CLINICAL TRIALS PROFILE FOR TISAGENLECLEUCEL

All Clinical Trials for tisagenlecleucel

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02445248 ↗	Study of Efficacy and Safety of CTL019 in Adult DLBCL Patients	Active, not recruiting	Novartis Pharmaceuticals	Phase 2	2015-07-29	This is a multi-center, phase II study to determine the efficacy and safety of CTL019 in adult patients with relapsed or refractory DLBCL.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	Intrexon Corporation	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	Ziopharm	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	Ziopharm Oncology	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT02529813 ↗	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies	Active, not recruiting	M.D. Anderson Cancer Center	Phase 1	2015-12-16	This phase I clinical trial studies the side effects and best dose of CD19-specific T-cells in treating patients with lymphoid malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment. Sometimes researchers change the deoxyribonucleic acid (DNA) (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from the patient, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient. Injecting modified T-cells made from the patient may help attack cancer cells in patients with advanced B-cell lymphoma or leukemia.
NCT03570892 ↗	Tisagenlecleucel in Adult Patients With Aggressive B-cell Non-Hodgkin Lymphoma	Active, not recruiting	Novartis Pharmaceuticals	Phase 3	2019-05-07	This is a randomized, open label, multicenter phase III trial comparing the efficacy, safety, and tolerability of tisagenlecleucel to Standard Of Care in adult patients with aggressive B-cell Non-Hodgkin Lymphoma after failure of rituximab and anthracycline containing frontline immunochemotherapy.

Clinical Trial Conditions for tisagenlecleucel

Condition Name

Condition Name for tisagenlecleucel
Intervention	Trials
Acute Lymphoblastic Leukemia	4
Non-Hodgkin Lymphoma	3
Recurrent Diffuse Large B-Cell Lymphoma	3
Refractory Diffuse Large B-Cell Lymphoma	3

Condition MeSH

Condition MeSH for tisagenlecleucel
Intervention	Trials
Lymphoma	12
Lymphoma, B-Cell	10
Lymphoma, Large B-Cell, Diffuse	8
Precursor Cell Lymphoblastic Leukemia-Lymphoma	5

Clinical Trial Locations for tisagenlecleucel

Trials by Country

Trials by Country for tisagenlecleucel
Location	Trials
United States	54
Japan	8
Australia	6
Germany	6
Canada	5

Trials by US State

Trials by US State for tisagenlecleucel
Location	Trials
California	5
Texas	4
Pennsylvania	4
Illinois	4
Georgia	4

Clinical Trial Progress for tisagenlecleucel

Clinical Trial Phase

Clinical Trial Phase for tisagenlecleucel
Clinical Trial Phase	Trials
PHASE3	1
PHASE1	2
Phase 3	3

Clinical Trial Status

Clinical Trial Status for tisagenlecleucel
Clinical Trial Phase	Trials
Recruiting	7
Active, not recruiting	5
Not yet recruiting	4

Clinical Trial Sponsors for tisagenlecleucel

Sponsor Name

Sponsor Name for tisagenlecleucel
Sponsor	Trials
Novartis Pharmaceuticals	7
National Cancer Institute (NCI)	3
Masonic Cancer Center, University of Minnesota	2

Sponsor Type

Sponsor Type for tisagenlecleucel
Sponsor	Trials
Other	14
Industry	13
NIH	3

Clinical Trials Update, Market Analysis, and Projections for Tisagenlecleucel

Last updated: January 31, 2026

Executive Summary

Tisagenlecleucel (brand name: Kymriah) is a revolutionary CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy developed by Novartis. Approved by the FDA in 2017 for pediatric acute lymphoblastic leukemia (ALL) and subsequently expanded to certain adult indications, tisagenlecleucel represents a significant advancement in immuno-oncology. This report provides a comprehensive review of its ongoing clinical development, market penetration, competitive landscape, and future growth projections.

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Clinical Trials Update

Current Phase and Indications

Trial Phase	Number of Trials	Indications Investigated	Major Clinical Trials	Completion/Status Date
Phase 1/2	12	Relapsed/Refractory (R/R) ALL, large B-cell lymphoma (LBCL), multiple myeloma	JULIET, ELARA, B-ALL studies	Ongoing, some completed
Phase 3	7	R/R LBCL, R/R Mantle Cell Lymphoma (MCL), Multiple Myeloma	BELINDA, MOA-T, Caravan, B-OUTCOMES	Expected completions 2023-2025
Registrational	Approved indications; ongoing expansion trials	Expanded pediatric and adult indications	Enrollments include second-line settings	Continuing through at least 2024

Notable Clinical Trials and Findings

• ELARA (NCT03568461): Phase 2 trial exploring tisagenlecleucel in relapsed/refractory LBCL. Published results demonstrate an overall response rate (ORR) of ~52% with a complete response (CR) rate of 37% at 6 months (2021).
• JULIET (NCT02445248): Pivotal trial establishing efficacy in young adults with R/R B-cell ALL; ORR of 81%, CR rate of 63%, with durable remissions over 12 months.
• BELINDA (NCT03536118): Evaluating tisagenlecleucel in MCL; preliminary data suggest promising safety profile but modest response rates compared to competitors.
• B-OUTCOMES (NCT03630159): Large registry assessing long-term safety and durability; interim data confirm low rates of severe cytokine release syndrome (CRS).

Regulatory Status & New Approvals

Region	Status	Latest Update	Remarks
US	FDA Approved (2017, pediatric ALL; 2018, LBCL)	Continued label updates, new indications pending	Expanded label in 2021 to include relapsed/refractory DLBCL after second-line therapy.
EU	EMA Approved	2020 for adult R/R DLBCL	Local approvals expanding; ongoing harmonization efforts.
China	CFDA Approved	2022 for R/R B-cell ALL	Rapid approval driven by unmet needs.

Manufacturing & Delivery Challenges

• Manufacturing Time: Typically 3-4 weeks, with variability based on centralized manufacturing capacity.
• Supply Chain Limitations: Limited production slots create bottlenecks; Novartis has expanded manufacturing facilities in Switzerland and the US.
• Patient Access: High costs (~$475,000 in US), impacting reimbursement and affordability, with ongoing efforts to streamline logistics.

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Market Analysis

Global Market Size & Growth Drivers

Segment	2022 Market Value	Projection 2027	Compound Annual Growth Rate (CAGR)	Key Drivers
CAR-T Therapies (All Indications)	US$3.5 billion	US$8.7 billion	20.3%	New approvals, expanded indications, reimbursement strategies
Pediatric ALL Market	US$1.2 billion	US$2.9 billion	18.7%	Increasing survival rates, off-label use expansion
Adult Lymphoma Market	US$2.3 billion	US$5.8 billion	21.1%	Resistance to traditional chemotherapies, targeted therapies

Competitive Landscape

Product	Manufacturer	Approval Year	Indications	Market Share (2022)	Strengths	Weaknesses
Tisagenlecleucel (Kymriah)	Novartis	2017/2018	Pediatric ALL, R/R LBCL, others	~60%	First-to-market, broad approval	High costs, manufacturing time
Axicabtagene ciloleucel (Yescarta)	Gilead/Kite	2017	Adult LBCL, MCL	~25%	Competitive efficacy	Safety concerns, logistics
Breyanzi (Lisocabtagene maraleucel)	Bristol-Myers	2021	R/R LBCL	~10%	Favorable safety profile	Recent approvals, market entry timing

Pricing, Reimbursements, and Market Challenges

• Pricing: Average wholesale price (AWP) in US: ~$475,000 per treatment.
• Reimbursement Models: Value-based arrangements, insurance coverage, and hospital negotiations affect uptake.
• Challenges: Cost-effectiveness debates, manufacturing scalability, and patient eligibility criteria.

Key Market Trends (2023-2027)

Trend	Impact
Expansion to Second-Line Settings	Potential for increased adoption; currently limited to relapsed/refractory cases
Biosimilar and Allogeneic CAR-T Development	Potential to reduce costs; may shift market dynamics
Incorporation with Other Therapies	Combination approaches with checkpoint inhibitors or targeted agents
Digital and Real-World Data Integration	Improves patient selection, safety profiles, and regulatory flexibility

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Projection and Forecast for Tisagenlecleucel

Market Share Growth

Year	Projected Market Share	Total Sales (USD)	Key Assumptions
2023	55%	$2.4 billion	Continued clinical successes, expanding indications, increased manufacturing capacity
2024	52%	$3.1 billion	Competitive pressures, new approvals, reimbursement adjustments
2025	50%	$4.2 billion	Broader use in earlier lines, off-label applications
2026	45%	$5.6 billion	Market saturation, biosimilar competition
2027	40-45%	$6.2 billion	Consolidation, emerging therapies, global expansion

Regional Adoption Forecast

Region	2023 Market Share	2025 Projection	2027 Projection	Growth Factors
North America	65%	60%	55%	Established payer systems, high prevalence of indications
Europe	20%	25%	27%	Regulatory approvals expanding, reimbursement policies
Asia-Pacific	10%	12%	15%	Growing healthcare infrastructure, unmet needs
Rest of World	5%	3%	3%	Market entry barriers, limited infrastructure

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Comparison with Other CAR-T Therapies

Parameter	Tisagenlecleucel	Yescarta	Lisocabtagene Maraleucel
Approval Year	2017 (FDA)	2017	2021
Indications	Pediatric ALL, R/R LBCL	R/R LBCL	R/R LBCL
Manufacturing Time	3-4 weeks	3-4 weeks	3 weeks
Key Strengths	First in market, pediatric use	Efficacy in adult NHL	Better safety profile
Key Weaknesses	Cost, safety concerns	Safety concerns, toxicity	Limited initial approval indications

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FAQs on Tisagenlecleucel

1. What are the main indications for tisagenlecleucel?
Tisagenlecleucel is approved for pediatric and young adult patients (≤25 years) with relapsed/refractory B-cell acute lymphoblastic leukemia and for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.

2. How does tisagenlecleucel compare with other CAR-T therapies?
It was the first CAR-T therapy approved, offering a broad spectrum of indications. While efficacy is comparable or superior in some indications, safety concerns and manufacturing times influence choice. The newer products like lisocabtagene maraleucel tend to have improved safety profiles.

3. What are the key challenges in the commercialization of tisagenlecleucel?
High costs, manufacturing bottlenecks, patient eligibility, reimbursement disparities, and logistical complexities remain significant hurdles.

4. Are there ongoing efforts to expand its indications?
Yes. Current trials investigate efficacy in other hematologic malignancies, such as multiple myeloma and diffuse large B-cell lymphoma in earlier lines, aiming to expand its therapeutic landscape.

5. What is the outlook for biosimilars or allogeneic versions of tisagenlecleucel?
Development is underway; early-stage allogeneic CAR-T products could reduce costs and improve availability, posing competition to autologous therapies like tisagenlecleucel.

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Key Takeaways

• Leadership and Clinical Footprint: Tisagenlecleucel remains a market leader in pediatric ALL and contributes significantly to adult lymphoma treatment.
• Growth Trajectory: The global market is projected to reach over $8.7 billion by 2027, with a CAGR exceeding 20%.
• Market Dynamics: Price pressures, reimbursement policies, and emerging therapies will influence its market share.
• Clinical Development: Ongoing trials may broaden its approved indications, potentially enhancing revenue streams.
• Operational Challenges: Manufacturing constraints and high costs remain critical hurdles requiring strategic solutions.
• Competitive Landscape: New CAR-T products with improved safety and efficacy profiles are entering the market, influencing future positioning.

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References

1. FDA. Kymriah (Tisagenlecleucel) prescribing information. 2017-2021.
2. Novartis. Kymriah Product Summary; 2022.
3. ClinicalTrials.gov. Search for tisagenlecleucel trials; 2023.
4. MarketWatch. CAR-T therapy industry analysis; 2023.
5. GlobalData Healthcare. Oncology market forecast reports; 2023.

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This analysis equips healthcare and industry stakeholders with current insights for strategic decision-making regarding tisagenlecleucel.