IVACAFTOR; LUMACAFTOR - Generic Drug Details

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

Ivacaftor; lumacaftor is the generic ingredient in one branded drug marketed by Vertex Pharms Inc and is included in two NDAs. There are twenty-three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Ivacaftor; lumacaftor has four hundred and sixty-one patent family members in thirty-six countries.

One supplier is listed for this compound.

Summary for IVACAFTOR; LUMACAFTOR

International Patents:	461
US Patents:	23
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Clinical Trials:	37
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for IVACAFTOR; LUMACAFTOR
DailyMed Link:	IVACAFTOR; LUMACAFTOR at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for IVACAFTOR; LUMACAFTOR

Dosage:

GRANULE;ORAL

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; LUMACAFTOR

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

Sponsor	Phase
Azienda Ospedaliera Universitaria Integrata Verona	PHASE2
Qanatpharma Canada LTD	Phase 1
Children's Hospital Medical Center, Cincinnati	N/A

See all IVACAFTOR; LUMACAFTOR clinical trials

Pharmacology for IVACAFTOR; LUMACAFTOR

Drug Class	Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
Mechanism of Action	Chloride Channel Activation Potentiators Cytochrome P450 2B6 Inducers Cytochrome P450 2C19 Inducers Cytochrome P450 2C8 Inducers Cytochrome P450 2C8 Inhibitors Cytochrome P450 2C9 Inducers Cytochrome P450 2C9 Inhibitors Cytochrome P450 3A Inducers Cytochrome P450 3A Inhibitors P-Glycoprotein Inducers P-Glycoprotein Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for IVACAFTOR; LUMACAFTOR

R07AX	Other respiratory system products
R07A	OTHER RESPIRATORY SYSTEM PRODUCTS
R07	OTHER RESPIRATORY SYSTEM PRODUCTS
R	Respiratory system

R07AX	Other respiratory system products
R07A	OTHER RESPIRATORY SYSTEM PRODUCTS
R07	OTHER RESPIRATORY SYSTEM PRODUCTS
R	Respiratory system

US Patents and Regulatory Information for IVACAFTOR; LUMACAFTOR

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	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No					Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-003	Sep 2, 2022		RX	Yes	No					Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-001	Jul 2, 2015		RX	Yes	Yes					Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-002	Aug 7, 2018		RX	Yes	Yes					Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-002	Sep 28, 2016		RX	Yes	No					Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-001	Jul 2, 2015		RX	Yes	Yes					Y

EU/EMA Drug Approvals for IVACAFTOR; LUMACAFTOR

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Vertex Pharmaceuticals (Ireland) Limited	Orkambi	lumacaftor, ivacaftor	EMEA/H/C/003954Orkambi tablets are indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation in the CFTR gene.Orkambi granules are indicated for the treatment of cystic fibrosis (CF) in children aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene.	Authorised	no	no	no	2015-11-19

International Patents for IVACAFTOR; LUMACAFTOR

Country	Patent Number	Title	Estimated Expiration
Hungary	E036512
Slovenia	1773816
New Zealand	602795
European Patent Office	1993360	FORMES SOLIDES DE N-[2,4-BIS(1,1-DIMETHYLETHYL)-5-HYDROXYPHENYL]-1,4-DIHYDRO-4-OXOQUINOLEINE-3-CARBOXAMIDE (SOLID FORMS OF N-[2,4-BIS(1,1-DIMETHYLETHYL)-5-HYDROXYPHENYL]-1,4-DIHYDRO-4-OXOQUINOLINE-3-CARBOXAMIDE)
Australia	2010249302	Modulators of ATP-Binding Cassette Transporters
Canada	2810655	MODULATEURS DE TRANSPORTEURS DE CASSETTE DE LIAISON A L'ATP (MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS)

Supplementary Protection Certificates for IVACAFTOR; LUMACAFTOR

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3170818	2090033-8	Sweden		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 AND (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3-CARBOXAMIDE OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; FIRST MARKETING AUTHORIZATION NUMBER SE: EG EU/1/15/1059, 2015-11-24; DEN 19 JUNI 2025 MEDDELADE PRV BESLUT OM RAETTAD SKYDDSTID FOER FOELJANDE TILLAEGGSSKYDD. 1790034-1 1490061-7 1790019-2 2090047-8 2390009-5 2390012-9 2090033-8 1790016-8 2190015-4 1990020-8 1090038-9 1190029-7 SKYDDSTIDEN FOER SAMTLIGA DESSA TILLAEGGSSKYDD AER FOERLAENGD MED EN DAG, I ENLIGHET MED PATENT- OCH MARKNADSDOMSTOLENS BESLUT I PMAE 7804-24.
1773816	35/2015	Austria		PRODUCT NAME: N-(5-HYDROXY-2,4DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLIN-3-CARBOXAMID ODER EIN PHARMAZEUTISCH ANNEHMBARES SALZ DAVON; REGISTRATION NO/DATE: EU/1/12/782/001, EU/1/12/782/002 (MITTEILUNG) 20120725
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 (IVACAFTOR) OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTRATION 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

Market Dynamics and Financial Trajectory for Ivacaftor and Lumacaftor

Last updated: February 15, 2026

Market Overview

Ivacaftor (brand name: Kalydeco) and lumacaftor (part of Orkambi, combined with ivacaftor) are cystic fibrosis (CF) therapies developed by Vertex Pharmaceuticals. Their commercialization landscape is shaped by regulatory approvals, competitive positioning, pricing strategies, and expanding indications.

Regulatory Approvals and Indications

• Ivacaftor was approved by the FDA in 2012 for CF patients with specific G551D mutations. Subsequent approvals expanded to over 30 mutations, with a broader pediatric label in 2015.
• Lumacaftor received FDA approval in 2015 as part of Orkambi (lumacaftor/ivacaftor), targeting patients aged 12 and older with homozygous Phe508del mutations.
• Key: The approval of Tezacaftor and Elexacaftor expanded the landscape, offering alternatives with different mutation spectra.

Market Size and Revenue

• Global CF market value was approximately $5.4 billion in 2022, projected to reach around $9 billion by 2030.
• Vertex’s revenue from CF treatments was $7.39 billion in 2022, with ivacaftor-based therapies accounting for roughly 80% of sales.
• Ivacaftor's annualized revenue exceeds $3 billion globally, driven by its label expansions and increasing diagnosed patient base.
• Orkambi markets primarily in the U.S., Europe, and Japan, generating significant revenue but facing reimbursement challenges due to pricing debates.

Pricing and Reimbursement

• Ivacaftor’s list price varies: in the U.S., it exceeds $300,000 annually per patient. Similar high-price points exist in Europe.
• Reimbursement landscape is complex; payers impose strict criteria. Medicaid and pharmacy benefit managers often restrict access, impacting overall sales volume.
• The high cost has prompted pricing negotiations and alternative access programs.

Market Dynamics Factors

• Competitive landscape: Orkambi faces competition from tezacaftor/ivacaftor (Symdeko) and elexacaftor/tezacaftor/ivacaftor (Trikafta), which offer broader mutation coverage and improved efficacy.
• Prescribed population growth: Increasing diagnosis rates, including in adults, expand the eligible patient base.
• Regulatory pressures: Push toward pricing transparency and value-based contracting influences revenue realization.
• Pipeline progress: Vertex's development of next-generation modulators and gene therapies could neutralize or supplement current revenue streams.

Financial Trajectory

• Revenue growth: Ivacaftor-driven revenues are expected to grow at mid-single digit percentages annually through 2030, driven by label expansions and increasing adult diagnoses.
• Margins: Gross margins are high (above 85%) due to the high price and low manufacturing costs. Operating margins will depend on R&D and commercialization expenses.
• Profitability outlook: Vertex projects sustained profitability, with a focus on pipeline diversification as patent expiries loom in the mid-2020s.

Impact of Patent Expiry and Biosimilars

• Patent exclusivity for ivacaftor is scheduled to extend into early 2025.
• Patent challenges and biosimilar entrants, especially in Europe, could pressure prices and reduce revenue; however, regulatory pathways for biosimilars remain complex, and market penetration may be slow.

Future Market Trends

• Growth driven by new mutation coverage, expanded age approvals, and personalized medicine.
• Entry of gene editing therapies could alter the landscape but remain years away.
• Pricing reforms in multiple geographies could impact profit margins.

Summary Table of Key Data

Metric	2022	Future projections (2025-2030)
Global CF market	$5.4B	$8.5–9B
Vertex CF revenue	$7.39B	+5–8% annually
Ivacaftor share	~80% of Vertex CF sales	Stable
Pricing per patient	>$300,000/year (U.S.)	Slight adjustments expected
Patent expiry	2025 (US/Europe)	+2 years extension potential

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Key Takeaways

• Ivacaftor remains a core revenue driver with robust growth prospects linked to expanded indications.
• Lumacaftor’s contribution is declining due to newer combination therapies with broader mutation coverage.
• Market competition from next-generation modulators is accelerating, with Elexacaftor/Tezacaftor/Ivacaftor (Trikafta) leading in market share.
• Pricing pressures and biosimilar entrants pose long-term risks but are partially offset by pipeline innovation.
• Continued growth depends on label expansions, adult diagnosis, and clinical advancements in CF management.

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FAQs

1. How does the patent expiry affect ivacaftor’s market?
Patent expiry in early 2025 could open the market to biosimilars and generics, potentially reducing revenue. However, regulatory, patent litigation, and market adoption complexities may delay significant erosion.

2. Are there generic or biosimilar versions available for ivacaftor?
No approved biosimilars exist as of 2023; biosimilar development is ongoing, primarily in Europe, but market penetration remains limited.

3. What competitive therapies are challenging ivacaftor’s dominance?
The main competitor is Trikafta, a three-drug combination offering broader mutation coverage and higher efficacy, capturing significant market share.

4. What factors influence the pricing strategy for ivacaftor?
Pricing depends on clinical value, regulatory environment, payer negotiations, and competitive alternatives. Value-based approaches are increasingly adopted.

5. How might pipeline therapies impact long-term revenue?
Innovations such as gene editing and next-generation modulators aim to treat CF more broadly, potentially displacing current therapies and redefining revenue streams.

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Sources

1. Vertex Pharmaceuticals. (2023). Annual Reports and Investor Presentations.
2. IQVIA. (2022). Global Oncology and Rare Disease Market Data.
3. FDA. (2022). Drug Approvals and Labeling.
4. Evaluate Pharma. (2023). Oncology and Rare Disease Industry Intelligence.
5. Health Economics and Outcomes Research. (2022). CF Market and Pricing Analysis.