Patient advocate slams FDA decision for CAR-T warning labels

The US FDA has come under criticism after demanding CAR-T therapies must have black box warnings (BBWs) on their labels to signify the risk of T-cell malignancies.

In November 2023, the US Food and Drug Administration (FDA) safety material about the risk of T-cell malignancies concerning individuals who are receiving chimeric antigen receptor (CAR) T cell therapies for the treatment of specific blood cancers.

This week, the agency issued safety and availability communications to the following companies regarding their respective CAR-T therapies: Bristol-Myers Squibb’s Abecma (idecabtagene vicleucel, commonly referred to as ide-cel) and Breyanzi (liso-cel), Johnson & Johnson’s Carvykti (ciltacabtagene autoleucel), Novartis’ Kymriah (tisagenlecleucel) and Kite Pharma’s Yescarta (axicabtagene ciloleucel).


The correspondence advised the firms that the Agency has “determined that the new safety information should be included in the labeling for all BCMA- and CD19-directed genetically modified autologous T cell immunotherapies.”

The FDA’s communications came under fire from Laurie Adami, a patient advocate from Cancer Research Institute. Following a 12-year battle with follicular non-Hodgkin lymphoma, Adami received Kite’s Yescarta and was cured after a month.

Adami took to LinkedIn and said the decision is “understandable but still a shame” as it will increase“fear for patients with a terminal illness as they deal with the physical, mental, and financial stress associated with managing these diseases. Treatment choices are difficult and complex, and the reality is if CAR-T has a BBW, so too should most cancer medications.”

“I wish these warnings and public releases would come with some commonsense data as well to put the warnings in perspective for patients. [The] FDA is clearly not talking to any of us patients who had no other options left besides CAR-T. Should we pass up on CAR-T and die instead? Not me.”

Anthony Davies, CEO of Dark Horse Consulting added he is “sensing mixed messages from the FDA” but that “BBW’s are more common than most people think,” also on the social media platform.

“Botox has one, for example, and I think 99.9% of users don’t realize, they simply warn potential patients of medication risk. A 30-month study of an ambulatory care facility showed >40% of patients had received one of more BBW’d medication. That said, they are the most stringent warning, indicating the possibility of serious injury or death – I don’t think there’s any doubt that these secondary malignancies fall into that category. So, for those commenting that patients should be able weigh the risk – that is exactly the goal of such labels.”

Last week, Susan Nichols, CEO of Propel Biosciences, spoke about the warning labels at Advanced Therapies Week in Miami, Florida, telling delegates that 22 of the 27,000 patients who have received commercially available therapies experienced t-cell malignancies.

Nichols referenced Peter Marks, the director of the Center for Biologics Evaluation and Research (CBER) at the FDA, comments at ARM’s cell and gene State of the Industry briefing where he said he is “not concerned at this low number at this time.”

Update: the FDA issued a revised letter to Kite and said its second approved CAR-T therapy Tecartus (brexucabtagene autoleucel) must carry a BBW.