Bristol Myers CAR-T therapy approved by FDA for earlier myeloma use

2024-04-05

细胞疗法上市批准免疫疗法临床结果加速审批

Dive Brief:

The Food and Drug Administration has cleared Bristol Myers Squibb and 2seventy Bio’s multiple myeloma cell therapy for earlier use treating the blood cancer, approving the CAR-T medicine for patients who have previously received at least two previous drug regimens.

The OK comes three weeks after a panel of FDA advisers agreed the benefit of earlier treatment outweighed the risks, including a concern raised by agency reviewers over data indicating an elevated risk of death among treated study participants in the first year of the companies’ main trial.

Overall, trial results showed the CAR-T therapy, Abecma, reduced the risk of disease progression or death by about half, compared to standard regimens. Bristol Myers cited patient crossover from the control arm to treatment as confounding survival data, while the advisory panel noted complications with the “bridging” therapy used prior to CAR-T treatment.

Dive Insight:

Bristol Myers and 2seventy had initially hoped to win an expanded Abecma approval by mid-December, but the FDA delayed its decision to consult with its advisory panel.

Convened in mid-March, the expert committee debated earlier use of both Abecma and a rival CAR-T therapy from Johnson & Johnson and Legend Biotech called Carvykti. Ultimately, they found the benefits of both to be substantial enough to look past the early death risk raised by the FDA, voting 11-0 in favor of Carvytki and 8-3 in support of Abecma.

Treatment benefit “is significant and offers our patients a chance of significant time off therapy with associated quality of life improvement,” said Christopher Lieu, a panelist and associate medical professor at the University of Colorado, at the March 15 meeting. “But it’s a closer margin than I think we would like, and patients will need to have in-depth discussions about the risks and benefits.”

Both therapies target a protein called BCMA that’s found on malignant B cells in people with multiple myeloma. Their earlier approvals for later-line treatment added another option to a growing arsenal of therapies that have helped improve outcomes for patients with the blood cancer.

Still, people with multiple myeloma often relapse or their cancer becomes resistant to one treatment, requiring a switch to different regimens.

Abecma was previously cleared for relapsed or refractory disease in adult patients who have received four or more prior regimens, including ones that involve three of the main classes of multiple myeloma drugs.

Now, Abecma can be used in after two lines of therapy, provided patients are “triple class-exposed,” or have received treatment that includes those three types of drugs.

The OK will allow Bristol Myers to market Abecma more widely, potentially boosting sales. “We see the approval as a generally positive development, allowing Abemca to expand into a larger patient pool, though the approval does not alter our base expectations for utilization of Abecma relative to competing agents in earlier line multiple myeloma, namely [Carvykti],” wrote Raymond James analyst Sean Mccutcheon in a Friday note to clients.

CAR-T therapies are personalized, built from the immune cells of each individual patient. Manufacturing is complex and can be challenging to get right every time. Bristol Myers said it has a manufacturing success rate of 94% providing Abecma in the commercial setting.