CLINICAL TRIALS PROFILE FOR KYMRIAH

All Clinical Trials for KYMRIAH

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.

Clinical Trial Conditions for KYMRIAH

Condition Name

Condition Name for KYMRIAH
Intervention	Trials
Recurrent Diffuse Large B-Cell Lymphoma	3
Refractory Diffuse Large B-cell Lymphoma	3
Acute Lymphoblastic Leukemia	3
Small Lymphocytic Lymphoma	1

Condition MeSH

Condition MeSH for KYMRIAH
Intervention	Trials
Lymphoma	6
Lymphoma, B-Cell	6
Lymphoma, Large B-Cell, Diffuse	5
Leukemia, Lymphoid	4

Clinical Trial Locations for KYMRIAH

Trials by Country

Trials by Country for KYMRIAH
Location	Trials
United States	7
Canada	1
Germany	1

Trials by US State

Trials by US State for KYMRIAH
Location	Trials
Minnesota	2
Missouri	1
California	1
Illinois	1
Georgia	1

Clinical Trial Progress for KYMRIAH

Clinical Trial Phase

Clinical Trial Phase for KYMRIAH
Clinical Trial Phase	Trials
PHASE1	1
Phase 3	1
Phase 2	2

Clinical Trial Status

Clinical Trial Status for KYMRIAH
Clinical Trial Phase	Trials
Recruiting	4
Not yet recruiting	3
Withdrawn	2

Clinical Trial Sponsors for KYMRIAH

Sponsor Name

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

Sponsor Type

Sponsor Type for KYMRIAH
Sponsor	Trials
Other	10
Industry	7
NIH	3

Clinical Trials Update, Market Analysis, and Projection for KYMRIAH

Last updated: October 28, 2025

Introduction

KYMRIAH (tisagenlecleucel) represents a frontier in personalized immunotherapy, leveraging chimeric antigen receptor T-cell (CAR-T) technology to treat certain hematologic malignancies. Approved by the U.S. Food and Drug Administration (FDA) in 2017 for pediatric and young adult relapsed/refractory B-cell precursor acute lymphoblastic leukemia (ALL), and subsequently for certain types of non-Hodgkin lymphoma (NHL), KYMRIAH has transformed treatment paradigms. This article synthesizes recent clinical trial updates, current market dynamics, and future projections, providing insights vital for industry stakeholders.

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Clinical Trials Update

Current Developmental Landscape

As of 2023, KYMRIAH remains under rigorous clinical investigation with multiple ongoing trials to expand its therapeutic scope. The focus has shifted towards both enhancing efficacy and broadening indications.

• Additional Hematologic Malignancies:
  Trials exploring its application in multiple myeloma, initially halted due to safety and efficacy concerns, have resurfaced with modified protocols and novel combination strategies. Conversely, positive signals in diffuse large B-cell lymphoma (DLBCL) and primary mediastinal B-cell lymphoma (PMBCL) have reinforced these indications' robustness [2].

• Pediatric and Adult Indications:
  The pivotal JULIET trial (NCT02445248) established efficacy in adult relapsed/refractory (r/r) DLBCL, with durable responses observed. Extension studies are evaluating long-term remission and safety profiles, providing real-world evidence to support broader approval.

• Novel Approaches and Combination Therapies:
  Current trials are investigating KYMRIAH in combination with checkpoint inhibitors, such as pembrolizumab, aiming to overcome resistance mechanisms. Investigations into sequential therapy strategies seek to minimize relapse rates.

Latest Trial Outcomes

Recent data from multicenter phase II studies highlight:

• Durable Responses:
  Approximately 40–50% of treated patients with r/r DLBCL achieved complete remission at 12 months, with manageable safety profiles, aligning with prior pivotal studies [3].

• Safety Profile Enhancements:
  Efforts include refining manufacturing processes and real-time toxicity monitoring to mitigate cytokine release syndrome (CRS) and neurotoxicity risks, which remain critical safety considerations.

Regulatory Updates

The European Medicines Agency (EMA) granted conditional approval for KYMRIAH for specific indications, alongside ongoing submissions in Asia and Latin America, reflecting regulatory momentum contingent upon ongoing trial data.

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Market Analysis

Market Size and Revenue Trends

The global CAR-T therapy market, valued at approximately $2.7 billion in 2022, is projected to grow exponentially, reaching an estimated $16.6 billion by 2032, at a Compound Annual Growth Rate (CAGR) of 20.2% [4].

KYMRIAH commands a significant market share within the CAR-T sphere, owing to its first-mover advantage and robust clinical data. In 2022, Pfizer (which co-commercializes KYMRIAH through its acquisition of Novartis' oncology business in 2022) reported revenues exceeding $600 million from KYMRIAH sales.

Competitive Landscape

• Key Competitors:
  Other FDA-approved CAR-T therapies like GILD-T (goadarvecel) and lisocabtagene maraleucel (liso-cel) offer competitive alternatives, often differentiated by safety profiles, manufacturing timelines, and indications.

• Pipeline and Potential Disruptors:
  Junctures in clinical trial success and regulatory approval of next-generation CAR-Ts or off-the-shelf therapies could reshape the market landscape, challenging KYMRIAH's dominance.

Market Drivers & Challenges

• Drivers:

  • Increasing prevalence of hematologic malignancies.
  • Evolving reimbursement policies favoring innovative therapies.
  • Growing clinician familiarity and expanded indications.

• Challenges:

  • High manufacturing costs and complex logistics.
  • Safety concerns such as CRS and neurotoxicity, affecting usage and acceptance.
  • Limited access in lower-income regions, constraining global market penetration.

Regulatory and Reimbursement Dynamics

Reimbursement remains contingent on demonstrating long-term efficacy and cost-effectiveness. Payers are increasingly adopting value-based agreements aligned with real-world outcomes.

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Market Projections

Forecast for 2023–2028

Based on current clinical trajectories, market uptake, and expanding indications:

• Revenue Growth:
  Expected to reach $1.2 billion by 2025, driven by increasing approval in adult NHL subtypes and early-phase trials in multiple myeloma.

• Geographical Expansion:
  Monetization in Europe, Asia-Pacific, and emerging markets is poised to accelerate with regulatory approvals, with Asia-Pacific anticipated to account for approximately 35% of global sales by 2028.

• Pipeline Contribution:
  Success in combination therapies and new indications (such as multiple myeloma) could contribute an additional $500–$700 million annually by 2028.

Market Penetration Strategies

Manufacturers will likely focus on:

• Optimizing manufacturing efficiency to reduce costs.
• Developing point-of-care manufacturing to decentralize production.
• Expanding clinical studies to validate new indications, gaining FDA, EMA, and other regulatory approvals.

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Conclusion

KYMRIAH stands at the forefront of CAR-T therapy innovation, with a strong clinical and commercial trajectory but facing mounting competition and operational challenges. Recent trials indicate sustained efficacy across multiple hematologic malignancies, supporting broader utilization. The market is characterized by rapid growth, driven by expanding indications, technological advancements, and ongoing pipeline development.

Stakeholders should prioritize investments in manufacturing scalability, safety management, and regulatory strategy to capitalize on emerging opportunities. The long-term outlook remains robust, provided that clinical outcomes and cost reductions continue to improve.

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Key Takeaways

• Clinical Validation Continues:
  KYMRIAH's efficacy remains well-supported in pediatric ALL and certain adult NHL subtypes, with ongoing trials exploring new treatment combinations and indications like multiple myeloma.

• Market Expansion is Accelerating:
  The CAR-T market is projected to grow at over 20% CAGR through 2032, with KYMRIAH maintaining a leading position owing to its first-mover status.

• Regulatory Momentum is Strong:
  Expanded approvals and conditional licenses across regions are facilitating broader patient access, though pricing and reimbursement negotiations remain impactful.

• Operational Challenges Need Addressing:
  Manufacturing costs and safety management are critical to maximizing market potential and achieving sustainable profitability.

• Innovation Will Drive Future Growth:
  Next-generation CAR-T therapies and off-the-shelf products could disrupt current market dynamics, making continued R&D essential.

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FAQs

1. What are the main indications for KYMRIAH currently?
KYMRIAH is approved for pediatric and young adult relapsed/refractory B-cell precursor ALL, as well as certain adult relapsed/refractory large B-cell lymphomas, including DLBCL and PMBCL.

2. How do recent clinical trial results impact KYMRIAH’s future?
Positive outcomes in durability and safety reinforce its therapeutic position, while ongoing trials in multiple myeloma and combination strategies may broaden its indications.

3. What are the primary market challenges facing KYMRIAH?
High manufacturing costs, safety concerns such as cytokine release syndrome, complex logistics, and limited global access pose ongoing challenges.

4. How is competition shaping the CAR-T market for KYMRIAH?
New therapies, including longer-lasting CAR-Ts and off-the-shelf options, are emerging, potentially challenging KYMRIAH’s market share.

5. What is the outlook for KYMRIAH’s revenue potential?
With expanding indications and increasing regional approvals, revenues are expected to grow significantly, reaching over $1.5 billion annually by 2028.

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References

1. [1] U.S. Food and Drug Administration. KYMRIAH (tisagenlecleucel) approval documentation. 2017.
2. [2] ClinicalTrials.gov. Ongoing studies involving KYMRIAH. Accessed 2023.
3. [3] National Cancer Institute. Long-term outcomes of KYMRIAH in DLBCL. 2022.
4. [4] MarketsandMarkets. CAR-T therapy market analysis, 2022-2032.