kalydeco Drug Patent Profile

Which patents cover Kalydeco, and when can generic versions of Kalydeco launch?

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

NDA:

207925

Dosage:

GRANULE;ORAL

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.

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					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
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-005	May 3, 2023		RX	Yes	No					Y
Vertex Pharms	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012		RX	Yes	Yes					Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No					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	207925-001	Mar 17, 2015
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-004	May 3, 2023
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-005	May 3, 2023
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-002	Mar 17, 2015
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019
Vertex Pharms	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012

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
Lithuania	3219705
Portugal	1773816
Canada	2865519
Mexico	2012001939
South Africa	201200722
Russian Federation	2019116577

Supplementary Protection Certificates for kalydeco

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
1773816	CR 2015 00038	Denmark		PRODUCT NAME: N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLIN-3-CARBOXAMID ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF; REG. NO/DATE: EU/1/12/782/001-002 20120725
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
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
1773816	C01773816/01	Switzerland		PRODUCT NAME: IVACAFTOR; REGISTRATION NO/DATE: SWISSMEDIC 62686 13.01.2014
1773816	PA2015028	Lithuania		PRODUCT NAME: IVACAFTORUM; REGISTRATION NO/DATE: EU/1/12/782/001 - EU/1/12/782/002 20120723

Market Dynamics and Financial Trajectory for KALYDECO (ivacaftor)

Last updated: July 27, 2025

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Introduction

KALYDECO, with the generic name ivacaftor, is a groundbreaking medication developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis (CF)—a genetic disorder characterized by thick, sticky mucus that compromises respiratory and digestive functions. Approved by the FDA in 2012 for patients with specific CF mutations, KALYDECO has evolved into a cornerstone in personalized medicine, significantly impacting both therapeutic markets and pharmaceutical financial trajectories. This report analyzes the current market landscape, key factors influencing demand, competitive positioning, and financial projections for KALYDECO over the coming decade.

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Market Dynamics

1. Growing Prevalence of Cystic Fibrosis

Globally, CF affects roughly 70,000 to 100,000 individuals, predominantly of Caucasian descent, with an estimated 1 in 3,000 live births in the U.S. affected by the disease [1]. Advances in genetic screening and newborn screening programs have led to earlier detection, increasing diagnosed cases and fueling demand for targeted therapies like KALYDECO.

2. Expansion of Approved Indications

Initially approved for CF patients with the G551D mutation, KALYDECO's indications expanded to include additional mutations such as S945L and G1349D. Notably, the European Medicines Agency (EMA) approved KALYDECO as a personalized medicine for multiple mutations, broadening its potential patient base. As of 2023, Vertex obtained FDA and EMA approvals for KALYDECO in patients with specific gating mutations, covering about 10% of the CF population [2].

Moreover, in 2017, Vertex initiated development of combination therapies (e.g., ORKAMBI and TRIKAFTA) that extend KALYDECO’s reach to patients with F508del mutations, which constitute approximately 90% of CF cases worldwide. These combinations underscore an integrated strategic approach to capturing larger market segments.

3. Competitive Landscape and Differentiators

While KALYDECO pioneered mutation-specific therapy, newer CF drugs have entered the market, notably Trikafta (elexacaftor + tezacaftor + ivacaftor), approved in 2019 for a broader patient group—including those with F508del mutation—creating direct competition and impacting KALYDECO’s traditional niche. However, KALYDECO retains a niche among patients with specific gating mutations who are ineligible for newer combination therapies.

4. Pricing and Reimbursement Environment

KALYDECO’s high price point—approximating $300,000 annually per patient in the U.S.—reflects its precision medicine nature and R&D investment. Reimbursement negotiations with insurers and government healthcare programs heavily influence market access, especially as payers push back against rising drug costs. Nevertheless, the high unmet medical need and clear clinical benefit sustain payor support for mutation-specific therapies.

5. Global Market Penetration and Access

Vertex’s distribution strategy emphasizes expanding global access, particularly in Europe and emerging markets. Patent protections until at least 2030 secure exclusivity, allowing premium pricing. However, biosimilar and generic entrants are unlikely in the immediate future due to patent protections and high manufacturing complexity, providing the company with a period of market exclusivity and profit maximization.

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Financial Trajectory

1. Revenue Generation and Growth Drivers

KALYDECO's revenue has historically exhibited strong growth, driven by expanding indications and a growing CF patient population. In 2022, Vertex reported KALYDECO sales exceeding $1.2 billion globally, reflecting robust demand primarily in the U.S. and Europe [3].

Increased adoption of approved mutation-specific indications and the complementarity of combination therapies elevate sales prospects. The upcoming expansion into additional geographic markets—such as Asia—may further augment revenues, contingent on regulatory approvals.

2. Impact of Combination Therapies and Competition

The launch of Trikafta and similar drugs has shifted market dynamics. While Trikafta offers broader reimbursement potential by treating F508del mutations in approximately 90% of CF patients, KALYDECO’s niche remains intact among mutation-specific subpopulations. Vertex strategically positions KALYDECO as part of combination regimens (e.g., with tezacaftor or elexacaftor) to maintain relevance.

However, competition from emerging biosimilars and potential generic formulations (post-2030 patent expiration) could commoditize the drug, pressuring prices and margins.

3. Pipeline and Future Growth Opportunities

Vertex’s pipeline includes next-generation CF modulators designed for broader mutation coverage and improved efficacy. Success in these development programs could either cannibalize KALYDECO’s sales or complement its existing portfolio, especially if new formulations demonstrate better tolerability or convenience.

Additionally, the platform approach of mutation-specific therapies may allow Vertex to leverage advances in gene editing, mRNA technology, and personalized medicine to extend KALYDECO’s market relevance or launch successor therapies.

4. Pricing Strategy and Market Access

Premium pricing sustains revenue streams, but escalating healthcare costs impose pressure on payers. Value-based agreements, outcomes-based reimbursement, and patient assistance programs are critical tools to uphold market penetration while addressing access challenges.

Vertex’s ability to negotiate favorable reimbursement terms and demonstrate clinical benefits remains vital for maintaining the drug’s financial trajectory.

5. Regulatory and Patent Considerations

Patent exclusivity until at least 2030 affords Vertex a period of protected market dominance. Regulatory decisions for new indications or label expansions can extend revenue streams. Conversely, patent challenges or the advent of generics could erode profit margins, emphasizing the importance of strategic patent management.

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Strategic Outlook and Market Forecast

Analysts project that KALYDECO will continue to generate substantial revenues until at least 2025, with growth potentially stabilizing or declining as newer therapies like Trikafta expand their market share. However, niche markets—such as rare mutations—will sustain parts of KALYDECO’s demand.

Long-term projections suggest a compound annual growth rate (CAGR) of approximately 2-3% through 2028, driven by global expansion and mutation identification. The entry of biosimilars post-2030 could significantly impact profitability, but aggressive patent protection, combination strategies, and pipeline innovations aim to prolong market viability.

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Key Takeaways

• Growth Drivers: Mutation-specific approvals, expanding global markets, and the integration of KALYDECO into combination therapies sustain revenue streams.
• Market Challenges: Competition from broader-coverage drugs like Trikafta, healthcare cost containment, and potential patent expiry pose risks.
• Financial Strategy: Maintaining premium pricing, expanding indications, and leveraging early pipeline successes are vital for continued growth.
• Long-Term Outlook: KALYDECO will likely remain a profitable niche therapy until biosimilar competition and patent expirations, with strategic diversification essential for sustained success.
• Investment Relevance: Stakeholders should monitor regulatory developments, pipeline progress, and market penetration strategies to inform investment decisions.

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FAQs

1. Will KALYDECO remain commercially viable after the entry of Trikafta?
Yes, KALYDECO will likely retain a niche market among patients with specific gating mutations ineligible for Trikafta, ensuring continued demand despite broader-acting therapies.

2. How does Vertex plan to extend KALYDECO’s market presence?
Vertex focuses on expanding indications, partnering for global access, developing combination therapies, and advancing pipeline innovations targeting less common mutations.

3. What is the risk of biosimilar competition for KALYDECO?
Biosimilar entry post-2030 is possible once patents expire, potentially reducing prices and market share, emphasizing the importance of patent protection and pipeline diversification.

4. How do healthcare reimbursement policies affect KALYDECO’s financial trajectory?
Reimbursement negotiations directly influence market access; value-based contracts and patient assistance programs are crucial for maintaining revenue streams amid cost containment pressures.

5. What are the prospects for growth in emerging markets?
Global expansion efforts, especially in regions with rising genetic screening programs, are expected to contribute to revenue growth, contingent on regulatory approvals and pricing strategies.

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References

[1] Cystic Fibrosis Foundation. "Cystic Fibrosis Foundation Patient Registry Annual Data Report." 2022.
[2] European Medicines Agency. "KALYDECO (ivacaftor): European Public Assessment Report." 2023.
[3] Vertex Pharmaceuticals. "Financial Reports and Investor Presentations." 2022.