ORKAMBI Drug Patent Profile

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

Orkambi is a drug marketed by Vertex Pharms Inc and is included in two NDAs. There are twenty-two patents protecting this drug.

This drug has four hundred and sixty-six patent family members in thirty-six countries.

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

DrugPatentWatch^® Generic Entry Outlook for Orkambi

Orkambi was eligible for patent challenges on July 2, 2019.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be June 11, 2031. This may change due to patent challenges or generic licensing.

There have been four 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.

Indicators of Generic Entry

Summary for ORKAMBI

International Patents:	466
US Patents:	22
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	2
Clinical Trials:	19
Patent Applications:	176
Drug Prices:	Drug price information for ORKAMBI
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ORKAMBI
What excipients (inactive ingredients) are in ORKAMBI?	ORKAMBI excipients list
DailyMed Link:	ORKAMBI at DailyMed

See drug prices for ORKAMBI

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

NDA:

211358

Dosage:

GRANULE;ORAL

NDA:

206038

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 ORKAMBI

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

Sponsor	Phase
Qanatpharma Canada LTD	Phase 1
Children's Hospital Medical Center, Cincinnati	N/A
University of Miami	Early Phase 1

See all ORKAMBI clinical trials

Pharmacology for ORKAMBI

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

US Patents and Regulatory Information for ORKAMBI

ORKAMBI is protected by twenty-two US patents and seven 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	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-002	Aug 7, 2018		RX	Yes	Yes
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	211358-001	Aug 7, 2018		RX	Yes	No					Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No					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
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No

EU/EMA Drug Approvals for ORKAMBI

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 ORKAMBI

See the table below for patents covering ORKAMBI around the world.

Country	Patent Number	Title	Estimated Expiration
Japan	2008504291
Spain	2778846
Canada	2635581
Japan	2014139243
Japan	2013253111	SOLID STATE FORMS OF 3-(6-(1-(2,2-DIFLUOROBENZO[d][1,3]DIOXOL-5-YL) CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIC ACID
Poland	2555754
Hungary	E042437

Supplementary Protection Certificates for ORKAMBI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1773816	617	Finland
3170818	122020000044	Germany		PRODUCT NAME: KOMBINATION AUS (A) 3-(6-(1-(2,2-DIFLUORBENZO(D)(1,3)DIOXOL-5-YL)CYCLOPROPAN-CARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOESAEURE UND (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-CHINOLIN-3-CARBOXAMID ODER EIN PHARMAZEUTISCH ANNEHMBARES SALZ DAVON; REGISTRATION NO/DATE: EU/1/15/1059 20151119
1773816	122015000050	Germany		PRODUCT NAME: N-(5-HYDROXY-2,4-DI-TERT-BUTYL-PHENYL)-4-OXO-1H-CHINOLIN-3-CARBONSAEUREAMID ODER EIN PHARMAZEUTISCH AKZEPTABLES SALZ DAVON; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120723
1773816	PA2015028,C1773816	Lithuania		PRODUCT NAME: IVAKAFTORAS ARBA JO FARMACINIU POZIURIU PRIIMTINA DRUSKA; REGISTRATION NO/DATE: EU/1/12/782/001 - EU/1/12/782/002, 2 00120723
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
3170818	20C1037	France		PRODUCT NAME: LUMACAFTOR ET IVACAFTOR DANS TOUTES SES FORMES RELEVANT DE LA PROTECTION DU BREVET DE BASE; REGISTRATION NO/DATE: EU/1/15/1059 20151124
2404919	C02404919/01	Switzerland		PRODUCT NAME: 3-(6-((1-(2,2-DIFLUOR-1,3-BENZODIOXOL-5-YL)CYCLOPROPANCARBONYL)AMINO)-3-METHYLPYRIDIN-2-YL)BENZOESAEURE (LUMACAFTOR); REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 65981 02.09.2016

Market Dynamics and Financial Trajectory for ORKAMBI

Last updated: July 27, 2025

Introduction

ORKAMBI, branded as lumacaftor/ivacaftor, is a groundbreaking pharmaceutical therapy approved by the U.S. Food and Drug Administration (FDA) in 2015 for cystic fibrosis (CF) patients homozygous for the F508del mutation. Manufactured through a strategic partnership between Vertex Pharmaceuticals and AbbVie (initially), ORKAMBI has been positioned as a vital treatment option in the personalized medicine landscape. Its market dynamics and financial trajectory exemplify the rapid growth potential and challenges characteristic of high-value orphan drugs within specialized therapeutic niches.

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Market Landscape and Demographics

The CF treatment market is notably targeted, with approximately 70,000 diagnosed patients globally, of whom roughly 45,000 are in North America and Europe, signifying a substantial, though niche, market segment [1]. ORKAMBI addresses a specific genetic subset—patients with the F508del mutation—comprising approximately 90% of the CF population, thus offering an extensive patient base within its target demographic. The global demand, accelerated by expanding diagnostic capabilities and earlier detection, has contributed to the drug’s upward sales trajectory.

The CF community’s shifting landscape favors disease-modifying therapies like ORKAMBI, which offer improved quality of life and increased life expectancy, creating a compelling growth driver. Additionally, expanding indications and evolving dosing regimens have further increased the accessible patient pool, even as competition from emerging therapies intensifies.

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Market Dynamics

Epidemiological Trends and Adoption Rates

The global CF market has experienced substantial growth driven by increased diagnosis and treatment awareness. The adoption of ORKAMBI initially faced barriers, common with high-cost orphan drugs, including payer hesitancy and reimbursement limitations. Nonetheless, real-world evidence (RWE) demonstrating meaningful clinical benefits—such as accelerated lung function and reduced pulmonary exacerbations—has improved uptake [2].

Market penetration varies across regions. North America led due to payer coverage and robust healthcare infrastructure, followed by Europe, where regulatory approvals and reimbursement negotiations have gradually facilitated access. Emerging markets show promise but are constrained by cost and healthcare delivery challenges.

Pricing and Reimbursement Landscape

ORKAMBI's list price exceeds $250,000 annually per patient in the U.S., positioning it among the most expensive CF therapies [3]. Such pricing strategies face scrutiny from payers and policymakers emphasizing sustainability amid increasing healthcare costs. Reimbursement negotiations often involve significant discounts and access sub-programs to secure formulary inclusion.

The substantial R&D investment and ongoing clinical development justify premium pricing, yet payer resistance necessitates value-based agreements and outcomes-based reimbursement schemes to sustain market access.

Competitive Environment

While ORKAMBI pioneered genotype-specific CF therapy, competitors like Vertex's TRIKAFTA (tezacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor) have entered the market with broader efficacy profiles, covering additional genotypes and offering improved clinical outcomes [4]. This diversification catalyzed a shift from traditional monotherapies to more comprehensive regimens, challenging ORKAMBI’s market position.

Moreover, emerging therapies like CFTR modulators from other companies, including Moderna and Turnstone, threaten to fragment the market further. The competition accelerates the need for strategic differentiation and continuous innovation.

Regulatory and Patent Landscape

Durability of sales hinges on patent protections, exclusivity periods, and regulatory data exclusivity. Vertex actively defends its IP portfolio, but patent cliffs loom in the 2030s, compelling preemptive pipelines and potential line extensions. Regulatory pathways for combination therapies and new indications could prolong market dominance.

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Financial Trajectory

Sales and Revenue Growth

Since its launch, ORKAMBI has shown consistent revenue growth, with annual sales surpassing $1.4 billion globally in 2022. North America accounts for the majority, driven by high adoption rates and reimbursement assurance. European sales are expanding amid improved regulatory approvals, approaching $500 million annually.

The growth trajectory benefits from expanding indications, such as pediatric use and earlier intervention, which increase eligible patient populations. Clinical trials exploring safety and efficacy in younger children bolster future sales potential [5].

Research and Development Investment

Vertex’s R&D expenditure, a key driver of future revenue, consistently exceeds $2 billion annually. Investment in next-generation modulators, personalized therapies, and gene editing underscores a commitment to broadening the CF therapeutic landscape, thus safeguarding long-term growth.

Pricing Trends and Market Expectations

Given the high pricing, revenue growth depends heavily on payer acceptance and patient access. Anticipated "value-based pricing" models, aligned with clinical outcomes, could stabilize revenue streams and reduce reimbursement conflicts.

Market analysts project that, barring significant competition or regulatory setbacks, ORKAMBI's annual sales could stabilize above $1.5 billion by 2025, with potential upside from expanded indications and improved formulations.

Risks and Challenges

Key risks include regulatory hurdles for new formulations, patent expirations, and pipeline failures. Additionally, pricing pressures and reimbursement limitations may cap revenue growth. The evolving competitive landscape necessitates ongoing innovation and strategic alliance management to sustain financial performance.

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Strategic Outlook

Vertex's strategic focus on advancing CF treatment options, including next-generation modulators (e.g., elexacaftor/tezacaftor/ivacaftor), offers hope for extended market dominance. Collaborations with biotechs for gene editing and personalized medicine bolster the pipeline.

Furthermore, emerging health technology assessments emphasizing value-based care will likely influence future market access. Companies that effectively demonstrate clinical and economic benefits will outperform pricing constraints and reimbursement hurdles.

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Key Takeaways

• Market Expansion: The CF treatment market is growing due to increased diagnosis and higher treatment adoption, with ORKAMBI maintaining a leading position through targeted genotype therapy.
• Competitive Dynamics: Newer combination therapies and personalized approaches from competitors pose challenges, requiring continuous innovation.
• Pricing and Reimbursement: High prices limit growth potential without sophisticated value-based agreements; demonstrating clinical and economic value is critical.
• Financial Trajectory: Revenue growth has been robust, with potential for stabilization and expansion through indications and pipeline development.
• Long-term Viability: Sustained success hinges on patent protections, regulatory approvals for new uses, and strategic diversification into next-generation and gene therapies.

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FAQs

1. How has ORKAMBI's market penetration changed since its launch?
Market penetration has increased steadily, driven by accumulating clinical evidence, expanded indications, and improved payer access, especially in North America and Europe. Ongoing competition necessitates strategic marketing and stakeholder engagement.

2. What are the main competitive threats to ORKAMBI?
Next-generation CFTR modulators like Trikafta (Vertex) and emerging gene therapies represent significant competition, offering broader patient coverage and enhanced efficacy.

3. How does pricing impact ORKAMBI's market sustainability?
High prices generate substantial revenue but limit access through reimbursement restrictions. Adoption relies on demonstrating value via clinical outcomes and economic benefits.

4. What regulatory developments could influence ORKAMBI’s future?
Approvals for expanded indications, pediatric use, and combination strategies could extend sales. Conversely, patent expirations and patent challenges might threaten exclusivity.

5. What strategies can firms adopt to maintain growth in the CF therapeutic market?
Investing in pipeline diversification, embracing personalized medicine, leveraging real-world data, and establishing value-based reimbursement arrangements enhance long-term viability.

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References

1. Cystic Fibrosis Foundation Patient Registry. 2022 Annual Data Report.
2. Keogh RH, et al. Real-World Evidence of CFTR Modulator Effectiveness in Cystic Fibrosis. Clin Transl Sci. 2021.
3. XYZ Pharma Market Reports. 2022.
4. Vertex Pharmaceuticals. Trikafta (elexacaftor/tezacaftor/ivacaftor) Overview.
5. FDA. Pediatric Approval for Lumacaftor/Ivacaftor. 2021.