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:	1,359
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:

11,147,770*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 North Carolina	Early Phase 1
University of Miami	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 fourteen US patents and ten 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	12,214,083*PED				Y
Vertex Pharms	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012		RX	Yes	Yes	7,495,103*PED				Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	7,495,103*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	207925-001	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

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
Serbia	61408	FARMACEUTSKA KOMPOZICIJA I NJENO ORDINIRANJE (PHARMACEUTICAL COMPOSITION AND ADMINISTRATIONS THEREOF)
Cyprus	1121572
Denmark	3219705

Supplementary Protection Certificates for KALYDECO

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

KALYDECO (ivacaftor): Market Dynamics and Financial Trajectory

Last updated: May 1, 2026

How has KALYDECO’s market evolved across CF pricing, access, and competitor pressure?

KALYDECO is a cystic fibrosis (CF) targeted therapy built around gating mutations and marketed by Vertex. The revenue base has been shaped by three forces: (1) label expansion and growing treatable mutation share, (2) payer contracting in the US and Europe, and (3) the steady shift of CF care toward combination regimens that can reduce the standalone addressable market.

Core market structure

• Use-case segmentation: ivacaftor is used for people with specific gating mutations and, in practice, competes with other Vertex CF products and non-Vertex options where patients and payers prefer combination coverage.
• Geographic split: Vertex’s CF commercial footprint is strongest in the US and Europe, where pricing is highest but payer scrutiny is also strongest.

Mutation share and label breadth

• KALYDECO’s treatable population has expanded as Vertex has added gating mutations and refined inclusion criteria through regulatory actions over time.
• The financial effect shows up as durable base revenue even as the broader CF pipeline shifted attention toward multi-drug combination regimens.

Pricing and access

• KALYDECO revenue is exposed to annual price management cycles, manufacturer contracting, and reimbursement rules that can create net price volatility even when gross list prices remain stable.
• In the US, the net price environment for specialty drugs is shaped by PBM and payer rebates, state-level coverage policies, and patient-assistance structures.

Competitive dynamics

• The dominant competitive set is not a single product but the CF regimen standard of care.
• As Vertex advanced combinations (and as competitors developed CF modulators and adjunct therapies), ivacaftor’s role increasingly functions inside broader regimen decisions rather than as the only modulatory option for many mutation-defined patients.

What do the financial trajectory markers show for KALYDECO?

Vertex does not report KALYDECO revenue as a standalone line item in its public segment reporting. However, investor reporting still provides a usable trajectory through Vertex’s product-level disclosures and the CF franchise trend.

Vertex franchise reality (directional)

• KALYDECO’s performance is best interpreted as the foundation revenue of the CF franchise, with later CF launches changing mix.
• Over time, KALYDECO’s revenue growth rate typically moderates as:
  • Combination regimens shift incremental patients away from monotherapy,
  • Price pressure and payer contracting create headwinds,
  • Patent-life events or label normalization can reduce marginal growth even when the category expands.

What to look for in Vertex financials Use these markers when assessing KALYDECO’s financial trajectory in Vertex reporting:

1. Net sales and product mix within the CF portfolio
2. International expansion and reimbursement changes (Europe and select Asia markets where access policy evolves)
3. R&D-driven label expansions that increase eligible patient counts (watch for regulatory headline timing)
4. Commercial milestones for CF combinations that can cannibalize or complement ivacaftor use
5. Gross-to-net dynamics (rebates, chargebacks, and payer mix)

How do net sales growth drivers and headwinds map to KALYDECO economics?

KALYDECO economics follow typical specialty drug levers: volume (eligible population), net price, and duration of use.

Growth drivers

• Increasing eligible population from mutation-specific label scope.
• Sustained dosing adherence because CF modulators are chronic treatments.
• Broader geographic reimbursement for CF modulators over time, increasing market access.

Headwinds

• Net price erosion through contracting and rebate pressure.
• Regimen mix shift toward combination therapies, reducing incremental monotherapy demand.
• Competition for specific mutation subsets as other companies pursue overlapping indications.
• Treatment line changes when clinicians prefer multi-drug approaches with higher overall efficacy.

How do reimbursement structures influence KALYDECO’s realized price?

Realized price is where specialty brands typically diverge from list price. For CF modulators, reimbursement is shaped by:

• Prior authorization workflows tied to genotype documentation.
• Formulary tier placement and managed care restrictions.
• Patient assistance and copay support in the US that affects patient persistence but does not always translate linearly into net sales.

This matters because KALYDECO’s growth path depends on the gap between:

• eligible patient growth and access, and
• realized net price after payer contracting.

What is the likely financial trajectory given Vertex’s CF product mix evolution?

The most defensible trajectory for KALYDECO is a maturing growth profile rather than sustained high growth once combinations dominate incremental uptake.

A practical framework:

• Base-case pattern: ivacaftor remains a high-margin anchor while the CF franchise expands.
• Mix effect: newer combinations capture increasing share of eligible patients over time, shifting revenue growth from ivacaftor to the broader portfolio.
• Net price: tends to compress gradually under payer negotiations.
• Regulatory and payer actions: can create short, discrete steps in volume (label expansion) that fade into a long tail.

What are the business implications for R&D, partnership, and investment decisions?

R&D strategy signals

• Ivacaftor remains a platform anchor for Vertex’s gating biology and regimen design.
• The financial trajectory is shaped by whether new entrants and internal pipeline designs can:
  • expand patient share further, or
  • reduce ivacaftor’s role in routine regimens.

Partnership and BD leverage points

• Look for opportunities where “mutation coverage” expands the total addressable patient pool without displacing existing regimens.
• The revenue impact is strongest when a new indication adds patients rather than replaces an existing regimen component.

Investment underwriting

• Underwrite KALYDECO through franchise-level sensitivity:
  • patient counts (mutation eligibility and age-based expansion),
  • net price trends in the US and major EU markets,
  • and mix shift into combination products.

Key performance indicators to track for KALYDECO’s trajectory

These KPIs translate market dynamics into financial outcomes:

1. CF modulators sales growth vs total Vertex growth (franchise resilience)
2. US net sales momentum in Vertex reporting periods (payer pressure signal)
3. International adoption metrics embedded in regional reporting (access signal)
4. Regulatory label changes for ivacaftor (volume step function)
5. Combination regimen uptake trends (mix cannibalization risk)

What is the essential fact pattern on KALYDECO’s commercialization and designation?

• Drug: KALYDECO (ivacaftor)
• Company: Vertex Pharmaceuticals
• Therapy class: CF transmembrane conductance regulator (CFTR) potentiator
• Commercial thesis: mutation-specific CFTR modulation with chronic dosing

Market map: where KALYDECO sits in CF treatment decision-making

Patient pathway (high-level)

• Genotype identifies eligibility for gating mutations.
• Clinicians select modulators based on mutation class, age, comorbidities, and regimen goals.
• Payers require genotype documentation and authorization, affecting conversion from eligible to treated.

Implication for revenue

• Conversion depends on access friction and payer coverage.
• Revenue growth depends less on broad CF incidence trends and more on genotype-eligible patient conversion and continued access.

Key Takeaways

• KALYDECO’s market dynamics follow mutation eligibility growth, payer access constraints, and a long-term mix shift toward CF combination regimens that can moderate ivacaftor standalone growth.
• The most reliable view of financial trajectory is through Vertex’s CF franchise trend and realized net sales behavior rather than expecting ivacaftor-only line-item reporting.
• Underwriting should focus on (1) eligible population conversion, (2) net price pressure, and (3) regimen mix changes driven by combination uptake and evolving clinical standards.

FAQs

1. Is KALYDECO the main revenue driver within Vertex’s CF portfolio?
   It is historically foundational, but growth rates can be affected by mix shift into combination regimens over time.

2. What primarily drives KALYDECO revenue: pricing or volume?
   Both matter. Volume depends on mutation eligibility conversion, while pricing depends on net reimbursement after contracting.

3. How does payer reimbursement affect KALYDECO performance?
   Prior authorization and formulary positioning influence conversion from eligible patients to treated patients, and rebate contracting influences realized net price.

4. Does combination therapy replace KALYDECO?
   It can reduce incremental monotherapy demand by shifting eligible patients into combination regimens, affecting mix even if total CF treatment demand grows.

5. What events most change the ivacaftor revenue curve?
   Label expansions, major access policy changes, and shifts in regimen standard of care that alter treated patient mix.

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References

[1] Vertex Pharmaceuticals. Annual Report on Form 10-K and SEC filings (product and financial disclosures). Vertex Investor Relations.
[2] FDA. KALYDECO (ivacaftor) prescribing information and labeling history. U.S. Food and Drug Administration.
[3] EMA. KALYDECO (ivacaftor) product information. European Medicines Agency.
[4] Vertex Pharmaceuticals. Press releases and regulatory updates for CFTR modulators and label expansions. Vertex Investor Relations.