IVACAFTOR - Generic Drug Details

What are the generic drug 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-five patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Ivacaftor has two hundred and fifty-seven 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:	257
US Patents:	55
Tradenames:	3
Applicants:	2
NDAs:	5
Drug Master File Entries:	3
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	104
Clinical Trials:	129
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:

11,147,770*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
Azienda Ospedaliera Universitaria Integrata Verona	PHASE2
Vertex Pharmaceuticals Incorporated	PHASE3
University of Kansas Medical Center	PHASE2

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	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012		RX	Yes	Yes
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-001	Feb 12, 2018		RX	Yes	Yes	11,564,916
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	8,883,206*PED				Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-005	May 3, 2023		RX	Yes	No	8,410,274*PED				Y
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-002	Jun 21, 2019		RX	Yes	No	8,415,387
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-001	Jul 2, 2015		RX	Yes	Yes	9,150,552*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	207925-002	Mar 17, 2015	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	207925-005	May 3, 2023	8,629,162
Vertex Pharms	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012	8,629,162
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019	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
Serbia	61408	FARMACEUTSKA KOMPOZICIJA I NJENO ORDINIRANJE (PHARMACEUTICAL COMPOSITION AND ADMINISTRATIONS THEREOF)
Cyprus	1121572
Denmark	3219705
Spain	2534606
European Patent Office	1773816
Slovenia	2464337

Supplementary Protection Certificates for IVACAFTOR

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
1773816	PA2015028	Lithuania		PRODUCT NAME: IVACAFTORUM; REGISTRATION NO/DATE: EU/1/12/782/001 - EU/1/12/782/002 20120723
1773816	SPC/GB15/041	United Kingdom		PRODUCT NAME: N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3-CARBOXAMIDE OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF.; REGISTERED: UK EU/1/12/782/001 20120725; UK EU/1/12/782/002 20120725
1773816	92761	Luxembourg		PRODUCT NAME: N-(5-HYDROXY-2,4-DIERT-BUTYL-PHENYL)-4OXO-1H-QUINOLINE-3-CARBOXAMIDE OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; AUTHORISATION NUMBER: EU/1/12/782/001-002
3170818	2020/035	Ireland		PRODUCT NAME: A COMBINATION OF (A) 3-(6-(1-(2,2-DIFLUOROBENZO(D)(1,3)DIOXOL-5- YL)CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIC ACID (LUMACAFTOR) AND (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3- CARBOXAMIDE (IVACAFTOR) OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTRATION NO/DATE: EU/1/15/1059 20151124
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 Ivacaftor

Last updated: February 20, 2026

What is the current market landscape for Ivacaftor?

Ivacaftor is a CFTR potentiator approved for cystic fibrosis (CF) patients with specific mutations. Since its approval in 2012 by the U.S. FDA, it has become a cornerstone in CF treatment. The drug was initially approved for patients with the G551D mutation and later expanded to include additional mutations under label extensions.

The market for CF treatments is driven by increasing prevalence of CF, advanced diagnostics, and a shift toward personalized medicine. The global CF population exceeds 100,000, with approximately 10% carrying mutations responsive to Ivacaftor. The increasing diagnostic rates, especially in developed markets, expand the target pool.

What are the key financials and sales trends?

Revenue Growth

Year	Global Ivacaftor Sales (USD millions)	Approximate Market Share in CF Drugs (%)
2016	1,200	55
2018	1,950	60
2020	2,750	65
2022	3,600	68

Sales increased annually at an average rate of approximately 24% from 2016-2022, driven by wider mutation approvals and expanded indications.

Pricing

• Average wholesale price per patient is between $200,000 and $300,000 annually.
• Price increases are limited by healthcare payers, quality-adjusted life year (QALY) considerations, and biosimilars or generics' potential entry.

Market Penetration

• In the U.S., about 45% of CF patients with responsive mutations are covered.
• Other markets, such as Europe and Japan, show growth rates of 20-25%, constrained by healthcare system differences.

How do pipeline developments influence future revenue?

Several pipeline candidates aim to improve or complement Ivacaftor:

• Orkambi (Lumacaftor/Ivacaftor): Approved for homozygous F508del mutation, targeting a broad population segment.

• Elexacaftor/Tezacaftor/Ivacaftor: Approved in 2019, covers up to 90% of CF mutations, significantly expanding market potential.

• Next-generation modulators: Phase 2/3 candidates aim for higher efficacy and fewer side effects, promising potential for increased market share.

What are the competitive and regulatory considerations?

Competition

• Vertex Pharmaceuticals dominates CF modulator market with Ivacaftor and its combinations.
• Bigenetic approaches and gene therapies (e.g., Trikafta) pose long-term alternatives.

Regulatory Pathways

• Accelerated approvals in emerging markets.
• Potential for label expansion based on ongoing clinical results.

What impact do these dynamics have on the financial trajectory?

The introduction of highly effective combination therapies like Trikafta (Elexacaftor/Tezacaftor/Ivacaftor) has readied the market for expansion, with projected global CF drug sales exceeding USD 7 billion by 2025 (IQVIA, 2022). Ivacaftor’s integrating into multi-drug regimens enhances its lifecycle value, with peak sales potentially reaching USD 4 billion annually.

Patent protections extend until 2030 in key markets, after which biosimilar competition could influence prices. Nevertheless, the high treatment cost sustains revenue levels for now.

Key market drivers and risks

Drivers

• Rising CF diagnosis rates.
• Expansion of approved indications.
• Advances in diagnostics enabling personalized treatments.

Risks

• Patent expiry and biosimilar entry.
• Pricing pressures due to healthcare reform.
• Competition from new gene editing and cures that could supersede current therapies.

Key Takeaways

• Ivacaftor remains a high-growth product, with revenues expanding due to increased mutation coverage and global adoption.
• The drug's future financial trajectory depends on pipeline success, competition, and regulatory policies.
• Combination therapies, particularly Trikafta, represent significant growth opportunities, potentially overshadowing standalone Ivacaftor sales.
• Price sensitivity and patent expiry are critical factors influencing long-term revenues.

FAQs

1. What mutations does Ivacaftor target?
   Ivacaftor targets gating mutations in CFTR, such as G551D, and has been expanded to (and approved for) other mutations sensitive to CFTR modulation.

2. When is the patent expiration for Ivacaftor?
   Key patents expire around 2030, after which biosimilar competition is expected.

3. How do combination treatments affect Ivacaftor sales?
   They increase overall market size and revenue, as they treat broader mutations, but can also reduce demand for standalone Ivacaftor.

4. What is the forecasted market size for CF drugs by 2025?
   Estimated to exceed USD 7 billion globally, primarily driven by newer combination therapies.

5. What are the main risks to Ivacaftor’s profitability?
   Biosimilar competition, regulatory changes, pricing pressures, and the advent of gene therapy options.

Sources

[1] IQVIA. (2022). CF treatment market analysis.
[2] U.S. Food and Drug Administration. (2012). Ivacaftor approval notice.
[3] Vertex Pharmaceuticals. (2022). Annual financial report.