Ivacaftor - Generic Drug Details

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

Ivacaftor is the generic ingredient in three branded drugs marketed by Vertex Pharms Inc and Vertex Pharms, and is included in five NDAs. There are fifty-four patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Ivacaftor has two hundred and sixty-two patent family members in thirty-six countries.

There are three drug master file entries for ivacaftor. One supplier is listed for this compound. There are two tentative approvals for this compound.

Summary for ivacaftor

International Patents:	262
US Patents:	54
Tradenames:	3
Applicants:	2
NDAs:	5
Drug Master File Entries:	3
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	104
Clinical Trials:	128
Drug Prices:	Drug price trends for ivacaftor
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ivacaftor
What excipients (inactive ingredients) are in ivacaftor?	ivacaftor excipients list
DailyMed Link:	ivacaftor at DailyMed

See drug prices for ivacaftor

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for ivacaftor

Constraining patent/regulatory exclusivity:

8,883,206*PED

Dosage:

GRANULE;ORAL

Constraining patent/regulatory exclusivity:

10,646,481*PED

Dosage:

TABLET;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 ivacaftor

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

Sponsor	Phase
Vertex Pharmaceuticals Incorporated	PHASE3
University of Kansas Medical Center	PHASE2
Qanatpharma Canada LTD	Phase 1

See all ivacaftor clinical trials

Generic filers with tentative approvals for IVACAFTOR

Applicant	Application No.	Strength	Dosage Form
		150MG	TABLET; ORAL
		75MG	GRANULE;ORAL
		50MG	GRANULE;ORAL

The 'tentative' approval signifies that the product meets all FDA standards for marketing, and, but for the patents / regulatory protections, it would approved.

Pharmacology for ivacaftor

Drug Class	Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
Mechanism of Action	Chloride Channel Activation Potentiators Cytochrome P450 2C9 Inhibitors Cytochrome P450 3A Inhibitors P-Glycoprotein Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for ivacaftor

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

Paragraph IV (Patent) Challenges for IVACAFTOR

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
KALYDECO	Oral Granules	ivacaftor	25 mg, 50 mg and 75 mg	207925	1	2022-04-13
KALYDECO	Tablets	ivacaftor	150 mg	203188	1	2020-06-10

US Patents and Regulatory Information for 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	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-002	Jun 21, 2019		RX	Yes	No	8,629,162
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	10,646,481*PED				Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-002	Mar 17, 2015		RX	Yes	Yes	11,147,770*PED				Y

Expired US Patents for ivacaftor

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-001	Mar 17, 2015	8,629,162
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-005	May 3, 2023	8,629,162
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-004	May 3, 2023	8,629,162

EU/EMA Drug Approvals for ivacaftor

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Vertex Pharmaceuticals (Ireland) Limited	Kalydeco	ivacaftor	EMEA/H/C/002494Kalydeco tablets are indicated:As monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).In a combination regimen with tezacaftor/ivacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T.In a combination regimen with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1).Kalydeco granules are indicated for the treatment of infants aged at least 4 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).	Authorised	no	no	no	2012-07-23

International Patents for ivacaftor

Country	Patent Number	Title	Estimated Expiration
World Intellectual Property Organization (WIPO)	2007079139
Canada	2810655
European Patent Office	3705477	MODULATEURS DE TRANSPORTEURS DE CASSETTE À LIAISON ATP (MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS)

Supplementary Protection Certificates for 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
3170818	C03170818/01	Switzerland		PRODUCT NAME: IVACAFTOR + LUMACAFTOR; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 65981 02.09.2016
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

Market Dynamics and Financial Trajectory for the Pharmaceutical Drug: Ivacaftor

Last updated: July 27, 2025

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Introduction

Ivacaftor, marketed as Kalydeco, represents a pivotal advancement in targeted therapy for cystic fibrosis (CF), a genetic disorder affecting approximately 70,000 individuals worldwide. Since its approval by the U.S. Food and Drug Administration (FDA) in 2012, ivacaftor has significantly influenced the pharmaceutical landscape, exemplifying personalized medicine's potential. This analysis explores the evolving market dynamics, financial trajectory, competitive landscape, and strategic factors shaping ivacaftor’s future.

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Market Landscape and Growth Drivers

Prevalence and Patient Demographics

Cystic fibrosis predominantly affects populations of European descent, with an estimated global prevalence of 1 in 2,500 to 3,500 live births. The advent of genotype-specific therapies like ivacaftor has shifted the treatment paradigm, making precision medicine central to CF management. As of 2022, over 20,000 patients globally have been treated with ivacaftor, reflecting its substantial adoption within its approved indications.

Genotype-Driven Therapeutic Expansion

Ivacaftor targets specific mutations—most notably G551D and other gating mutations—constituting approximately 4-8% of CF cases globally. The initial focus on G551D mutation was augmented by subsequent approvals covering additional mutations such as S549N, G1349D, and others, broadening the treatable patient population. This mutation-specific approach aligns with trends in personalized medicine, boosting market penetration.

Regulatory and Labeling Developments

Since its initial approval, the FDA and European Medicines Agency (EMA) have extended ivacaftor’s labeling to include pediatric populations and additional mutations. Notably, in 2017, the approval of a fixed-dose combination with tezacaftor expanded indications for patients with F508del mutation, accounting for an estimated 50% of CF cases, further enlarging the eligible population.

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Market Dynamics: Competitive and Regulatory Factors

Market Penetration and Pricing Strategies

Ivacaftor commands premium pricing, reflective of its innovative mechanism and clinical benefits. As of recent reports, the drug’s annual cost exceeds $300,000 per patient in the U.S., placing it among the most expensive medications. Despite high costs, strong efficacy and improved quality of life outcomes foster payer willingness to reimburse, especially given the life-threatening nature of CF.

Emergence of Combination Therapies

The market has evolved with the development of next-generation correctors and potentiators. The introduction of lumacaftor/ivacaftor (Orkambi), tezacaftor/ivacaftor (Symdeko), and notably the triple combination elexacaftor/tezacaftor/ivacaftor (Trikafta) revolutionized treatment, demonstrating superior efficacy and expanding the treatable population to include F508del homozygous patients—who comprise approximately 90% of CF patients globally. Ivacaftor remains a core component within these regimens, underpinning its ongoing relevance.

Competitive Landscape and Market Share

While ivacaftor pioneered targeted CF therapy, the market increasingly favors combination regimens with broader genotype applicability. Nonetheless, ivacaftor retains a critical role, either as monotherapy for gating mutations or as part of fixed-dose combinations. Competition from emerging therapies, including gene editing and mRNA-based approaches, could reshape the landscape over the next decade.

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Financial Trajectory and Business Performance

Revenue Growth Trajectory

Gilead Sciences, the manufacturer of ivacaftor, reported steady sales growth driven by increased patient access and expanded indications. For instance, 2020’s global sales exceeded $1.5 billion, with subsequent years reflecting continued expansion. Market expansion into pediatric cohorts and countries with emerging markets further bolstered revenue.

Impact of Pricing and Market Access

Pricing strategies significantly influence revenue. High costs are offset by payer acceptance driven by clinical benefits and the high burden of disease. Strategic negotiations and patient assistance programs mitigate affordability challenges, facilitating sustained market penetration.

Research and Development Investment

Investments into next-generation CF therapies impact the financial trajectory. Gilead has committed substantial resources to enhance existing formulations and develop novel agents, which could eventually cannibalize ivacaftor sales but also solidify market leadership through sustained innovation.

Patent Life and Lifecycle Management

Patent protection, initially granted in 2012, has been extended through formulation and combination patent filings, granting market exclusivity into the late 2020s. The patent landscape will influence future generic entry, though robust patent defenses and orphan drug incentives cushion revenue streams.

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Regulatory and Market Outlook

Future Market Expansion

The global CF drug market is projected to grow at a compound annual growth rate (CAGR) of approximately 10-14% over the next five years, buoyed by expanded indications, increased diagnosis rates, and improved healthcare infrastructure. The emergence of early intervention strategies and new combination therapies promises further growth.

Pipeline and Innovation

Ongoing clinical development aims to address non-responders and optimize dosing strategies. Gene therapy candidates and mRNA platforms represent promising areas, with some early-stage agents potentially disrupting the current paradigm.

Pricing and Access Pressures

Healthcare systems increasingly scrutinize high-cost therapies, with payers demanding evidence of cost-effectiveness. Regulatory agencies and insurers are likely to exert pressure on pricing models, influencing future revenue potential.

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Strategic Recommendations for Stakeholders

• Pharmaceutical Companies: Invest in combination therapies and personalized medicine approaches to extend market share and address unmet needs.
• Investors: Monitor patent expiry timelines and emerging pipeline candidates that could impact ivacaftor’s position.
• Healthcare Providers: Emphasize early diagnosis and mutation-specific treatment plans to optimize patient outcomes.
• Policy Makers: Balance innovation incentives with affordability to ensure broad access to life-changing therapies.

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Key Takeaways

• Market Expansion: Ivacaftor benefits from a unique position as a mutation-specific therapy with growing global adoption, especially as its indications widen.
• Revenue Drivers: High pricing aligned with demonstrated clinical benefit sustains strong revenue streams, but increasing competition from combination regimens poses challenges.
• Pipeline and Innovation: Future growth hinges on pipeline advancements, including next-generation modulators and gene therapies, which could redefine treatment paradigms.
• Regulatory Landscape: Continued regulatory support and patent protections are vital for revenue stability, while pricing pressures could impact profitability.
• Strategic Positioning: The evolution toward broader genotype coverage emphasizes the importance of innovation and strategic lifecycle management.

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FAQs

1. What is the primary mechanism of action of ivacaftor?
   Ivacaftor is a potentiator that enhances the opening probability of the CFTR chloride channel, improving chloride transport in specific gating mutations like G551D.

2. How has ivacaftor impacted the treatment landscape for cystic fibrosis?
   It pioneered targeted, mutation-specific therapy, significantly improving lung function and quality of life for eligible patients, and catalyzed the development of combination therapies.

3. What factors influence ivacaftor’s pricing strategy?
   The high cost reflects its innovative nature, clinical efficacy, and small patient population, with payer reimbursement driven by demonstrable health benefits.

4. What is the projected future for ivacaftor within the CF treatment ecosystem?
   While its standalone use may decline with broader combination therapies, ivacaftor remains integral within these regimens and may serve as a foundation for further innovation.

5. Are there emerging therapies that could compete with ivacaftor?
   Yes, gene editing, mRNA therapies, and next-generation CFTR modulators are in development, potentially offering more comprehensive or curative options in the future.

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References

[1] Cystic Fibrosis Foundation. "Cystic Fibrosis Prevalence Data." 2021.
[2] Gilead Sciences. "Kalydeco (Ivacaftor) Prescribing Information." 2022.
[3] ReportLinker Research. "Global Cystic Fibrosis Market Outlook," 2022.
[4] FDA. "Ivacaftor (Kalydeco) Approval Letter," 2012.
[5] EvaluatePharma. "Pharmaceutical Market Outlook," 2022.