Elexacaftor, ivacaftor, tezacaftor; ivacaftor - Generic Drug Details

What are the generic sources for elexacaftor, ivacaftor, tezacaftor; ivacaftor and what is the scope of freedom to operate?

Elexacaftor, ivacaftor, tezacaftor; ivacaftor is the generic ingredient in one branded drug marketed by Vertex Pharms Inc and is included in two NDAs. There are thirty-five patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Elexacaftor, ivacaftor, tezacaftor; ivacaftor has five hundred and twenty-three patent family members in forty-eight countries.

One supplier is listed for this compound.

Summary for elexacaftor, ivacaftor, tezacaftor; ivacaftor

International Patents:	523
US Patents:	35
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Clinical Trials:	23
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for elexacaftor, ivacaftor, tezacaftor; ivacaftor
DailyMed Link:	elexacaftor, ivacaftor, tezacaftor; ivacaftor at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for elexacaftor, ivacaftor, tezacaftor; ivacaftor

Dosage:

TABLET, TABLET;ORAL

Dosage:

GRANULES;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for elexacaftor, ivacaftor, tezacaftor; ivacaftor

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Vertex Pharmaceuticals Incorporated	PHASE3
University of Kansas Medical Center	PHASE2
University of North Carolina, Chapel Hill	Phase 4

See all elexacaftor, ivacaftor, tezacaftor; ivacaftor clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for elexacaftor, ivacaftor, tezacaftor; ivacaftor

R07AX	Other respiratory system products
R07A	OTHER RESPIRATORY SYSTEM PRODUCTS
R07	OTHER RESPIRATORY SYSTEM PRODUCTS
R	Respiratory system

US Patents and Regulatory Information for elexacaftor, ivacaftor, tezacaftor; ivacaftor

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	TABLET;ORAL	212273-002	Jun 8, 2021		RX	Yes	No
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	GRANULES;ORAL	217660-001	Apr 26, 2023		RX	Yes	No			Y	Y
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	TABLET;ORAL	212273-002	Jun 8, 2021		RX	Yes	No			Y
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	TABLET;ORAL	212273-002	Jun 8, 2021		RX	Yes	No			Y
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	GRANULES;ORAL	217660-001	Apr 26, 2023		RX	Yes	No

Expired US Patents for elexacaftor, ivacaftor, tezacaftor; ivacaftor

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	TABLET;ORAL	212273-002	Jun 8, 2021
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	GRANULES;ORAL	217660-001	Apr 26, 2023
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	GRANULES;ORAL	217660-002	Apr 26, 2023
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	TABLET;ORAL	212273-001	Oct 21, 2019

International Patents for elexacaftor, ivacaftor, tezacaftor; ivacaftor

Country	Patent Number	Title	Estimated Expiration
Russian Federation	2382779
Russian Federation	2009141996	МОДУЛЯТОРЫ ТРАНСПОРТЕРОВ АТФ-СВЯЗЫВАЮЩЕЙ КАССЕТЫ
China	110267948
European Patent Office	3219705
Australia	2013204751	Modulators of ATP-Binding Cassette Transporters

Supplementary Protection Certificates for elexacaftor, ivacaftor, tezacaftor; ivacaftor

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1773816	15C0045	France		PRODUCT NAME: IVACAFTOR, OU L'UN DE SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120725
2826776	2021C/517	Belgium		PRODUCT NAME: SYMKEVI - TEZACAFTOR/IVACAFTOR; EEN COMBINATIE VAN (A) (R)-1-(2,2-DIFLUOROBENZO(D)(1,3)DIOXOL-5-YL)-N-(1-(2,3-DIHYDROXYPROPYL)-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1H-INDOL-5-YL)CYCLOPROPANECARBOXAMIDE OF EEN VANUIT FARMACEUTISCH OOGPUNT GESCHIKT ZOUT DAARVAN EN (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3-CARBOXAMIDE OF EEN VANUIT FARMACEUTISCH OOGPUNT GESCHIKT ZOUT DAARVAN; AUTHORISATION NUMBER AND DATE: EU/1/18/1306 20181106
2826776	SPC/GB21/025	United Kingdom		PRODUCT NAME: A COMBINATION OF (A) TEZACAFTOR AND (B) IVACAFTOR; REGISTERED: UK EU/1/18/1306 (NI) 20181106; UK FURTHER MAS ON IPSUM 20181106
1773816	CA 2015 00038	Denmark		PRODUCT NAME: IVACAFTOR, N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLIN-3-CARBOXAMID ELLER ET FARMACEUTISK ACCEPTABLET SALT DERAF; REG. NO/DATE: EU/1/12/782/001-002 20120723
2826776	132021000000062	Italy		PRODUCT NAME: UNA COMBINAZIONE DI (A) TEZACAFTOR O UN SUO SALE FARMACEUTICAMENTE ACCETTABILE E (B) IVACAFTOR O UN SUO SALE FARMACEUTICAMENTE ACCETTABILE(SYMKEVI); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/18/1306, 20181106

Market Dynamics and Financial Trajectory for Elexacaftor, Ivacaftor, Tezacaftor: An In-Depth Analysis

Last updated: July 30, 2025

Introduction

The pharmaceutical landscape surrounding cystic fibrosis (CF) therapeutics has undergone transformative evolution over recent years, driven by a wave of targeted therapies. Among these, the combination regimens involving elexacaftor, ivacaftor, and tezacaftor have proven pivotal, reshaping treatment paradigms and influencing market dynamics extensively. This analysis explores the key factors shaping the market trajectory of these drugs, assessing their commercial potential, competitive positioning, regulatory influences, and future outlook.

Market Overview

CF is a hereditary, life-limiting genetic disorder affecting approximately 70,000 individuals worldwide, with a significant unmet medical need despite advancements in supportive care. The advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulators—specifically combinations like elexacaftor/tezacaftor/ivacaftor (ETI)—has marked a shift toward personalized medicine, targeting the disease’s molecular underpinnings.

Pfizer's Trikafta (elexacaftor/tezacaftor/ivacaftor) received regulatory approvals in 2019, primarily in North America. Subsequently, its approval expanded globally, fueling a substantial market uptick. The innovation lies in its capacity to treat a broader subset of CF patients—up to 90%—including those with minimal function mutations.

Key Market Drivers

1. Expanding Patient Population and Eligibility

The primary driver is the expanding eligible patient base. Previously limited to patients with specific mutations, the triple therapy's efficacy in a wider genetic cohort has significant commercial implications. The high prevalence of the F508del mutation, which affects over 70% of CF patients globally, has amplified market potential.

2. Clinical Efficacy and Patient Outcomes

Robust clinical data demonstrate significant improvements in lung function, quality of life, and reduction in pulmonary exacerbations. These promising outcomes foster higher prescription rates and bolster payer willingness to reimburse, underpinning revenue growth.

3. Regulatory Approvals and Reimbursement Policies

Accelerated approvals across various regions have catapulted sales. Notably, in 2020, the U.S. FDA granted accelerated approval for Trikafta, followed by EMA approval in Europe. Reimbursement policies favor high-cost but high-efficacy treatments, especially when supported by robust clinical data.

4. Strategic Partnerships and Market Penetration

Pharmaceutical collaborations and licensing agreements facilitate penetration into emerging markets. Pfizer's strategic expansion into markets like China and Latin America is instrumental, given the increasing CF diagnosis rates and improving healthcare infrastructure.

Market Challenges and Limitations

1. Price Points and Cost-Effectiveness Concerns

The high wholesale acquisition cost (~$311,000 annually in the U.S.) poses access challenges, especially in low- and middle-income countries. Payers' scrutiny over cost-effectiveness assessments impacts reimbursement policies, potentially constraining market growth.

2. Competitive Landscape

While ETI dominates current markets, competition from alternative therapies and next-generation CFTR modulators remains robust. Vertex Pharmaceuticals, a pioneer in CF treatment, continues to innovate, possibly introducing variants or gene therapies that could alter current dynamics.

3. Patent and Patent Expiry Risks

Patent protections secure exclusivity; however, the expiration or challenges to patents could invite generic or biosimilar entrants, impacting pricing and market share.

Financial Trajectory

1. Revenue Projections

Pfizer’s recent earnings reports highlight a rapidly scaling financial trajectory for Trikafta. For FY2022, Trikafta generated approximately $8 billion globally—up from about $5.9 billion in 2021 [1]. Analysts project a compounded annual growth rate (CAGR) of over 20% through 2027, driven by increased adoption and expanding indications.

2. Market Penetration and Growth Strategies

Pfizer's aggressive marketing, expanding geographic reach, and growing indications are fueling growth. Additionally, the continued pipeline development, including triple combination therapies tailored for more rare mutations, promises additional revenue streams.

3. Investment in R&D and Future Therapies

Investment in next-generation modulators and gene editing approaches signals the ongoing commitment to improving and expanding treatment options. These investments are poised to sustain long-term financial growth and market relevance.

4. Cost and Pricing Dynamics

While high prices contribute significantly to revenues, pressure to reduce costs and address reimbursement hurdles pose sustainability concerns. Pricing negotiations and value-based care models are expected to influence future revenue streams.

Regulatory and Policy Environment

Regulatory agencies increasingly emphasize accelerated pathways, real-world evidence, and cost-effectiveness. Reimbursement frameworks are shifting toward outcome-based agreements, affecting how pharmaceutical companies strategize market entry and expansion.

Competitive Landscape

1. Vertex Pharmaceuticals

As the market leader in CFTR modulators, Vertex's portfolio—including Trikafta, Symdeko, and earlier agents—dominate sales. Its ongoing pipeline aims to address rare mutations and reduce treatment costs. Strategic alliances and acquisitions bolster its market position.

2. Emerging Competitors

Several biotech firms are exploring alternative modalities—gene therapy, mRNA-based approaches, and alternative small molecules—to challenge current therapies. Though still in development, these innovations could disrupt current market shares if proven efficacious and economically viable.

Future Outlook

The market for elexacaftor, ivacaftor, and tezacaftor is poised for sustained growth over the next decade. Factors such as ongoing indications expansion, increasing diagnosis rates, and technological innovations will underpin this trajectory. The potential integration with gene therapy or curative approaches remains a long-term horizon, promising even more transformative shifts.

Conclusion

The fusion of cutting-edge biology, regulatory agility, and strategic commercialization has propelled elexacaftor/tezacaftor/ivacaftor into a dominant market position within CF therapeutics. While challenges related to pricing, competition, and patent protections persist, the overall financial outlook remains positive, driven by expanding patient access and robust clinical efficacy. Stakeholders must navigate regulatory landscapes and market access barriers creatively to sustain growth.

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Key Takeaways

• Market expansion is driven by broadening eligibility and clinical efficacy. The triple therapy’s ability to treat a higher percentage of CF patients extends market reach significantly.
• Revenue growth projected at a CAGR of over 20%, with current sales exceeding $8 billion globally. Expanding indications and geographic reach will sustain this momentum.
• Pricing and reimbursement dynamics remain critical, with high drug costs challenging affordability and access. Innovative payor strategies and value-based agreements are increasingly relevant.
• Intense competition from Vertex and emerging biotech firms warrants continuous innovation. Next-generation therapies and gene editing are potential disruptors.
• Regulatory environment favors accelerated approvals and real-world evidence growth, supporting market penetration. Strategic partnerships enhance market access in emerging regions.

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FAQs

1. How do elexacaftor, ivacaftor, and tezacaftor work together in CF treatment?
These agents function as CFTR modulators, enhancing the function of defective CFTR proteins. Ivacaftor increases channel opening, while tezacaftor and elexacaftor improve protein stability and trafficking, collectively restoring chloride transport in affected cells.

2. What is the global market outlook for these drugs?
The global market is expected to grow substantially, fueled by expanding indications, increased awareness, and geographic expansion, particularly in Europe, Asia-Pacific, and Latin America.

3. What are the main barriers to market growth for these drugs?
High treatment costs, restrictive reimbursement policies, patent expirations, and competition from emerging therapies are primary barriers.

4. Are there any upcoming therapies that could impact this market?
Yes, gene therapy and personalized curative approaches are in development, promising to potentially replace or complement current CFTR modulators.

5. How does reimbursement impact the financial success of these drugs?
Reimbursement policies directly influence patient access and drug sales. Outcome-based agreements and negotiations are crucial for maintaining profitability and market share.

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References

[1] Pfizer Inc. Financial Reports, 2022.
[2] U.S. Food and Drug Administration. Trikafta Approval Announcement, 2019.
[3] European Medicines Agency. EMA Approval for Trikafta, 2020.