Bristol Myers Squibb in hot water after FDA rebuffs CAR-T therapy on manufacturing concerns
Bristol Myers Squibb plans to refile its submission for ide-cel by July. (Bristol Myers Squibb)
As part of its $74 billion Celgene pickup, Bristol Myers Squibb offered future payouts based on FDA approval for three key pipeline assets. Already in jeopardy after one delay, that payoff looks even more tenuous now that the FDA has made a rare—and embarrassing—refusal to even consider one of those approval filings.
Bristol on Wednesday revealed the FDA had refused to review its submission for ide-cel (bb2121), a CAR-T cell therapy for multiple myeloma co-developed with bluebird bio and acquired in the drugmaker's buyout of Celgene in late 2019.
Bristol said the FDA highlighted concerns about the manufacturing portion of ide-cel's filing rather than clinical or non-clinical data. The drugmaker expects to refile by July.
How to Streamline Your Clinical Research Organization's Processes End to End
Learn how implementing one platform leads to data consistency and ultimately facilitate faster clinical trials while reducing overall trial costs, leave behind spreadsheets and home-grown tools for a predictable trial and the ability to forecast unit delivery resulting in the optics you need to ensure a successful trial, and hear experts share industry trends of what is affecting the Clinical Research Organization industry today.
Ide-cel was one of five key pipeline assets Bristol picked up in its Celgene deal, and its approval by March 2021 was one of three regulatory milestones for investors holding contingent value rights in the $74 billion transaction.
In a call with analysts Wednesday morning, Bristol CEO Giovanni Caforio said the FDA's pushback on scanty documentation—specifically in the application's chemistry, manufacturing and control section—was usually reserved for the preliminary review process. An expedited approval could still be accomplished when the submission is refiled, Caforio contended.
"We believe we submitted a completed dossier to the FDA, so what we are really discussing here is the level of detail the FDA has requested," Carforio said.
Bluebird CEO Nick Leschly mirrored Carforio's comments on a separate analyst call, calling the FDA's letter "unusual" and stressing that no new data were requested.