Bristol Myers Squibb has received a March 27 PDUFA date for its anti-BCMA CAR T-cell therapy. The priority review action date sets Bristol Myers up to win approval for the bluebird bio-partnered drug in time to satisfy the terms of its acquisition of Celgene.
The FDA rejected the original ide-cel application in May, telling Bristol Myers and bluebird to return with answers to questions related to the CMC side of the filing. Bristol Myers refiled for approval in late July. The filing gave Bristol Myers a shot at getting ide-cel approved in time for Celgene investors to receive an additional $9 share under the terms of the $74 billion megamerger of the companies.
With the FDA granting ide-cel priority review, Bristol Myers is on track to satisfy the ide-cel side of the Celgene agreement. However, it remains possible that the FDA’s review of the application will throw up issues that could delay approval or lead the agency to reject ide-cel.If that happens, $7 billion will just evaporate.
Manufacturing is one area of concern. As Bristol Myers noted in a financial filing in August, COVID-19 could prevent the FDA from carrying out the on-site facility inspection needed to determine whether to approve ide-cel. Bluebird CEO Nick Leschly discussed manufacturing inspections on an analyst call last week.
“It’s certainly something that’s very high on the agenda. The agency is trying to figure out how we do this in an expeditious manner so that we get these things done so that they don’t hold up what are really interesting programs,” Leschly said on a call hosted by Morgan Stanley.
Further delays to the program would prevent Bristol Myers from getting ide-cel to market in time to secure a payday for Celgene investors and shrink or eliminate its head start over BCMA CAR-T therapy rival Johnson & Johnson. J&J is expected to seek approval by the end of the year.
Bristol Myers and J&J will both encounter an FDA that has shown a willingness to deliver shocks to the cell and gene therapy companies in recent months, with BioMarin and Sarepta among the other biotechs to be rocked by rejections. Leschly said the events show a need for better communication.
“There’s no hiding communication between a number of us on the industry side and the FDA needs to get better. I think Peter [Marks at the FDA] would agree with that. We need to find a way to make sure there are fewer surprises. We shouldn’t get a surprise letter on ide-cel without having any kind of dialogue beforehand,” Leschly said.
Leschly said poor communication, which potentially stems from the FDA’s need to handle COVID-19 vaccine tasks on top of its mushrooming cell and gene therapy workload, can be fixed. If, as some people have speculated, the rejections reflect the FDA taking a harder line on cell and gene therapies and raising the bar for approval, the impact may be more long lasting. Leschly is skeptical of that idea. Over the coming months, the ide-cel filing may shed light on whether Leschly’s positivity is justified.