- The Food and Drug Administration has rejected an application for approval of an experimental multiple myeloma cell therapy from Bristol Myers Squibb and Bluebird bio, dealing a surprise and potentially costly setback to the partner companies.
- Bristol Myers and Bluebird aim to provide the FDA with new details that it requested, which involve documentation of manufacturing processes, and to resubmit their application by the end of July.
- The "refuse-to-file" letter for ide-cel, as the cell therapy is called, is the second regulatory delay for Bristol Myers in the past week, after the FDA extended its review of another cancer cell therapy the pharma acquired in a 2019 buyout of Celgene. This latest hurdle, however, could put at risk a $9 per share payment to former Celgene shareholders that's based on winning drug approvals by certain dates.
Bristol Myers' entry into the cell therapy field is not going quite as planned.
In acquiring Celgene for $74 billion last year, Bristol Myers also bought into the biotech's exploration of CAR-T treatment for cancer, picking up a handful of experimental drugs that rely on engineering immune cells to attack tumors.
The two most advanced cell therapies, ide-cel and another from Celgene called liso-cel, were singled out in a deal provision that would have Bristol Myers pay an additional $9 per Celgene share should both win approval, along with a third drug for multiple sclerosis.
The past week has been particularly stressful for holders of that contingent value right, or CVR, with delays for first liso-cel and now ide-cel. The third drug, ozanimod, was approved by the FDA in March and is now sold as Zeposia.
On May 6, the FDA pushed back by three months its review of liso-cel for approval in treating large B-cell lymphoma. Bristol Myers had submitted, after its application, additional information "which was deemed to constitute a major amendment" to the company's application. Bristol didn't provide specifics, only saying the FDA needed more time to review that information. The FDA has to approve liso-cel by the end of the year for CVR holders to have a chance at a payout.
The refuse-to-file for ide-cel is another unexpected, and potentially more damaging, blow.
If Bristol Myers and Bluebird are able to resubmit their drug application by the end of July, as is now planned, there should still be enough time for Bristol Myers to meet the March 31 approval deadline set out in the CVR. Company executives expressed confidence they're still on track, but it's now a riskier proposition.
"We believe we submitted a complete dossier to the FDA," said Bristol Myers CEO Giovanni Caforio on a Wednesday morning call with investors. "What we are really discussing here is the level of detail and information that the FDA has requested, which typically is part of the review process rather than the submission."
In a separate call, Bluebird CEO Nick Leschly described the information requested by the FDA as "more on the administrative, documentation side."
Both companies emphasized no new clinical or non-clinical data have been requested by the FDA, and indicated they could quickly provide the details demanded by the agency. Analysts from Mizuho Securities USA and Stifel, writing in notes to clients Wednesday, both believe ide-cel should still win approval on time.
Still, the news is a setback in what's become a very competitive race to develop cell therapies for multiple myeloma, a persistent and deadly cancer of the bone marrow. If approved, ide-cel would be the first CAR-T treatment for the cancer type, following similar drugs from Novartis and Gilead that treat leukemia and lymphoma.
And, for Bluebird, the manufacturing questions raised by the FDA around ide-cel follow issues and delays experienced by the company with its gene therapy Zynteglo, which is launched in Europe but not yet approved in the U.S.