In a blow to Bristol Myers Squibb and bluebird bio’s hopes to gain a speedy approval for ide-cel (idecabtagene vicleucel), the US Food and Drug Administration (FDA) issued a ‘refusal to file’ letter.
According to the companies, the FDA decided that the chemistry, manufacturing and control (CMC) section of the biologics license application (BLA) lacked the detail necessary to process the application.
The companies noted that the only problems raised by the FDA related to the manufacturing process, with no additional clinical or non-clinical data necessary.
BMS plans to resubmit the BLA by July of this year, at the latest.
In an investor call after the news, Giovanni Caforio, CEO of BMS, stated that, during pre-BLA discussions with the FDA, the company was under the impression that it had submitted a ‘complete dossier’.
BMS had taken the lead role in the creation of the BLA and the CEO stated the company’s reaction to the news was one of ‘surprise and disappointment’.
Further than this, Samit Hirawat, CMO of BMS, stated, “What the FDA is requesting includes specific details regarding the CMC module of the application, which is typically required in the regulatory review process rather than in the initial application.”
As a result, he added that BMS believes that it should be possible to quickly turn around the application process, once these details are taken care of.
No further information was revealed on what CMC processes required additional detail.
However, bluebird is no stranger to managing the complexity of manufacturing cell and gene therapies alongside regulators. Last year, the company received conditional approval for its gene therapy in Europe, only to delay its launch as it worked with regulators to refine the manufacturing process.
Earlier this week, BMS had decided to take the responsibility of all ex-US manufacturing for ide-cel into its own hands, as part of a $200m (€183m) deal that also saw bluebird give up rights to ex-US milestone payments and royalties.