SHERIDAN, WYOMING – July 1, 2025 – The U.S. Food and Drug Administration’s recent decision to remove Risk Evaluation and Mitigation Strategies (REMS) from approved CAR T cell therapies marks a pivotal shift that could reshape access to these potentially curative treatments and drive significant market expansion across the cell and gene therapy industry.
The agency eliminated REMS requirements for BCMA- and CD19-directed CAR T therapies produced by major developers including Bristol Myers Squibb, Johnson & Johnson, and Gilead/Kite. REMS had been in place to address the two syndromes most often associated with the therapies: cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). But the FDA has now determined that “established management guidelines” and the “extensive experience” of the medical and hematology community are sufficient to diagnose and manage these risks, BMS said in a press release Thursday, adding that the change is “likely to help further accelerate cell therapy into the community center setting.”
Industry Leaders Welcome FDA’s Bold Move
In a statement Friday, Prasad, director of the Center for Biologics Evaluation and Research (CBER), called the move a “bold step,” adding that dropping REMS “expedites the delivery of potentially curative treatments to patients and reduces burden on providers.”
“Overall, we view the removal of the REMS program for CAR-T cell therapies as a positive development for the space, as it supports the notion that the FDA is working to minimize red tape and increase access to potentially curative treatments for patients,” William Blair said in a note to investors on Friday. The FDA also significantly shortened the length of time patients must wait after treatment before driving from eight to two weeks and cut the requirement to stay near a healthcare facility from four to two weeks.
In an emailed statement to BioSpace, Stephen Majors, vice president of global communications at The Alliance for Regenerative Medicine, added that the change is a “patient friendly move that will significantly reduce the burden on patients seeking transformative treatments for deadly blood cancers.” He added that the move “reflects the agency’s thoughtful consideration of real-world evidence and evolving understanding of CAR-T cell therapies, based on recent dialogue with ARM and our members.”
Meanwhile, Benjamin McLeod, founder of Convey Bio wrote on LinkedIn, “Regulatory trust has finally caught up with clinical practice.”
Increased Patient Access a Boon for CAR T Developers
CAR T cell therapy—which involves the extraction of a patient’s own T cells and adding a chimeric antigen receptor (CAR) that directs them to target and kill cancer cells before expanding and reinfusing those T cells back into the patient’s bloodstream—can be lifesaving. However, only about 2 in 10 patients receive this treatment, according to BMS, due to “the confluence of complex logistical and geographic barriers affecting patients and providers.”
The FDA’s new requirements should lower that burden, experts agree. In a note to investors on Friday, Leerink Partners wrote, “We believe the reduction in CAR-T burden for patients and caregivers, enabled by FDA’s new monitoring and driving requirements, will drive expansion of the US cell therapy market.” Leerink relayed one KOL’s belief that if the FDA reduced monitoring requirements and driving restrictions to 14 days, “he would expect rates of CAR-T infusion to double.”
In its press release last week, BMS wrote that label changes “reinforce [the company’s] continued efforts to collaborate across the healthcare ecosystem, with the ultimate goal of reaching more patients and democratizing access to cell therapy.”
In an interview with BioSpace, Lynelle Hoch, president of the cell therapy organization at BMS, noted that with CAR T cell therapies moving into earlier lines of treatment, patients tend to have jobs and childcare responsibilities, “and the idea of being at an academic center, potentially hours away from home for four weeks with a caregiver, and then not being able to drive, really impedes their ability to get these CAR Ts.”
Additionally, Hoch said it is “well documented” that because CAR T therapies are delivered at “a select set of academic centers” and come with logistical barriers, there are “significant socioeconomic and racial disparities” in terms of patient access to these medicines. Hoch pointed to recently published research showing that individuals with an income of less than $40,000 were nearly 60% less likely to receive a CAR T therapy than those with an income greater than $100,000 thousand, and that Black and Hispanic patients are only half as likely to receive a CAR T than other patient populations.
In terms of infrastructure, sites might be ready to accommodate these additional patients. The two KOLs Leerink consulted said their centers “now have excess capacity and could absorb increases in demand.”
Hoch added that with the label changes, some of the infrastructure requirements—including the need for community hospitals to have a contractual relationship with an affiliated adverse event hospital—have now been reduced.
Momentum for CAR T Pipeline and Autoimmune Indications
Such increased uptake should fuel further CAR T development. At a recent investor dinner hosted by William Blair, BMS management “cited community uptake as a key driver of CAR-T market expansion and Breyanzi growth,” the firm wrote. Breyanzi, a CD19-directed CAR T cell therapy, is approved for five indications, including relapsed or refractory (R/R) chronic lymphocytic leukemia, small lymphocytic lymphoma and R/R mantle cell lymphoma, while a second BMS CAR T, BCMA-directed Abecma, is marketed for patients with relapsed or refractory multiple myeloma.
A representative for Gilead/Kite told BioSpace in an email that the labels for Yescarta (FDA-approved for relapsed or refractory (R/R) large B-cell lymphoma and R/R follicular lymphoma) and Tecartus (R/R B-cell precursor acute lymphoblastic leukemia and R/R mantle cell lymphoma) have been updated to reflect the recent FDA changes.
While Hoch wouldn’t speculate on any specific near-term sales projections for Breyanzi and Abecma, she said “there is no question that this was an important step forward in being able to increase the number of patients that are eligible for CAR T to receive a CAR T.”
Beyond the potential for market expansion for approved CAR T therapies, William Blair noted possible readthrough benefits for those in development, such as such as Arcellx and Gilead’s anito-cel, whose positive neurotoxicity profile could help it compete against J&J and Legend’s Carvykti in relapsed or refractory multiple myeloma.
“This update also suggests that we may not see REMS requirements for CD19 and BCMA autologous CAR-T cell therapies approved in the future, such as Arcellx’s anito-cel,” the firm wrote.
Further, William Blair said the news is a “positive signal” to companies developing CAR T therapies for autoimmune diseases, including BMS, Autolus and Cabaletta Bio, “given that the safety profile is more tolerable than in oncology.” This is a field that is currently exploding, as evidenced by AbbVie’s $2.1 billion buyout of Capstan Therapeutics and its in vivo edited CAR T therapy for B cell–mediated autoimmune diseases on Monday.
From a regulatory standpoint, Hoch agreed that last week’s CAR T label changes are indicative of the FDA’s broader thinking around the cell and gene therapy space. “I think the FDA’s decision signals an increase in confidence in the safety profile cell therapy. It’s also reflective of making a data-driven decision,” she said, noting that over 30,000 patients have been treated with CAR T therapies. “To us, it shows a very strong signal from the FDA.”