KALYDECO Drug Patent Profile

When do Kalydeco patents expire, and what generic alternatives are available?

Kalydeco is a drug marketed by Vertex Pharms Inc and Vertex Pharms and is included in two NDAs. There are fourteen patents protecting this drug and two Paragraph IV challenges.

This drug has two hundred and fifty-seven patent family members in thirty-six countries.

The generic ingredient in KALYDECO is ivacaftor. There are three drug master file entries for this compound. One supplier is listed for this compound. Additional details are available on the ivacaftor profile page.

DrugPatentWatch^® Generic Entry Outlook for Kalydeco

Kalydeco was eligible for patent challenges on January 31, 2016.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be February 13, 2030. This may change due to patent challenges or generic licensing.

There have been five patent litigation cases involving the patents protecting this drug, indicating strong interest in generic launch. Recent data indicate that 63% of patent challenges are decided in favor of the generic patent challenger and that 54% of successful patent challengers promptly launch generic drugs.

There are two tentative approvals for the generic drug (ivacaftor), which indicates the potential for near-term generic launch.

Indicators of Generic Entry

Summary for KALYDECO

International Patents:	257
US Patents:	14
Applicants:	2
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	94
Clinical Trials:	26
Patent Applications:	1,513
Drug Prices:	Drug price information for KALYDECO
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for KALYDECO
What excipients (inactive ingredients) are in KALYDECO?	KALYDECO excipients list
DailyMed Link:	KALYDECO at DailyMed

See drug prices for KALYDECO

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

Constraining patent/regulatory exclusivity:

8,883,206*PED

NDA:

207925

Dosage:

GRANULE;ORAL

Constraining patent/regulatory exclusivity:

10,646,481*PED

NDA:

203188

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 KALYDECO

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

Sponsor	Phase
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2
University of Kansas Medical Center	Early Phase 1
University of North Carolina	Early Phase 1

See all KALYDECO clinical trials

Pharmacology for KALYDECO

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

Paragraph IV (Patent) Challenges for KALYDECO

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 KALYDECO

KALYDECO is protected by seventeen US patents and eleven FDA Regulatory Exclusivities.

This potential generic entry date is based on patent 10,646,481.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

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	KALYDECO	ivacaftor	GRANULE;ORAL	207925-001	Mar 17, 2015		RX	Yes	No	10,272,046*PED				Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-002	Mar 17, 2015		RX	Yes	Yes
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	8,629,162
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-005	May 3, 2023		RX	Yes	No	8,354,427*PED				Y
Vertex Pharms	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012		RX	Yes	Yes	10,646,481*PED				Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	9,670,163*PED				Y

Expired US Patents for KALYDECO

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-004	May 3, 2023	8,629,162
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-001	Mar 17, 2015	8,629,162
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019	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	207925-002	Mar 17, 2015	8,629,162
Vertex Pharms	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012	8,629,162

EU/EMA Drug Approvals for KALYDECO

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 KALYDECO

When does loss-of-exclusivity occur for KALYDECO?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Australia

Brazil

Canada

Chile

China

Croatia

Cyprus

Denmark

Eurasian Patent Organization

European Patent Office

Finland

Hong Kong

Hungary

Israel

Japan

Lithuania

Mexico

Montenegro

New Zealand

Norway

Poland

Portugal

Russian Federation

San Marino

Serbia

Singapore

Slovenia

South Africa

South Korea

Spain

Ukraine

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering KALYDECO around the world.

Country	Patent Number	Title	Estimated Expiration
Japan	5409010
European Patent Office	2502902	Modulateurs de transporteurs de cassette de liaison a l ́ATP (Modulators of ATP-binding cassette transporters)
European Patent Office	2502912	Modulateurs de transporteurs de cassette de liaison à l ́ATP (Modulators of ATP-binding cassette transporters)
Mexico	2014010253	COMPOSICION FARMACEUTICA Y ADMINISTRACIONES DE LA MISMA. (PHARMACEUTICAL COMPOSITION AND ADMINISTRATION THEREOF.)
Japan	2009522278
European Patent Office	3705477

Supplementary Protection Certificates for KALYDECO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
1773816	617	Finland
3170818	132020000000103	Italy		PRODUCT NAME: UNA COMBINAZIONE DI (A) LUMACAFTOR E (B) IVACAFTOR O UN SUO SALE FARMACEUTICAMENTE ACCETTABILE(ORKAMBI); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/15/1059, 20151124
3170818	2020C/005	Belgium		PRODUCT NAME: ORKAMBI (LUMACAFTOR + IVACAFTOR); AUTHORISATION NUMBER AND DATE: EU/1/15/1059 20151124
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	300748	Netherlands		PRODUCT NAME: IVACAFTOR, OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120725

Market Dynamics and Financial Trajectory of Kalydeco (Ivacaftor)

Last updated: December 30, 2025

Executive Summary

Kalydeco (ivacaftor), developed by Vertex Pharmaceuticals, is a groundbreaking cystic fibrosis (CF) therapy that first gained regulatory approval in 2012. Its innovative mechanism—targeting specific genetic mutations—positioned it uniquely in personalized medicine. The drug’s market dynamics are shaped by its targeted patient population, approval scope, competitive landscape, pricing strategies, and evolving regulatory environment. Financially, Kalydeco has demonstrated strong revenue growth, with significant contributions from expanding indications and global market penetration. This analysis explores key factors influencing its market and revenue trajectory, compares it with competitors, and assesses future growth prospects.

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1. Introduction to Kalydeco: Indication and Mechanism

What is Kalydeco?

• Active Ingredient: Ivacaftor
• Approval Date: 2012 (FDA), subsequent approvals internationally
• Primary Indication: Cystic fibrosis caused by specific G551D mutation and other gating mutations
• Mechanism of Action: Potentiates CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) protein function by increasing channel open probability

Market Relevance

Kalydeco exemplifies targeted therapy, tailored to genetic subtypes of CF, enabling more effective and personalized treatments.

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2. Market Dynamics

2.1. Patient Population and Market Size

• Global CF Population: Approximately 100,000 diagnosed patients worldwide
• US Population: ~30,000 CF patients, with ~4-5% harboring G551D and similar mutations amenable to Kalydeco (per FDA labels)
• Mutation-specific Market: Initially limited but expanding as approvals extend

2.2. Regulatory Milestones and Indications Expansion

Year	Regulatory Body	Indications Approved	Key Highlights
2012	FDA	G551D mutation	First targeted CF therapy
2014	EMA	Same as FDA	Broadened access
2018	FDA	Additional gating mutations	Expanded eligible mutations
2020+	Regulatory agencies	Multiple mutations and pediatric populations	Continued expansion, including in European and Asian markets

2.3. Pricing and Reimbursement Environment

• Pricing: List prices approximate $294,000 annually in the US (per 2022), reflecting high R&D costs and targeted efficacy
• Reimbursement: Insurance coverage increasingly negotiated through pharmacy benefit managers (PBMs), with regional variation
• Access Challenges: High drug cost influences market penetration rates

2.4. Competition and Market Share

Competitor	Drug Name	Indication Scope	Market Share (Estimated, 2023)
Vertex Pharmaceuticals	Kalydeco (Ivacaftor)	Gating mutations; expanded over time	~60%
Vertex Pharmaceuticals	Trikafta (Elexacaftor/Tezacaftor/Ivacaftor)	Broader CF population including F508del	~30%
Others	Orkambi, Symdeko	F508del mutation subgroup	Remaining market share

Note: Trikafta, approved in 2019, significantly impacted Kalydeco's market share via expanding indications.

2.5. Pricing Strategies and Market Penetration

• Tiered Pricing: Differentiated by region, based on economic status
• Patient Assistance Programs: To improve access and mitigate cost barriers
• Market Penetration: Accelerated through public health initiatives and expanded indications

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3. Financial Trajectory of Kalydeco

3.1. Revenue Growth and Trends

Year	Approximate Revenue (USD)	Change from Previous Year	Comments
2012	$145 million	—	Initial launch, limited patient pool
2014	$370 million	+155%	Broadened indications, increased adoption
2016	$593 million	+60%	Expansion into additional jurisdictions
2018	$927 million	+56%	Introduction of Trikafta influenced sales trends
2020	$1.2 billion	+29%	As indications further expanded
2022	~$1.5 billion	+25%	Peak sales period; growth rate moderates

Source: Vertex’s Annual Reports [1], [2], [3]

3.2. Cost and Investment Analysis

• R&D Expenses: Significant investment into mutation-specific research
• Manufacturing Cost: High, attributable to complex bioprocessing
• Regulatory Cost: Substantial, given the genetic testing components of indications
• Profit Margin: Estimated gross margin around 85%; net margins affected by R&D and commercialization expenses

3.3. Profitability and Future Revenue Drivers

• Key Drivers:
  • Expansion into pediatric and broader mutation populations
  • Increasing global approvals
  • Entry into emerging markets (Asia, Latin America)
• Potential Risks:
  • Regulatory delays or rejections
  • Market saturation
  • Competition from new gene-editing therapies

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4. Market Drivers and Challenges

4.1. Growing Personalized Medicine Paradigm

• Increasing genetic testing adoption feeds demand
• Expanding indications align with FDA's trend towards broad subset approvals

4.2. Regulatory and Reimbursement Landscape

• Shifts toward value-based pricing
• Escalating healthcare cost containment pressures

4.3. Competitive Dynamics

• Emergence of combination therapies like Trikafta dilutes Kalydeco’s market share
• Patent expirations approaching in specific jurisdictions
• Potential biosimilars or generics unlikely due to biologic complexity

4.4. Patent and Exclusivity Status

Patent Expiry	Comments
2029–2030	Patents on Ivacaftor composition and formulation
Market exclusivity	Expected to extend through regulatory data protections

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5. Comparative Analysis: Kalydeco vs. Trikafta

Aspect	Kalydeco	Trikafta (Elexacaftor/Tezacaftor/Ivacaftor)
Indications	Gating mutations only	Broader; includes F508del with gating mutations
Years to Market	2012	2019
Revenue (2022)	~$1.5 billion	~$2.8 billion
Price (annual)	~$294,000	~$310,000
Market Penetration	Limited to mutation-specific groups	Largest CF therapy by revenue
Development Cost	High, but focused on mutation-specific drugs	Higher, due to combination and broader application

Conclusion: Trikafta’s broader indication has overshadowed Kalydeco, but Kalydeco remains relevant in mutation-specific therapy.

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6. Future Outlook

6.1. Indication Expansion and Global Reach

• Potential approval for additional mutations
• Extension into pediatric populations (<2 years)
• Entry into Asian markets with expanding healthcare infrastructure

6.2. Potential Disruptors

• Gene editing (CRISPR-based therapies)
• Next-generation CFTR correctors and potentiators
• Regulatory policy shifts towards affordability

6.3. Revenue Forecast (2023–2028)

Year	Estimated Revenue (USD)	Key Assumptions
2023	~$1.6 billion	Continued expansion, stable prices
2024	~$1.7 billion	Increased global access
2025	~$1.8 billion	Further indications and pediatric approvals
2026	~$1.9 billion	Market saturation begins, incremental growth
2027	~$2.0 billion	Mature market, competition ongoing

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7. Key Takeaways

• Kalydeco pioneered the targeted therapy approach in CF but faces competition from newer combination treatments with broader indications.
• Revenue growth remains strong but is moderated by expanding competitors, high pricing, and regulatory evolution.
• Global expansion and indication expansion are central to sustaining its market trajectory.
• The drug’s high costs reinforce the importance of negotiation with payers and targeted patient access programs.
• Innovations in gene editing and personalized medicine could challenge Kalydeco’s market dominance in the long term.

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FAQs

1. How has the emergence of Trikafta affected Kalydeco's market?

Trikafta, approved in 2019, reports revenues of approximately $2.8 billion in 2022, surpassing Kalydeco due to its broader indication covering over 90% of CF patients. Consequently, Kalydeco’s share has declined, but it maintains relevance for mutation-specific subsets.

2. What regulatory approvals are expected to impact Kalydeco in the coming years?

Ongoing trials aim for approval in children under two and additional mutation types. Regulatory agencies in Asia and Latin America are also reviewing submissions, promising further market access.

3. What are the primary challenges for Kalydeco’s future growth?

Key challenges include increasing competition from more comprehensive therapies, high drug pricing constraining reimbursement, patent expirations, and the potential advent of gene editing therapies disrupting the current treatment paradigm.

4. How does Kalydeco’s pricing compare globally?

Prices vary significantly: the US lists approximately $294,000 annually, while European and other markets often negotiate rebates and discounts, leading to lower net prices.

5. What is the outlook for Kalydeco’s use in pediatric populations?

Regulatory bodies are evaluating data for use in children under two, which could substantially expand its market size, positively impacting future revenues.

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References

[1] Vertex Pharmaceuticals. (2022). Annual Report.

[2] FDA. (2012–2022). Official approval documents and updates.

[3] European Medicines Agency. (2014–2022). Summary of product characteristics.

[4] IQVIA. (2023). State of the Pharmaceutical Market Data.

[5] ClinicalTrials.gov. (2022). Ongoing studies on CF therapies.

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This analysis equips stakeholders with a comprehensive understanding of Kalydeco’s market landscape, financial trajectory, and strategic outlook, enabling informed decision-making amidst a rapidly evolving CF treatment market.