RegulationAccelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (South Korea), Regenerative Medicine Advanced Therapy (United States), Priority Review (United States), PRIME (European Union)

Structure/Sequence

Sequence Code 631391916

Clinical Trials associated with Tisagenlecleucel

NCT04419909

/ Not yet recruitingPhase 1IIT

Retreatment With CTL019/CTL119 in Patients With Late Relapse of B-Cell Lymphomas

This research study is designed to evaluate the effects of retreatment with CTL019/CTL119 in patients with late relapse of B-cell lymphomas.

Start Date01 May 2026

Sponsor / Collaborator

University of Pennsylvania

NCT06785818

/ RecruitingNot Applicable

A Registry to Assess Long-Term Safety of Patients With B-Lymphocyte Malignancies Treated With Tisagenlecleucel in South Korea

This study is multicenter, primary data collection, non-interventional registry study to assess long-term safety, secondary malignancy risk, and effectiveness of tisagenlecleucel in patients with B-cell malignancies in a routine clinical practice setting in Korea.

Start Date12 Mar 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06408194

/ RecruitingPhase 1IIT

Phase I/Ib Clinical Trial of Autologous CD22 Chimeric Antigen Receptor (CAR) T Cells Following Commercial CD19 CAR T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies

The primary purpose of this study is to determine safety, feasibility, and the Maximum Tolerated Dose (MTD)/Recommended Phase 2 Dose (RP2D) of CD22 Chimeric Antigen Receptor T-Cell Therapy (CART) cells when administered 28 to 42 days after an infusion of a commercial CAR called Tisagenlecleucel, to children and young adults with relapsed or refractory B-cell leukemia.

Start Date13 May 2024

Sponsor / Collaborator

Stanford University

100 Clinical Results associated with Tisagenlecleucel

100 Translational Medicine associated with Tisagenlecleucel

100 Patents (Medical) associated with Tisagenlecleucel

654

Literatures (Medical) associated with Tisagenlecleucel

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Navigating the economic value of treatment sequencing in large B-cell lymphoma: insights into optimizing value and patient outcomes of CAR T-cell and other available therapies in Brazil

Article

Author: Nabhan, Samir ; Kievit, Bradley ; Ball, Graeme ; Blissett, Rob

BACKGROUND:

The advent of chimeric antigen receptor t-cell therapy (CAR T) has ushered in a new treatment paradigm in relapsed or refractory (R/R) diffuse large b-cell lymphoma (DLBCL) in which patients have the potential to be cured of their disease. However, as a result of non-clinical barriers and eligibility misinterpretations, patients may not receive CAR T therapy and are instead misallocated to treatment pathways which may result in suboptimal costs and outcomes. We sought to quantify the financial impact of these misallocations across the treatment sequences of therapies available to patients with R/R DLBCL in Brazil.

METHODS:

We developed a patient-level discrete event simulation model to compare treatment options within the sequence of available therapies for DLBCL in Brazil. A cost offset analysis was performed to analyze the financial impact of possible DLBCL treatment sequences with axicabtagene ciloleucel (axi-cel), epcoritamab, and standard-of-care (SoC) therapies. Sensitivity analyses were conducted to examine costs and outcomes for axi-cel compared to tisagenlecleucel (tisa-cel), another available CAR T therapy in Brazil.

RESULTS:

We found that using axi-cel before epcoritamab in the 3L setting was cost-saving compared to using epcoritamab before axi-cel. Compared to both epcoritamab and SoC therapies, cost offsets were observed across all treatment lines due to the prolonged survival of treating with axi-cel compared to other options, and savings were greater when axi-cel was administered earlier in the treatment sequence. Sensitivity analyses demonstrated that treating with axi-cel in 3L was less expensive than treating with tisa-cel.

CONCLUSION:

Administering CAR T therapies like axi-cel before epcoritamab or SoC in R/R DLBCL can lead to substantial cost offsets as fewer patients ultimately progress to subsequent treatment lines. Optimal R/R DLBCL outcomes and costs can be achieved in Brazil through maximizing the potential for cure by treating eligible patients with axi-cel ahead of other available therapies.

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Budget impact of introducing glofitamab for treatment of relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy in the United States

Article

Author: Li, Jia ; Mahmoudjafari, Zahra ; Masaquel, Anthony ; Parisé, Hélène ; Bercaw, Eric ; Bognar, Katalin ; Wang, Si-Tien

BACKGROUND:

Glofitamab is a T-cell engaging bispecific monoclonal antibody that was granted accelerated approval from the United States Food and Drug Administration for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified or large B-cell lymphoma arising from follicular lymphoma, after ≥2 lines of systemic therapy (3L+).

METHODS:

A budget impact model was developed for a hypothetical blended commercial/Medicare health plan with 1,000,000 members. Comparators were axicabtagene ciloleucel (Axi-cel), lisocabtagene maraleucel (Liso-cel), tisagenlecleucel (Tisa-cel), loncastuximab tesirine, polatuzumab vedotin + bendamustine + rituximab, rituximab + gemcitabine + oxaliplatin, tafasitamab + lenalidomide, and epcoritamab (Epcor). Total costs included those for drugs, wastage, administration, grade ≥3 adverse reactions, and all-grade cytokine release syndrome) and routine care. Market shares were based on internal projections and expert opinions. Total and per-member per-month (PMPM) net budget impacts over 3 years were calculated.

RESULTS:

Approximately nine patients were projected to be eligible for 3L + DLBCL treatment in a health plan of 1,000,000 members. The introduction of glofitamab as a treatment option resulted in estimated total and PMPM cost savings of $728,697 and -$0.0202, respectively, over 3 years. Costs were reduced across all cost categories but particularly in drug costs. Among the newer therapies, total 3-year cost per treated patient was lowest for glofitamab: $226,658 versus Tisa-cel = $564,113; Axi-cel = $540,002; Liso-cel = $516,272; and Epcor = $335,293. Across all sensitivity analyses, the inclusion of glofitamab had minimal PMPM budget impact, ranging from -$0.0256 to -$0.0108.

CONCLUSIONS:

With the lowest 3-year total cost per treated patient among the newer therapies, glofitamab being an available option in the 3L + DLBCL market is estimated to save a hypothetical 1,000,000-member health plan $728,697 in cumulative total costs and $0.0202 in PMPM costs over 3 years.

01 Dec 2025·TRANSFUSION AND APHERESIS SCIENCE

Novel leukocytapheresis method using highly concentrated sodium citrate solution for the manufacturing of tisagenlecleucel

Article

Author: Fujii, Keiko ; Maeda, Yoshinobu ; Ikeuchi, Kazuhiro ; Kitamura, Wataru ; Abe, Masaya ; Otsuka, Fumio ; Washio, Kana ; Fujii, Nobuharu

For the manufacturing of tisagenlecleucel (tisa-cel) requires the non-mobilized mononuclear cell collection (MNC). CD3⁺ cell collection is performed using the same protocol as autologous peripheral blood stem cell harvest (auto-PBSCH), but this procedure necessitates the same target CD3⁺ cell yields regardless of age or body weight, which may take several days especially in pediatric and small female patients with low white blood cell counts. We previously demonstrated a novel method using highly concentration sodium citrate (HSC), which reduced the need for an anticoagulant (AC) solution and shortened the procedure time in auto-PBSCH. This novel method was expected to offer advantages for smaller patients, prompting us to investigate its application in leukocytapheresis for the manufacturing of tisa-cel. We retrospectively analyzed consecutive leukocytapheresis data obtained using Spectra Optia continuous MNC mode between November 2022 and June 2024 at our institution (n = 9). In six of nine patients, pre-leukocytapheresis CD3⁺ cell counts were less than 500 /μL, but all could obtain the target CD3⁺ cell yields in one day upon processing blood volume adjustment. When we compared patients who had received CD3⁺ cell collection using normal-concentration sodium citrate (NSC) as our previously reported using propensity score-matched pair analysis, the total AC solution volume was significantly lower (1168 vs. 316 mL, p < 0.001) and procedure time was significantly shorter (254 vs. 228 min, p = 0.04) in the HSC group compared to the NSC group. In conclusion, this procedure was also useful for non-mobilized MNC. Our findings warrant validation in a larger patient cohort.

288

News (Medical) associated with Tisagenlecleucel

09 Dec 2025

·www.fiercebiotech.com

Rainbows, pickleball and puppies: Inside the ‘enduring activism’ and ‘cautious optimism’ at ASH 2025

The mural was adorned with buttons free for the attendees’ taking, which featured pro-science messages like: “What do we want? Evidence-based science; When do we want it? After peer review.”\n People come to Orlando, Florida to escape. With Disney World, SeaWorld and Universal Studios all in the area, there are endless opportunities to leave behind the challenges of the real world for a bit of magic and relaxation. But if hematologists walking into the American Society of Hematology (ASH) annual meeting—which is being held at the Orange County Convention Center in Orlando from Dec. 6 to 9—were hoping to forget about the struggles facing science in the U.S. for just a few days, they likely were disappointed. Imagery around the conference, especially in the main entry, repeatedly referenced the dire situation that U.S. science faces amid the second Trump administration.A large mural reading “Stand Up for Science” was adorned with buttons free for the attendees’ taking. The pins featured pro-science messages like: “What do we want? Evidence-based science; When do we want it? After peer review.” On the reverse side, a rainbow mural celebrating diversity in hematology stood out—a stark contrast to the anti-DEI policies that have become a staple of the Trump administration\'s leadership.Columns were decked out with a message from outgoing ASH President Belinda Avalos, M.D., encouraging attendees to “fight for science, fight for research, fight for hematology, and fight for our patients.” The slogan “Fight 4 Hematology” served as decoration on the conference floor and tables, and guidance on how hematologists can advocate for their field was plastered next to Avalos’ message and on a large flight of stairs.But step a little further into ASH Central and the fun-filled Orlando atmosphere takes over. Take your pick: Play a game of pickleball? Take a snooze in a futuristic nap pod? Pet some adorable puppies? It was here that the true nature of 2025’s ASH meeting revealed itself.“You\'re seeing an enduring activism,” Jake Van Naarden, president of Lilly Oncology for Eli Lilly, told Fierce Biotech at the conference. “The academic funding situation and the challenges they\'re in is real,” he said.Given the long-standing nature of the activism, the topic isn\'t dominating conversations at the conference, Van Naarden added. Instead, the focus has been on new data, advances in care and catching up with old friends. Van Naarden’s sentiments were echoed by multiple other attendees that Fierce spoke with, and match the mood at the American Society of Clinical Oncology meeting this past spring.“This is exactly what my experience is here, too,” Anne Kerber, M.D., head of hematology, oncology and cell therapy at Bristol Myers Squibb, told Fierce at ASH. “The overall field has been making such tremendous progress for patients,” she said, adding that the focus on that progress “unites this whole ASH community.”That community came out in full-force to Florida. A record-breaking number of abstracts were submitted to the conference, with more than 8,200 accepted, Avalos said during a Dec. 1 media preview.“You could go across this conference and you would see so many potential breakthroughs for patients with hematological disease,” P.K. Morrow, M.D., Takeda’s head of oncology, told Fierce. “I feel like ASH is meeting the moment in terms of staying scientifically grounded.”Morrow called the conference “really exceptional,” a description that became clear during the late-breaking abstract presentations on Dec. 9. That\'s when Phoebe Ho, Ph.D., of the University of Sydney, presented data on the first four patients treated with Kelonia Therapeutics’ in vivo CAR-T therapy for relapsed and refractory multiple myeloma.No cancer was detected in any of the patients one month after treatment, as measured by flow cytometry and genetic sequencing. After Ho\'s talk, attendees lined up to ask questions, starting their queries by first describing the data as “beautiful” and “fantastic.” Robert Negrin, M.D., a hematologist at Stanford University and the incoming president of ASH, told Ho the study\'s result \"takes your breath away.\" In vivo CAR-T, where gene therapy is used to tweak T cells so that they target cancer, has surged in popularity this year. The method is designed to improve upon ex vivo CAR-T, where the cells are removed from patients and gene edited in the lab before being reinfused, a field that kicked off with the 2012 treatment of pediatric leukemia patient Emily Whitehead and subsequent approval of Novartis’ Kymriah (tisagenlecleucel).“We\'re excited about in vivo,” Cindy Perettie, executive vice president and global head of Gilead’s cell therapy unit Kite Pharma, told Fierce at ASH before Ho’s presentation. Kite presented data for its buzzy next-gen ex vivo CAR-T anito-cel at the conference, and has also made recent buys into in vivo.“I\'m really excited by the meeting. We\'re seeing a lot of really interesting data,” she added. “There\'s a lot of options for patients now.” Cautious optimismDeveloping new treatments requires strong science, but it also demands extensive capital to fund clinical trials and a stable regulatory environment that enables advance planning. On these fronts, 2025 has been a year of concern.There were no biotech IPOs from February to September, a prolonged dry period, until LB Pharmaceuticals broke the spell. Profound layoffs and leadership turnover at the FDA has prompted fears of disrupted drug reviews and the loss of American biotech leadership. And Trump policies around “most favored nation” (MFN) drug pricing and pharmaceutical tariffs have caused consternation throughout the year.Now, the industry may have found its footing in the more turbulent world. “People are at a point where there\'s cautious optimism,” Adam Rosenthal, Ph.D., founder and CEO of Star Therapeutics, told Fierce at ASH. “I think on a lot of uncertainties that were there earlier this year, like around tariffs and MFN, that seems to be at a steadier spot.”“On the FDA, that just continues to be a new surprise every month,” he continued. “We\'ll see if and when that settles out.”The ASH meeting launched mere days after longtime FDA oncology leader Richard Pazdur, M.D., announced plans to retire just weeks after ascending to head up the agency\'s Center for Drug Evaluation and Research.Multiple executives from Big Pharma companies told Fierce at ASH that despite the chaos gripping the FDA, their interactions with the agency haven’t changed.“Merck continues to work closely with the FDA on our development programs and we appreciate the patient-centered focus that the FDA has,” Gregory Lubiniecki, M.D., vice president of global development at Merck Research Laboratories, told Fierce. Kite’s interactions with the agency “have been going really well,” Kite\'s Perettie said. “And we continue open dialog with them on our next-generation products.”Van Naarden, Morrow and Kerber also said they haven’t seen changes in their relations with the agency at Lilly, Takeda and BMS, respectively.“We\'re actually not seeing any differences, to be honest,” Hesham Abdullah, M.D., GSK’s global head of oncology, told Fierce at the conference. “From our end, things are continuing to run as planned.”“We don\'t get special treatment,” Lilly cancer leader Van Naarden said. “If we haven\'t been seeing it, I assume no one’s seeing that as yet.”But that\'s not necessarily the case. While ASH was ongoing, on Dec. 7, the FDA missed an approval decision deadline for a new indication of Agios Pharmaceuticals’ Pyrukynd (mitapivat) in thalassemia. Along with Agios, numerous other smaller pharmas and biotechs have faced delays or surprising decisions from the FDA since Trump took office in January. Denali Therapeutics, Regenxbio, Neurizon, Coya Therapeutics, KalVista, Stealth Biotherapeutics and Omeros have all been dealt delays or missed deadlines by the agency, despite FDA Commissioner Marty Makary, M.D., pledging in June that the agency was “on track” to meet its deadlines. Others, like Capricor Therapeutics and Replimune, have been handed high-profile drug rejections for reasons that reportedly contradict past guidance they’ve received from the FDA.When Fierce mentioned that the agency has missed some PDUFA deadlines, Van Naarden highlighted the FDA’s recent on-time approvals of Lilly\'s breast cancer med Inluriyo (imlunestrant) and expanded label for BTK inhibitor Jaypirca (pirtobrutinib).“This is what it normally looks like,” he said of the recent approvals. “I don\'t know if that changes in the future.\"The discrepancy between the words of Big Pharma leaders and the experiences of smaller companies raises questions about possible FDA favoritism for certain companies, a concern also raised by lawmakers currently probing the agency’s newly launched “national priority” voucher program.“FDA does not give preferential treatment to any company, and any suggestion that reviews are influenced by a company’s size or name is simply wrong,” Andrew Nixon, the head of communications for the Department of Health and Human Services, which oversees the FDA, told Fierce Biotech. The fight goes onThough the data has been strong and the mood buoyant at this year’s ASH meeting, that doesn’t mean the fight for hematology care has been won.“This has been a really unprecedented time for us in terms of funding by our U.S. federal government, and we have been very concerned about the ability to continue to be able to do research,” outgoing ASH leader Avalos told Fierce. Hematologists have largely answered the call, she said, advocating for their field at the local or national level, donating to the ASH foundation and sharing stories about the importance of their work for patients.When the Trump administration began cutting federal science funding from the National Institutes of Health and elsewhere, ASH responded by pledging more than $12 million to cover for lost grants. The group has now increased the number of awards available to $20 million this year, Avalos said.“Obviously we can\'t sustain that, but we\'re hoping that there will be interventions that occur over the next year at least,” she explained.And as suggested by the rainbow mural featured at the conference, ASH has not shied away from the importance of diversity in hematology. In December of last year, the organization conducted a pair of surveys to identify roadblocks to improving diversity in hematology trials and elucidate the strategies ASH members use to advance DEI in their own research.Part of ASH’s commitment to diversity, which also includes a series of funding opportunities across career levels, stems from hematology’s own history with sickle cell disease. The condition predominantly affects people of African origin, and the disease has long suffered from neglect from the medical community.“Sickle cell disease has been one of the diseases that has been long ignored,” Avalos said. “We\'ve known the cause of sickle cell disease for many, many decades, yet very few treatments have been available.”ASH now runs the Center for Sickle Cell Disease Initiatives, which launched in 2015, and the disease was the topic of several special interest sessions at this year’s meeting.Like many of the other attendees, Avalos was thrilled by the quality of data presented at ASH and thinks the optimistic vibe is a good sign for hematology’s future.“Optimism is important,\" she said. \"Because that\'s what keeps driving us.\"

Cell TherapyASH

25 Nov 2025

·www.pharmaceutical-technology.com

UK-developed leukaemia cell therapy to be offered on NHS

NICE took ALL’s severity into account when weighing up the treatment’s benefits. Credit: pinkeyes via Shutterstock.com. A UK-developed cell therapy to treat a form of leukaemia has been greenlit for use on England’s National Health Service (NHS). The National Institute for Health and Care Excellence (NICE), the NHS spending watchdog, recommended Aucatzyl (obecabtagene autoleucel) for the treatment of relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL). ALL is a rare and aggressive type of blood and bone marrow cancer, with the B-cell subtype being more common than T-cell leukaemia. Standard treatment for the condition involves chemotherapy and steroids, though targeted treatments are also approved for NHS use. NICE stated that its independent committee applied greater weighting to the benefits that the new therapy would offer over current treatments due to B-cell ALL’s severity. Developed by University College London spinout Autolus Therapeutics, Aucatzyl is a CAR-T treatment. This personalised modality involves genetically modifying the patient’s own T cells to recognise and fight cancer cells. Autolus said it will launch Aucatzyl in England and Wales imminently, with plans also in place to pursue patient access in Scotland. 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 NICE’s director of medicines evaluation Helen Knight said: “This drug has the potential to offer a more effective and less toxic alternative to standard treatments, with fewer side effects. This could potentially be a life-saving drug, which will make a huge difference to people’s lives, including spending less time in hospital.” Evidence from a clinical trial has shown high rates of remission in patients whose cancer has either returned after treatment or failed to respond to initial therapy. In the pivotal FELIX study (NCT04404660), 77% of those who had at least one Aucatzyl infusion went into remission. NICE said evidence suggests that Autolus’ drug improves survival when compared to other immunotherapies. NICE reports that Aucatzyl works just as well as Kymriah (tisagenlecleucel), another CAR-T developed by Novartis that was recommended for ALL patients on the NHS in 2024. Kymriah is available for people aged 25 years and under, while Aucatzyl is recommended for those aged 26 years and over. A third CAR-T, Kite Pharma’s Tecartus (brexucabtagene autoleucel), is also available to ALL patients aged 25 years and over. The NICE recommendation is the latest vote of confidence from UK regulatory agencies on advanced therapies, despite a handful of pharma companies withdrawing from the sector this year. In August 2024, the Association of the British Pharmaceutical Industry (ABPI) called on the NHS to take more action to adopt more cell and gene therapies, highlighting that a one-time treatment can save the healthcare system’s money over a patient’s lifetime, despite the high upfront cost. NICE has since greenlit several new advanced therapies, including Casgevy (exagamglogene autotemcel) for the treatment of sickle cell disease. The therapy was the world’s first approved medicine using the CRISPR/Cas9 gene editing system. The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has also outlined major changes to regulation for rare disease treatments, saying it will speed up development pathways so therapies can reach patients sooner. 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 eBook 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 eBook. 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 . Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

Cell TherapyImmunotherapyDrug Approval

29 Oct 2025

·www.biospace.com

Novartis Presents New Data From Potential First Targeted Sjögren’s Treatment

iStock/ JHVEPhoto Late-breaking Phase III data show ianalumab, Novartis’ dual-targeted antibody, reduced disease activity and patient burden in Sjögren’s disease, the pharma reported at the American College of Rheumatology Convergence congress on Wednesday. It’s the second most common rheumatic autoimmune disease—and there is no targeted treatment available. Novartis aims to change that with ianalumab, presenting new data Wednesday showing reduced disease activity and patient burden in two Phase III trials in Sjögren’s disease, becoming the first drug to do so. Ianalumab, pronounced yan-al-umab, is named after the Roman god Janus for its dual mechanism of action as a B cell depleting antibody that also targets the BAFF receptor. “The core premise was that this dual mechanism should allow us to more effectively deliver tissue level B cell depletion in difficult to treat autoimmune diseases,” Shreeram Aradhye, president of development and chief medical officer at Novartis, told BioSpace in an interview prior to the presentation. That premise seemed to pay off, with ianalumab eliciting a greater reduction in disease activity by week 16 with improvements sustained through week 52 compared to placebo, as measured by the EULAR Sjögren’s Syndrome Disease Activity Index, meeting the studies’ primary objective. In addition, patients treated with ianalumab also saw consistent improvements in dryness, pain and fatigue—all hallmark symptoms of Sjögren’s. The data, from the Phase III NEPTUNUS-1 and NEPTUNUS-2 trials, were presented at the American College of Rheumatology Convergence congress in Chicago on Wednesday. Novartis first announced topline results from NEPTUNUS-1 and NEPTUNUS-2 in August. Aradhye characterized Sjögren’s as a “difficult, heterogeneous disorder,” with varied manifestations. Sjögren’s is estimated to afflict between 3 million and 4 million people in the U.S., around 90% of whom are women, according to Sjogren’s Advocate . Current options for Sjögren’s are only symptomatic treatments, with patients turning to eye drops and unapproved immunomodulators, Aradhye said. The general theme that has emerged, based on discussions with rheumatologists and patient representatives, is that if a targeted drug is approved with “a pro is reasonably safe . . . there is the expectation that people will want to try it,” he added. Novartis plans to submit the Phase III results to global regulators early next year. “It’s an exciting time for our immunology portfolio,” Aradhye said, highlighting both the ianalumab data in Sjögren’s and a successful Phase III readout last week for Cosentyx, first approved in 2015 to treat plaque psoriasis, in people over 50 with polymyalgia rheumatica. Besides Sjögren’s, which is listed on Novartis’ pipeline page as the lead indication for ianalumab, the drug is also in Phase III trials for systemic lupus erythematosus (SLE) and lupus nephritis, in Phase II for systemic sclerosis, and in earlier-stage development for three hematological indications. Aradhye expects the lupus trials to read out in the 2027 timeframe. He explained Novartis’ drug development philosophy. “We’ve had this common theme around saying that when we understand a drug and its mechanism of action, we will evaluate it in multiple indications simultaneously.” Immunology is one of four key therapeutic areas at Novartis, the others being cardiovascular, renal and metabolic, neuroscience and oncology. Novartis has had a “long legacy” in immunology, Aradhye said, pointing to the company’s work in transplantation and with Cosentyx. In this space, Novartis is committed to another emerging theme: the use of CAR T in autoimmune diseases —something that has been front and center at ACR this week. This is somewhat of a natural fit for Novartis, given its ownership of Kymriah, a CD-19 directed CAR T that in 2017 became the first cell-based gene therapy approved by the FDA, to treat acute lymphoblastic leukemia. Cell therapy 5 CAR T Cell Therapies With Autoimmune Readouts in 2025 Having established success in cancer, biopharma is now looking to leverage CAR T therapies against a new target, autoimmune disorders, with several early- to mid-stage readouts expected this year. March 17, 2025 · 9 min read · Tristan Manalac Novartis will also present biomarker data at ACR from a Phase I/II study of its autologous CD-19 CAR T therapy, rapcabtagene autoleucel (YTB323), in SLE. YTB323 has previously shown “good proliferation of the clones, a decrease in B cells, reconstitution over time and then a meaningful control of disease activity” in lupus and lupus nephritis, Aradhye said. Novartis has dominated the biopharma headlines this past week, announcing its intent on Sunday to acquire neuromuscular drug developer Avidity Biosciences for around $12 billion—the year’s second largest deal—and reporting Q3 earnings on Tuesday. Novartis also announced on Tuesday the successful completion of its $1.4 billion purchase of Tourmaline Bio.

Phase 3ImmunotherapyCell TherapyClinical ResultAcquisition

100 Deals associated with Tisagenlecleucel

External Link

KEGG	Wiki	ATC	Drug Bank
-	Tisagenlecleucel	L01	DB13881

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Recurrent B Acute Lymphoblastic Leukemia	South Korea	Novartis AG	05 Mar 2021
Refractory B Acute Lymphoblastic Leukemia	South Korea	Novartis AG	05 Mar 2021
Follicular Lymphoma	Canada	Novartis Pharmaceuticals Canada, Inc.	05 Sep 2018
Acute Lymphoblastic Leukemia	European Union	Novartis Europharm Ltd.	23 Aug 2018
Acute Lymphoblastic Leukemia	Iceland	Novartis Europharm Ltd.	23 Aug 2018
Acute Lymphoblastic Leukemia	Liechtenstein	Novartis Europharm Ltd.	23 Aug 2018
Acute Lymphoblastic Leukemia	Norway	Novartis Europharm Ltd.	23 Aug 2018
Diffuse large B-cell lymphoma recurrent	European Union	Novartis Europharm Ltd.	23 Aug 2018
Diffuse large B-cell lymphoma recurrent	Iceland	Novartis Europharm Ltd.	23 Aug 2018
Diffuse large B-cell lymphoma recurrent	Liechtenstein	Novartis Europharm Ltd.	23 Aug 2018
Diffuse large B-cell lymphoma recurrent	Norway	Novartis Europharm Ltd.	23 Aug 2018
Diffuse large B-cell lymphoma refractory	European Union	Novartis Europharm Ltd.	23 Aug 2018
Diffuse large B-cell lymphoma refractory	Iceland	Novartis Europharm Ltd.	23 Aug 2018
Diffuse large B-cell lymphoma refractory	Liechtenstein	Novartis Europharm Ltd.	23 Aug 2018
Diffuse large B-cell lymphoma refractory	Norway	Novartis Europharm Ltd.	23 Aug 2018
Recurrent Follicular Lymphoma	European Union	Novartis Europharm Ltd.	23 Aug 2018
Recurrent Follicular Lymphoma	Iceland	Novartis Europharm Ltd.	23 Aug 2018
Recurrent Follicular Lymphoma	Liechtenstein	Novartis Europharm Ltd.	23 Aug 2018
Recurrent Follicular Lymphoma	Norway	Novartis Europharm Ltd.	23 Aug 2018
Refractory Follicular Lymphoma	European Union	Novartis Europharm Ltd.	23 Aug 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	United States	Novartis Pharma AG	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	United States	China Novartis Institutes for BioMedical Research Co., Ltd.	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	United States	Novartis Pharmaceuticals Corp.	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	China	Novartis Pharmaceuticals Corp.	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	Japan	China Novartis Institutes for BioMedical Research Co., Ltd.	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	Japan	Novartis Pharmaceuticals Corp.	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	Japan	Novartis Pharma AG	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	Australia	Novartis Pharmaceuticals Corp.	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	Australia	Novartis Pharma AG	07 May 2019
Aggressive B-Cell Non-Hodgkin Lymphoma	Phase 3	Australia	China Novartis Institutes for BioMedical Research Co., Ltd.	07 May 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03568461 (ELARA, ASH2025)	Phase 2	Follicular Lymphoma anti-CD19 chimeric antigen receptor (CAR)	97	Tisagenlecleucel	dsajwrxupk(pjutqzvxbk) = 1 event jrudaenqao (ekoysopqnu ) View more	Positive	06 Dec 2025
				Tisagenlecleucel (CR)
ASH2025	Not Applicable	Large B-cell lymphoma \| Mantle-Cell Lymphoma \| Precursor B-Cell Lymphoblastic Leukemia-Lymphoma	430	Immunoglobulin replacement therapy (IGRT)	frkaxxzdbx(yigqsbuqmc) = Infection was probable in 43 (18.1%). Of episodes with a pathogen identified, 58.3% were viral, 40.7% bacterial, 0.5% fungal and 0.5% other. The primary focus was respiratory in 75%. Infection was grade ≥3 in 44% including grade 5 in 6.8%. ojfgjxppsl (obobbrxtqj ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Secondary Central Nervous System Lymphoma	62	axicabtagene ciloleucel	ewiysncera(nynhbsbjmw) = rkknjviued hnpjkxmlmy (nokyqkieda ) View more	Positive	06 Dec 2025
				tisagenlecleucel	ewiysncera(nynhbsbjmw) = nhzsuuojiq hnpjkxmlmy (nokyqkieda ) View more
ASH2025	Not Applicable	Diffuse large B-cell lymphoma recurrent	632	Tisagenlecleucel (tisa-cel)	kjarrrtcai(jtjkzquksr) = sgvpetpujg jfftftsirr (jcltmilczu ) View more	Positive	06 Dec 2025
				Axicabtagene ciloleucel (axi-cel)	kjarrrtcai(jtjkzquksr) = wjinqtvjlz jfftftsirr (jcltmilczu ) View more
ASH2025	Not Applicable	Diffuse large B-cell lymphoma recurrent	320	Axicabtagene ciloleucel	mdbgpmbhxt(mathazuemo): HR = 1.33 (95.0% CI, 1.04 - 1.7) View more	Positive	06 Dec 2025
				Tisagenlecleucel
ASH2025	Not Applicable	Diffuse Large B-Cell Lymphoma	1,022	Axicabtagene Ciloleucel	xoxgzrbeid(gtarvqlhrg) = orqqoyuhac hspzynjxgn (bvndgvdavm, 23 - 34) View more	Negative	06 Dec 2025
				Glofitamab	xoxgzrbeid(gtarvqlhrg) = dkwtecwpdi hspzynjxgn (bvndgvdavm, 11 - 22) View more
PS1922 (EHA2025)	Not Applicable	T-cell/histiocyte rich large B-cell lymphoma	33	CAR-T (Patients with T Cell Histiocyte-Rich Large B-Cell Lymphoma)	pwdrpvqxzt(myzivcbtqi) = fjonryesks mbuzyxoqbf (euvpnnyfah ) View more	Negative	14 May 2025
ELARA (EHA2025)	Phase 2	Follicular Lymphoma CD19	97	Tisagenlecleucel	ptfmsqiztq(tweigvumin) = median OS was not reached as assessed by independent review committee mhvpybkvwh (jwxgbnqwed ) View more	Positive	14 May 2025
NCT03568461 (ELARA, EHA2025)	Phase 2	Follicular Lymphoma age \| sex \| race ... View more	98	Tisagenlecleucel	cdgcbdikme(iwkhvxjxow) = yepdfaulfz vfmpkkdsyi (walemrmage ) View more	Positive	14 May 2025
				Mosunetuzumab	cdgcbdikme(iwkhvxjxow) = bodavdofin vfmpkkdsyi (walemrmage ) View more
PB3221 (EHA2025)	Not Applicable	Large B-cell lymphoma chemorefractory disease \| elevated lactate dehydrogenase (LDH)	-	Tisagenlecleucel	ykhpsfplno(pczznsdcib) = hmthznaenn wjbxjrhkjt (cnuxhfokkv ) View more	Positive	14 May 2025
				Tisagenlecleucel (Allogeneic HSCT)	ykhpsfplno(pczznsdcib) = hfwrojpnae wjbxjrhkjt (cnuxhfokkv ) View more

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