an agreement with the FDA regarding the company providing data on these assays during the review of its filing. As a result of this and the expected shifts related to COVID-19, bluebird said it does not anticipate completing its LentiGlobin rolling submission for beta-thalassaemia until the second or third quarters of next year, revised from prior guidance of the second half of 2020. However, the drugmaker said that if completion of the submission does get pushed back that far, it "may have the opportunity to seek approval for a broader patient population, including patients with β00 genotypes, in addition to paediatric patients." Meanwhile, bluebird said it continues to hold talks with the FDA about a regulatory path for LentiGlobin in sickle-cell disease, and anticipates giving an update by the end of 2020.

Moreover, the FDA talks on LentiGlobin are also leading bluebird to believe that the filing for its Lenti-D gene therapy in cerebral adrenoleukodystrophy (CALD) will be submitted in mid-2021 as well. However, the company noted that it remains on track to submit an EU application for Lenti-D in CALD by the end of this year.

In regards to idecabtagene vicleucel (ide-cel), also known as bb2121, bluebird reiterated recent guidance from partner Bristol Myers Squibb that the companies are on track to file the CAR-T cell therapy for the treatment of fourth-line multiple myeloma some time during the first half of 2020. Bluebird also noted that ongoing clinical trials of LentiGlobin for beta-thalassaemia and sickle-cell disease, and Lenti-D for CALD, as well as the study of bb21217, a next-generation anti-BCMA CAR-T cell therapy based on ide-cel, "all currently remain active, but the effects of the COVID-19 pandemic is resulting in disruptions to study conduct across these programmes."

Further, as a partner in the development of ide-cel, Bristol Myers Squibb has temporarily suspended screening, enrollment and apheresis in their cellular therapy clinical studies, which bluebird says includes the KarMMa-2, KarMMa-3 and KarMMa-4 studies. However, it noted that the decision is "not considered an urgent safety measure" and will not affect FDA submission activities for ide-cel.

Meanwhile, in January, bluebird announced the launch of LentiGlobin in Germany, where it is marketed as Zynteglo, for certain patients with transfusion-dependent beta-thalassaemia. However, it now anticipates treatment of the first commercial patient in Germany to be pushed back to the second half of 2020. It added that while it intends to continue engaging in reimbursement discussions and moving ahead with commercial preparations in the priority launch markets in Europe, the COVID-19 pandemic will likely "impact its ability to achieve market access and reimbursement" in the region.

EU regulators conditionally approved Zynteglo last July, with bluebird setting the price at €1.575 million over a five-year installment plan.

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