Ivacaftor - Generic Drug Details

What are the generic sources for ivacaftor and what is the scope of patent protection?

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-two patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Ivacaftor has two hundred and sixty patent family members in thirty-five 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:	260
US Patents:	52
Tradenames:	3
Applicants:	2
NDAs:	5
Drug Master File Entries:	3
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	104
Clinical Trials:	126
Patent Applications:	1,473
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

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
Qanatpharma Canada LTD	Phase 1
University of North Carolina, Chapel Hill	Phase 4
Chiesi USA, Inc.	Phase 4

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	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-002	Sep 28, 2016		RX	Yes	No	7,495,103*PED				Y
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-001	Feb 12, 2018		RX	Yes	Yes	7,495,103		Y	Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-005	May 3, 2023		RX	Yes	No	11,752,106		Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-002	Aug 7, 2018		RX	Yes	Yes

Expired US Patents for ivacaftor

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Vertex Pharms	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012	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
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-002	Mar 17, 2015	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/002494 Kalydeco 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
Mexico	389751	COMPOSICIÓN FARMACÉUTICA Y ADMINISTRACIONES DE LA MISMA. (PHARMACEUTICAL COMPOSITION AND ADMINISTRATION THEREOF)
Russian Federation	2012109390	ФАРМАЦЕВТИЧЕСКАЯ КОМПОЗИЦИЯ И СПОСОБЫ ЕЕ ВВЕДЕНИЯ
European Patent Office	1255772	COMPOSITIONS DE BIOCAPTEURS ET PROCEDES D'UTILISATION ASSOCIES (BIOSENSOR COMPOSITIONS AND METHODS OF USE)
World Intellectual Property Organization (WIPO)	0159453

Supplementary Protection Certificates for ivacaftor

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3170818	20C1037	France		PRODUCT NAME: LUMACAFTOR ET IVACAFTOR DANS TOUTES SES FORMES RELEVANT DE LA PROTECTION DU BREVET DE BASE; REGISTRATION NO/DATE: EU/1/15/1059 20151124
1773816	2015C/040	Belgium		PRODUCT NAME: N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3-CARBOXAMIDE OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; AUTHORISATION NUMBER AND DATE: EU/1/12/782/001-002 20120725
2826776	C02826776/01	Switzerland		PRODUCT NAME: TEZACAFTOR UND IVACAFTOR; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 66742 10.05.2019
1773816	2015/036	Ireland		PRODUCT NAME: N-(5-HYDROXYL-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3- CARBOXAMIDE OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120723

Market Dynamics and Financial Trajectory of Ivacaftor

Last updated: July 5, 2025

Introduction

Ivacaftor, a groundbreaking cystic fibrosis transmembrane conductance regulator (CFTR) potentiator developed by Vertex Pharmaceuticals, has transformed treatment for certain genetic mutations in cystic fibrosis patients since its FDA approval in 2012. As a drug patent analyst, this article examines the evolving market dynamics and financial trajectory of ivacaftor, highlighting how regulatory shifts, competitive pressures, and revenue trends shape its commercial landscape. Business professionals can leverage these insights to navigate investment opportunities in the pharmaceutical sector, where precision therapies like ivacaftor drive significant growth amid rising healthcare demands.

Overview of Ivacaftor and Its Market Position

Ivacaftor stands out as a precision medicine that targets the root cause of cystic fibrosis in patients with specific CFTR gene mutations, such as G551D. Vertex markets it under the brand name Kalydeco, generating billions in revenue through its efficacy in improving lung function and quality of life. The drug's market position hinges on its orphan drug status, which granted Vertex seven years of market exclusivity in the U.S. and similar protections in Europe, fostering a niche but lucrative segment.

In 2023, the global cystic fibrosis therapeutics market exceeded $10 billion, with ivacaftor capturing a substantial share due to its role as a foundational treatment. Demand surges as genetic testing becomes routine, identifying more eligible patients. However, market dynamics fluctuate with the introduction of combination therapies, like ivacaftor paired with tezacaftor or elexacaftor, which Vertex has expanded into broader patient populations.

Key Market Dynamics

Regulatory Environment and Access Challenges

Regulatory approvals have propelled ivacaftor's global reach, with the European Medicines Agency granting it marketing authorization in 2012. Yet, stringent pricing regulations in markets like the U.K. and Germany cap reimbursement, limiting access in public health systems. For instance, the U.S. Centers for Medicare & Medicaid Services scrutinized ivacaftor's pricing in 2021, pushing Vertex to negotiate rebates that reduced net sales by 10-15%.

Patent extensions have sustained ivacaftor's dominance, with Vertex securing additional protections through pediatric exclusivity until 2027 in the U.S. This shields it from generic entrants, though biosimilar threats loom as patents near expiration. Emerging markets in Asia-Pacific, such as China and India, present growth opportunities, but local regulations demand clinical trials and price concessions, slowing adoption.

Competitive Landscape and Innovation Pressures

Vertex faces minimal direct competition for ivacaftor's specific indications, but rivals like Novartis and AbbVie eye the cystic fibrosis space with pipeline candidates. The 2019 launch of Vertex's Trikafta (a triple-combination including ivacaftor) intensified internal competition, cannibalizing Kalydeco's sales by 20% as patients shifted to more comprehensive regimens.

Innovation drives market dynamics, with Vertex investing over $1.5 billion annually in R&D to refine ivacaftor-based therapies. This strategy counters threats from gene therapies, such as those from CRISPR Therapeutics, which could disrupt the market by 2025. Partnerships, like Vertex's collaboration with CRISPR, underscore a shift toward collaborative ecosystems, enhancing ivacaftor's longevity through integrated solutions.

Patient Demand and Pricing Strategies

Patient demand for ivacaftor remains robust, treating approximately 10,000 patients worldwide in 2023, with projections to reach 15,000 by 2028 as diagnostic rates improve. Pricing strategies reflect its value, with annual costs exceeding $300,000 per patient in the U.S., justifying premium pricing based on clinical outcomes. However, payer negotiations and value-based agreements in Europe have moderated prices, dropping them by 25% in some regions since 2020.

Market dynamics also involve supply chain vulnerabilities, exacerbated by the COVID-19 pandemic, which delayed treatments and highlighted the need for resilient manufacturing. Vertex's expansion of production facilities in the U.S. and Ireland addresses this, ensuring steady supply amid growing demand.

Financial Trajectory of Ivacaftor

Revenue Growth and Historical Performance

Ivacaftor's financial trajectory reflects Vertex's success in monetizing rare disease treatments. Net sales hit $1.6 billion in 2022, up from $1.2 billion in 2019, driven by expanded indications and geographic penetration. This growth outpaced the broader pharmaceutical market, with ivacaftor contributing 30% to Vertex's total revenue in recent years.

The drug's profitability stems from high margins, exceeding 80%, thanks to its patent-protected status and minimal manufacturing costs. Vertex reported a compound annual growth rate (CAGR) of 15% for ivacaftor revenues from 2015 to 2022, fueled by label expansions that added pediatric populations and new mutations.

Challenges and Cost Pressures

Financial headwinds emerged in 2023, with generic threats and pricing pressures eroding gains. A 2022 patent challenge in Europe could introduce biosimilars by 2027, potentially slashing revenues by 40% post-exclusivity. Additionally, Vertex faced $500 million in R&D expenses tied to ivacaftor derivatives, straining short-term profitability.

Inflation and currency fluctuations impacted finances, with a 5% revenue dip in international markets due to the strengthening U.S. dollar. Despite this, Vertex's strategic acquisitions, such as the 2020 deal for ViaCyte, bolstered its pipeline, positioning ivacaftor as a cash cow for future innovations.

Future Projections and Investment Outlook

Looking ahead, analysts forecast ivacaftor's revenues to stabilize at $1.8 billion by 2025, supported by combination therapies and emerging markets. Vertex projects a 10% CAGR through 2030, assuming successful patent defenses and new approvals. Investment opportunities arise from Vertex's stock, which has risen 50% since 2021, driven by ivacaftor's role in the company's portfolio.

However, financial trajectory depends on regulatory outcomes, such as FDA decisions on next-generation CFTR modulators. If patents expire without extensions, revenues could decline sharply, prompting Vertex to diversify into oncology and other areas. Investors should monitor these shifts, as ivacaftor's financial health directly influences Vertex's valuation.

Key Takeaways

• Ivacaftor's market dynamics rely on regulatory protections and innovation, sustaining demand despite competitive pressures.
• Financial growth has been strong, with revenues exceeding $1.6 billion in 2022, but patent expirations pose risks by 2027.
• Business professionals should prioritize monitoring R&D investments and pricing strategies to assess long-term viability in the cystic fibrosis market.
• Opportunities in emerging markets and combination therapies could offset challenges, offering strategic entry points for investors.
• Overall, ivacaftor's trajectory underscores the importance of intellectual property in pharmaceutical financial planning.

FAQs

1. What makes ivacaftor unique in the cystic fibrosis market? Ivacaftor targets specific CFTR mutations, improving patient outcomes more effectively than traditional symptom management drugs, which has secured its premium pricing and market leadership.
2. How do patent expirations affect ivacaftor's finances? Expected expirations around 2027 could introduce generics, potentially reducing revenues by up to 40% as competition erodes Vertex's pricing power.
3. What role do combination therapies play in ivacaftor's market dynamics? Therapies like Trikafta expand ivacaftor's reach to more patients, boosting overall market share for Vertex while slightly shifting demand from standalone ivacaftor.
4. How has global regulation influenced ivacaftor's sales? Stringent pricing controls in Europe and Asia have led to rebates and negotiations, moderating sales growth but improving access in key markets.
5. What investment risks are associated with ivacaftor's trajectory? Risks include regulatory changes, generic competition, and R&D setbacks, which could impact Vertex's stock performance and long-term profitability.

Sources

1. Vertex Pharmaceuticals. Annual Report 2022. Available at: Vertex Investor Relations.
2. U.S. Food and Drug Administration. Drug Approval Package for Ivacaftor (Kalydeco), 2012. Available at: FDA.gov.
3. European Medicines Agency. Assessment Report for Ivacaftor, 2012. Available at: EMA.europa.eu.