Lisocabtagene maraleucel

iStock/ Natalya Kosarevich Cell and gene therapy leaders say the agency’s decision to remove the Risk Evaluation and Mitigation Strategies that had been attached to approved CAR T cancer therapies reflects “thoughtful consideration of real-world evidence” and “regulatory trust.” FDA leaders Marty Makary and Vinay Prasad have struck a tone of urgency when it comes to bringing innovative medicines to the U.S. market, launching a new priority review voucher program for certain drugs and vowing to “rapidly make available” rare disease therapies. Last week, the agency took another step in this direction, removing guardrails around six CAR T cell therapies. Specifically, the FDA removed the Risk Evaluation and Mitigation Strategies (REMS) that had been in place for BCMA- and CD19-directed CAR T therapies for multiple myeloma and certain types of leukemia and lymphoma, made by Bristol Myers Squibb, Johnson & Johnson, Gilead/Kite and others. 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.” 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.” Analysts and cell therapy groups lauded the move. “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. 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 patients with relapsed or refractory chronic lymphocytic leukemia (CLL) and small lymphocytic 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 pro 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 pro 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.”

CAR-Ts, the first of which were initially approved in June 2025, have demonstrated a wealth of efficacy in haematological oncology indications. Credit: Nemes Laszlo via Shutterstock. The US Food and Drug Administration (FDA) has removed the risk evaluation and mitigation strategies (REMS) requirements for all approved autologous chimeric antigen receptor (CAR-T) cell immunotherapies, saying the access barrier is no longer needed for the modality. REMS is a safety programme that the FDA requires for certain serious safety concerns to help ensure the benefits of the medication outweigh its risks. It limits the access of approved drugs to certain hospitals and clinics that had to be specially certified for therapy administration and have access to anti-inflammatory medication. CAR-Ts, the first of which were initially approved in June 2025, have demonstrated a wealth of efficacy in haematological oncology indications by boosting the patients’ own T-cells to fight cancer cells. However, their use comes with the risks of cytokine release syndrome (CRS) and neurological toxicities. The FDA commented that REMS is no longer necessary to ensure that the benefits of BCMA-directed and CD19-directed CAR-T cell immunotherapies outweigh their risks. It added that the elimination of the access barrier would minimise burden on healthcare systems. Bristol Myers Squibb’s Abecma (idecabtagene vicleucel) and Breyanzi (lisocabtagene maraleucel), and Johnson & Johnson/Legend Biotech’s Carvykti (ciltacabtagene autoleucel) will have their REMS requirements removed. Novartis’ Kymriah (tisagenlecleucel), along with Gilead Sciences’ Tecartus (brexucabtagene autoleucel) and Yescarta (axicabtagene ciloleucel), also stand to benefit from the FDA’s decision. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence A seventh FDA-approved CAR-T therapy – Autolus’ Aucatzyl (obecabtagene autoleucel) – will not be affected because it never carried a REMS requirement . “FDA has determined that the safe and effective use of CAR T cell immunotherapies for the indicated population can be assured without a REMS. Adverse event reporting for CRS and neurological toxicity have remained stable,” the agency said in a statement. William Blair analysts commented in a research note: “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 minimise red tape and increase access to potentially curative treatments for patients.” The agency said that safety information can be adequately conveyed using drug product labelling. This includes a boxed warning for the risks of CRS and neurological toxicities. A Gilead spokesperson told Pharmaceutical Technology : “This [decision] underscores the knowledge and experience that CAR-T treaters have gained to safely administer these therapies, and we are pleased that they will help lessen the burden on healthcare professionals and patients, enabling more people to receive these potentially curative treatments.” While continuous monitoring and assessment will still be carried out by the FDA, the removal of REMS is expected to facilitate patient access and is viewed as a positive development for the sector. “FDA expects that the REMS elimination, and these labelling updates, will help improve access to these products, particularly for patients who live in rural areas, while ensuring safe and effective administration to patients who need them,” the agency commented. William Blair analysts agreed, saying that “the removal of the REMS requirements could increase access and use of these therapies in the community settings”. “This update also suggests we may not see REMS requirements for CD19 and BCMA autologous CAR-T cell therapies in the future,” the research note added. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Navigating cell therapy process development The cell therapy manufacturing sector is rapidly evolving, driven by the need to balance innovative process development with rigorous GMP standards. 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