Tisagenlecleucel - Biologic Drug Details

Summary for tisagenlecleucel

Tradenames:	1
High Confidence Patents:	0
Applicants:	1
BLAs:	1
Suppliers: see list	1
Recent Clinical Trials:	See clinical trials for tisagenlecleucel

Recent Clinical Trials for tisagenlecleucel

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
Memorial Sloan Kettering Cancer Center	PHASE3
Princess Maxima Center for Pediatric Oncology	PHASE3
Stanford University	PHASE1

See all tisagenlecleucel clinical trials

Pharmacology for tisagenlecleucel

Mechanism of Action	CD19 Receptor Interactions
Physiological Effect	Increased T Lymphocyte Activation
Established Pharmacologic Class	CD19-directed Chimeric Antigen Receptor Genetically-modified Autologous T Cells
Chemical Structure	CD19-specific Chimeric Antigen Receptor

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

1. Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

2. DrugPatentWatch analysis and brand-side disclosures

These patents were identified from searching drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

3. Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for tisagenlecleucel Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for tisagenlecleucel Derived from DrugPatentWatch Analysis and Company Disclosures

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Novartis Pharmaceuticals Corporation	KYMRIAH	tisagenlecleucel	Injection	125646	10,357,514	2035-04-07	DrugPatentWatch analysis and company disclosures
Novartis Pharmaceuticals Corporation	KYMRIAH	tisagenlecleucel	Injection	125646	10,829,735	2036-07-21	DrugPatentWatch analysis and company disclosures
Novartis Pharmaceuticals Corporation	KYMRIAH	tisagenlecleucel	Injection	125646	11,439,665	2041-09-15	DrugPatentWatch analysis and company disclosures
Novartis Pharmaceuticals Corporation	KYMRIAH	tisagenlecleucel	Injection	125646	11,633,430	2042-05-23	DrugPatentWatch analysis and company disclosures
Novartis Pharmaceuticals Corporation	KYMRIAH	tisagenlecleucel	Injection	125646	8,637,307	2030-05-10	DrugPatentWatch analysis and company disclosures
Novartis Pharmaceuticals Corporation	KYMRIAH	tisagenlecleucel	Injection	125646	8,906,682	2033-07-10	DrugPatentWatch analysis and company disclosures
Novartis Pharmaceuticals Corporation	KYMRIAH	tisagenlecleucel	Injection	125646	8,911,993	2033-07-10	DrugPatentWatch analysis and company disclosures

3) Low Certainty: US Patents for tisagenlecleucel Derived from Patent Text Search

These patents were obtained by searching patent claims

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Novartis Pharmaceuticals Corporation	KYMRIAH	tisagenlecleucel	Injection	125646	11,332,451	2040-09-16	Patent claims search

International Patents for tisagenlecleucel

Country	Patent Number	Estimated Expiration
Spain	3039539
Japan	2024056708
Mexico	2016013239
Poland	3129470
Australia	2017203087
Costa Rica	20220507
South Korea	20240042250

Supplementary Protection Certificates for tisagenlecleucel

Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
C02649086/01	Switzerland		PRODUCT NAME: TISAGENLECLEUCEL; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 66778 18.10.2018
2019007	Norway		PRODUCT NAME: TISAGENLEKLEUCEL; REG. NO/DATE: 20180827
355 4-2019	Slovakia		PRODUCT NAME: TISAGENLEKLEUCEL; REGISTRATION NO/DATE: EU/1/18/1297 20180827
PA2019502,C2649086	Lithuania		PRODUCT NAME: TISAGENLEKLEUCELAS; REGISTRATION NO/DATE: EU/1/18/1297 20180823
SPC/GB19/011	United Kingdom		PRODUCT NAME: TISAGENLECLEUCEL; REGISTERED: UK EU/1/18/1297 (NI) 20180823; UK PLGB 00101/1101 20180823
LUC00104	Luxembourg		PRODUCT NAME: KYMRIAH - TISAGENLECLEUCEL; AUTHORISATION NUMBER AND DATE: EU/1/18/1297 20180827
CR 2019 00006	Denmark		PRODUCT NAME: TISAGENLECLEUCEL; REG. NO/DATE: EU/1/18/1297 20180827

Market Dynamics and Financial Trajectory for the Biologic Drug: Tisagenlecleucel

Last updated: July 27, 2025

Introduction

Tisagenlecleucel (marketed as Kymriah) stands as a pioneering CAR-T cell therapy developed by Novartis, revolutionizing the treatment landscape for certain hematologic malignancies. Approved initially by the U.S. Food and Drug Administration (FDA) in 2017 for pediatric acute lymphoblastic leukemia (ALL) and later for adult relapsed/refractory large B-cell lymphoma, tisagenlecleucel embodies the paradigm shift toward personalized immuno-oncology. Its market trajectory is shaped by an intricate interplay of scientific innovation, regulatory pathways, competitive dynamics, and evolving reimbursement landscapes.

Market Introduction and Initial Adoption

Tisagenlecleucel's emergence as the first CAR-T therapy to secure FDA approval fundamentally altered therapeutic approaches for pediatric ALL and adult lymphomas. Its initial FDA approval in 2017 was based on promising pivotal trials demonstrating durable remissions. The innovative nature of tisagenlecleucel, along with its Breakthrough Therapy designation, accelerated its market entry. Early adoption was facilitated by high unmet clinical needs, especially in relapsed/refractory populations with limited options.

However, initial adoption faced hurdles: manufacturing complexities, high costs (approximately $475,000 per treatment course in the US initially), and logistical challenges related to patient-specific cell manufacturing. These factors contributed to a cautious uptake during the first years but gradually improved as infrastructure and experience matured.

Market Expansion and Clinical Indications

Subsequent clinical data and regulatory extensions expanded tisagenlecleucel’s indications, including follicular lymphoma and in certain European markets. This diversification fostered broader accessibility, although the primary revenue driver remains pediatric ALL and adult DLBCL.

Additionally, ongoing trials aim to expand its use in multiple myeloma, multiple solid tumors, and other hematologic malignancies. While these developments promise future growth, many are still in investigational stages, making near-term financial impact limited.

Competitive Landscape and Market Dynamics

The CAR-T space has become increasingly crowded, with competitors such as Gilead's Yescarta (axicabtagene ciloleucel) and Breyanzi (lisocabtagene maraleucel), and more pipeline products from Janssen, BMS, Celgene, and innovative biotech companies. The competition exerts downward pressure on prices and necessitates differentiation strategies, including safety profiles, manufacturing efficiencies, and broader indications.

Regulatory agencies are also emphasizing manufacturing quality and safety standards, influencing operational costs and deployment. Additionally, the shift toward allogeneic ("off-the-shelf") CAR-T products presents potential disruption, potentially reducing manufacturing costs and improving access, though tisagenlecleucel remains autologous.

Pricing, Reimbursement, and Market Access

Tisagenlecleucel’s high initial price point reflected the costs of personalized manufacturing, with subsequent pricing negotiations influencing its market penetration. Payers have increasingly scrutinized the cost-effectiveness of CAR-T therapies, emphasizing durable responses and quality-adjusted life years (QALYs).

Value-based pricing agreements, including outcomes-based reimbursement models, are emerging to mitigate financial risk for payers and providers. In markets with robust reimbursement frameworks—such as the US Medicaid and private insurers—access has expanded, although cost remains a barrier in some healthcare systems.

Manufacturing and Supply Chain Evolution

Manufacturing capacity and logistics are critical to meeting demand and optimizing revenue. Novartis has invested heavily in improving manufacturing processes, including automation and centralized production facilities, reducing time-to-treatment and costs. These advances contribute to more predictable supply and better scalability, which are essential for market growth.

However, manufacturing complexities continue to restrain rapid expansion, especially for countries with limited infrastructure. Supply chain disruptions, exemplified during the COVID-19 pandemic, underscored vulnerabilities requiring strategic mitigations.

Financial Trajectory and Revenue Projections

Tisagenlecleucel’s initial revenues were modest, reflecting conservative uptake and reimbursement hurdles. However, as manufacturing efficiency improves and indications expand, revenue growth is expected to accelerate. Fitch Ratings estimated that the global CAR-T market could reach $15 billion by 2027, with tisagenlecleucel capturing a significant share, especially in pediatric ALL and diffuse large B-cell lymphoma (DLBCL) segments.

In 2021, Novartis reported approximately $650 million in CAR-T sales, a figure projected to grow at a compound annual growth rate (CAGR) exceeding 30% over the next five years. The growth is predicated on uptake in eligible patient populations, expanding indications, and technological advancements reducing costs.

Regulatory and Competitive Challenges

Regulatory agencies are increasingly demanding real-world evidence to support continued commercialization and reimbursement. Any safety concerns or manufacturing issues could impede growth.

Competition from emerging allogeneic CAR-T therapies could threaten tisagenlecleucel’s market share, especially if these off-the-shelf products demonstrate comparable efficacy with lower costs and simplified logistics.

Furthermore, the patent landscape and potential biosimilar entrants pose long-term risks, necessitating continual innovation and pipeline development.

Future Outlook and Strategic Considerations

Looking ahead, the commercialization trajectory of tisagenlecleucel hinges on several factors:

• Successful expansion into new indications.
• Technological innovations that lower production costs.
• International market penetration, particularly in emerging economies.
• Evolving reimbursement policies favoring value-based care.
• Competition from next-generation CAR-T cell therapies and allogeneic products.

Novartis’s strategic focus includes streamlining manufacturing, enhancing safety profiles, and conducting pivotal trials to broaden its therapeutic scope. The integration of digital health tools and biomarker-driven patient selection may further optimize outcomes and market penetration.

Key Market Trends Impacting Tisagenlecleucel’s Financial Pathway

• Personalized medicine innovation: CAR-T therapies embody precision oncology, demanding tailored manufacturing but offering high margins.
• Cost containment pressures: Payers' increasing emphasis on cost-effectiveness will influence reimbursement strategies and access.
• Technological advancements: Automated manufacturing and allogeneic approaches could eventually disrupt current autologous models.
• Regulatory evolution: Approval processes and post-market surveillance shape commercialization timelines and product liability considerations.
• Strategic partnerships: Collaborations to develop next-generation CARs or expand manufacturing capacity are pivotal.

Conclusion

Tisagenlecleucel remains a landmark biologic, with its market trajectory poised for substantial growth driven by expanding indications, improved manufacturing, and rising global adoption. Nonetheless, competitive pressures, pricing adjustments, and evolving regulatory and reimbursement environments introduce uncertainty. Strategic innovation and operational excellence will be critical for sustaining its financial success over the coming decades.

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Key Takeaways

• Tisagenlecleucel's innovative approach catalyzed a new therapeutic class, with significant market opportunities in hematologic malignancies.
• Growth is driven by expanded indications, manufacturing improvements, and international market penetration, but faces competitive and economic challenges.
• Price and reimbursement strategies increasingly rely on outcomes-based models to sustain profitability and market access.
• Advancements in allogeneic CAR-T therapies and digital health tools are potential disruptors.
• Strategic investments in pipeline development and manufacturing scalability are vital for long-term financial performance.

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FAQs

1. What are the primary drivers of tisagenlecleucel’s revenue growth?
The main drivers include expansion into new indications, increased manufacturing capacity, broader geographic access, and improved reimbursement strategies.

2. How does tisagenlecleucel compare to other CAR-T therapies in terms of market share?
Tisagenlecleucel maintains a strong position, especially in pediatric ALL, but faces competition from Yescarta and newer allogeneic products, which could alter market share dynamics.

3. What are the main challenges impacting tisagenlecleucel’s financial trajectory?
Manufacturing complexities, high costs, reimbursement hurdles, and emerging competition from off-the-shelf CAR-T therapies pose significant challenges.

4. How might technological advances influence the future of tisagenlecleucel?
Innovations like automated manufacturing and allogeneic cell sources could reduce costs, improve supply chain efficiency, and expand access.

5. What strategic actions should Novartis consider to optimize tisagenlecleucel’s market performance?
Focusing on pipeline expansion, strengthening manufacturing infrastructure, adopting outcome-based reimbursement models, and exploring new indications will enhance long-term revenue prospects.

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References

[1] FDA. FDA Approves Kymriah for relapsed or refractory follicular lymphoma. 2022.
[2] Novartis. Annual Report 2022.
[3] Fitch Ratings. Global CAR-T Cell Therapy Market Outlook. 2023.
[4] MarketWatch. CAR-T Cell Therapy Market Forecast. 2023.