Tisagenlecleucel

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. This eBook, brought to you by industry leaders Cytiva, explores how biotech firms can address common challenges — including minimizing contamination risks from manual processes, overcoming resource limitations, and creating scalable, robust designs. Industry experts discuss strategies including automation, strategic outsourcing, and flexible equipment selection to streamline processes and reduce regulatory risks. To unlock expert insights on optimizing your cell therapy pathway from clinical development to commercialization, fill in your details now. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic You have a right to withdraw your consent at any time, by clicking here . We may still continue to send you service-related and other non-promotional communications. For more information relating to our privacy practices, we invite you to review our privacy policy . 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Dive Brief:The Food and Drug Administration is easing limitationsit imposed around the complex cancer drugs known as CAR-T therapies, removing several onerous requirements as well as loosening restrictions on which facilities can provide treatment.The FDAs action applies to therapies made by Bristol Myers Squibb, Gilead Sciences, Johnson & Johnson and Novartis that are used to treat several types of blood cancer. Specifically, it removes so-called Risk Evaluation and Mitigation Strategies from the drugs labeling, which are used to help manage serious side effects of treatment.According to the FDA, these REMS requirements are no longer necessary to ensure safe use of these CAR-T therapies as both physicians and hospitals are now well versed in managing the two syndromes most commonly associated with the drugs.Dive Insight:Since the FDAs approval of Novartis Kymriah for leukemia in 2017, six more CAR-T therapies have reached market. These medicines are made from a patients own immune cells, extracted from the body and engineered in a lab to hunt down specific proteins found on the surface of malignant clones.For some people with leukemia, lymphoma or multiple myeloma, they can prompt powerful responses and even long-lasting remission. But their administration also comes with notable risks, including a hyperactive immune response known as CRS and neurological toxicity dubbed ICANS.When CAR-T therapies were new, physicians and the hospitals they worked at faced a learning curve handling these treatment-associated conditions, which require specific management and drugs to bring under control.The REMS imposed by the FDA was designed to help mitigate these risks by requiring hospitals receive special certifications before they could administer treatment, and mandating specialized reporting of side effects to the agency.Given the established management guidelines and extensive experience of the medical hematology [and] oncology community in diagnosing and managing the risks of CRS and neurologic toxicities across products in the class of BCMA- and CD19-directed autologous CAR-T cell immunotherapies, FDA has determined that the safe and effective use of CAR-T cell immunotherapies for the indicated population can be assured without a REMS, the agency said in a Thursday statement. BCMA and CD19 are the protein targets of the seven approved CAR-T therapies.Reporting rates for CRS and ICANS have remained stable, the FDA added.Alongside removal of the REMS, the regulator also reduced its requirement that patients remain nearby the facility they received treatment from one month to two weeks. And its limitation on how long patients must wait before they can drive or operate machinery again after treatment is lowered from two months to two weeks.Removal of the REMS should lower the logistical hurdles patients face in receiving CAR-T treatment as well as the cost of administration, the Alliance for Regenerative Medicine, a trade group, said in a post to LinkedIn.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 U.S. cell therapy market, Daina Graybosch, an analyst at Leerink Partners, wrote in note to clients.In a statement, Bristol Myers, which sells the CAR-T therapies Abecma and Breyanzi, noted that only two in 10 eligible patients receive the treatments due to the confluence of complex logistical and geographic barriers affecting patients and providers.The changes could help expand use of CAR-T from specialized medical institutions, where its most commonly given, to community health centers, Bristol Myers said.Breyanzi, Kymriah and Gileads Yescarta and Tecartus are cleared to treat lymphoma, although Kymriah is more commonly used for leukemia, where it is also approved. Abecma and J&Js Carvykti are used for multiple myeloma.The most recently OKd CAR-T treatment, Autolus Therapeutics Aucatzyl for lymphoma, was the first to be cleared without an initial REMS program. '