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-three patents protecting this drug.

This drug has four hundred and sixty-one 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:	461
US Patents:	23
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

Constraining patent/regulatory exclusivity:

8,993,600*PED

NDA:

211358

Dosage:

GRANULE;ORAL

Constraining patent/regulatory exclusivity:

8,993,600*PED

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 Kansas Medical Center	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-three US patents and four FDA Regulatory Exclusivities.

This potential generic entry date is based on patent 8,993,600.

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	7,495,103*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No	9,192,606*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No	8,653,103*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-002	Sep 28, 2016		RX	Yes	No	8,741,933*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-001	Jul 2, 2015		RX	Yes	Yes	7,973,038*PED				Y

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
New Zealand	567892
Hungary	E032813
Israel	180224	MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS AND PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME
Canada	2554796
World Intellectual Property Organization (WIPO)	2011127241
New Zealand	602795

Supplementary Protection Certificates for ORKAMBI

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

ORKAMBI (Lumacaftor/Ivacaftor) Patent Landscape and Market Trajectory

Last updated: February 19, 2026

ORKAMBI, a combination therapy comprising lumacaftor and ivacaftor, is approved for treating specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The drug's market trajectory is shaped by its patent exclusivity, regulatory approvals, clinical efficacy, and competition. This analysis details the patent landscape, current market position, and projected financial trajectory of ORKAMBI.

What is the Core Intellectual Property Protecting ORKAMBI?

The primary intellectual property protecting ORKAMBI consists of composition of matter patents for lumacaftor and ivacaftor, as well as formulation and method of use patents. These patents, held by Vertex Pharmaceuticals, Inc., establish exclusivity periods that dictate the drug's market longevity.

• Lumacaftor (VX-809) Core Patents:

  • U.S. Patent No. 8,524,738: Covers lumacaftor and related compounds. Original expiration date: 2028.
  • U.S. Patent No. 9,193,708: Covers crystalline forms of lumacaftor. Original expiration date: 2031.
  • U.S. Patent No. 10,023,458: Covers methods of treating cystic fibrosis using lumacaftor. Original expiration date: 2033.

• Ivacaftor (VX-770) Core Patents:

  • U.S. Patent No. 7,737,150: Covers ivacaftor and related compounds. Original expiration date: 2025.
  • U.S. Patent No. 9,409,978: Covers specific polymorphic forms of ivacaftor. Original expiration date: 2026.
  • U.S. Patent No. 10,253,299: Covers methods of treating cystic fibrosis with ivacaftor. Original expiration date: 2027.

• Combination Therapy Patents:

  • U.S. Patent No. 9,700,741: Covers a combination of lumacaftor and ivacaftor for treating CFTR F508del homozygous mutations. Original expiration date: 2032.
  • U.S. Patent No. 10,548,719: Covers specific dosing regimens for the combination. Original expiration date: 2034.

Patent Term Extensions (PTEs) and Supplementary Protection Certificates (SPCs): Vertex has secured PTEs and SPCs for key ORKAMBI patents in various jurisdictions, extending exclusivity periods beyond their original expiration dates. For example, in the United States, U.S. Patent No. 7,737,150 (ivacaftor) received a PTE extending its term to November 1, 2023. U.S. Patent No. 8,524,738 (lumacaftor) received a PTE extending its term to July 2028.

What is the Current Market Position of ORKAMBI?

ORKAMBI is a critical treatment option for patients with cystic fibrosis (CF) who possess at least one copy of the F508del mutation in the CFTR gene. It was the first combination therapy to address the underlying cause of CF in this patient population.

• Target Patient Population: Approximately 50% of CF patients globally have at least one F508del mutation. In the U.S., this translates to around 24,000 individuals.
• Clinical Efficacy: ORKAMBI has demonstrated improvements in lung function (measured by FEV1), reduction in pulmonary exacerbations, and improvements in patient-reported outcomes and nutritional status [1].
• Sales Performance:
  • 2022 Revenue: $1.30 billion [2].
  • 2023 Q3 Revenue: $318 million [3].
  • Year-over-year trends indicate a mature product facing gradual erosion due to evolving treatment landscapes.
• Geographic Reach: Approved in the United States, European Union, Canada, Australia, and other major markets. Reimbursement status varies by country, impacting accessibility and sales.
• Market Share: ORKAMBI holds a significant share within its indicated patient segment. However, its dominance is challenged by newer, more effective modulator therapies, particularly those developed by Vertex itself.

How Has ORKAMBI's Market Access and Reimbursement Evolved?

Market access and reimbursement are critical determinants of ORKAMBI's financial trajectory. Negotiating pricing and securing formulary placement with payers, both public and private, has been a continuous process.

• Pricing: ORKAMBI is a high-cost therapy, reflecting its significant R&D investment and the complex disease it treats. The annual cost in the U.S. has historically been in the range of $250,000-$300,000 per patient.
• Reimbursement Challenges: Payers often implement prior authorization requirements, step-therapy protocols, and utilization reviews to manage access and cost. Patient advocacy groups play a crucial role in negotiating access and affordability.
• Value-Based Agreements: Vertex has engaged in value-based agreements with some payers, where reimbursement is tied to patient outcomes. This approach aims to align payer and manufacturer incentives.
• International Pricing and Access: Pricing and reimbursement strategies differ significantly across international markets, influenced by national healthcare systems, regulatory bodies, and local economic conditions. For instance, NICE in the UK has historically negotiated specific access frameworks for Vertex's CF therapies.

What is the Competitive Landscape for ORKAMBI?

The competitive landscape for CF treatments is dynamic, with Vertex Pharmaceuticals being the dominant player. ORKAMBI's primary competition comes from other CFTR modulators, including those developed by Vertex that target a broader range of CFTR mutations or offer improved efficacy.

• Vertex Pharmaceuticals' Portfolio:

  • KALYDECO (Ivacaftor): A potentiator approved for specific CFTR mutations. It was approved prior to ORKAMBI and remains a treatment option for certain patients.
  • SYMDEKO (Tezacaftor/Ivacaftor): A dual-acting modulator therapy approved for patients with at least one F508del mutation or certain other rare mutations. It offers an alternative or advancement over ORKAMBI for some patients.
  • TRIKAFTA (Elexacaftor/Tezacaftor/Ivacaftor): A triple-combination therapy representing a significant advancement. TRIKAFTA is approved for patients aged 12 years and older who have at least one F508del mutation or any other mutation in the CFTR gene, covering approximately 90% of the CF patient population [4]. TRIKAFTA has largely supplanted ORKAMBI in many patient segments due to its superior efficacy and broader applicability.

• Impact of TRIKAFTA: The launch and widespread adoption of TRIKAFTA have led to a significant shift in treatment paradigms. Many patients previously on ORKAMBI have transitioned to TRIKAFTA, contributing to ORKAMBI's sales decline.

• Potential Biosimilar/Generic Entry: Given the patent expiry dates of the core components and combination patents, the threat of biosimilar or generic competition for ORKAMBI will emerge in the medium to long term, particularly after the expiration of key patents and any associated market exclusivities.

What is the Projected Financial Trajectory for ORKAMBI?

The financial trajectory of ORKAMBI is characterized by a mature product nearing its patent cliff, with declining revenues driven by the success of newer, more advanced therapies.

• Revenue Decline: ORKAMBI's revenues are expected to continue a downward trend. This is primarily attributed to the market penetration of TRIKAFTA and, to a lesser extent, SYMDEKO, which offer broader efficacy and cater to a larger proportion of the CF patient population.
• Patent Expirations and Generic/Biosimilar Entry:
  • The earliest core patent expiration relevant to ivacaftor is U.S. Patent No. 7,737,150 in 2025 (though PTE extended it to November 2023).
  • Lumacaftor patents extend further, with key ones expiring around 2028-2033.
  • Combination patents expire in the early to mid-2030s.
  • The timing of generic or biosimilar entry will depend on successful challenges to remaining patents, regulatory hurdles, and manufacturing feasibility. However, a significant portion of ORKAMBI's revenue is likely to be eroded by the mid-to-late 2030s.
• Continued Contribution: Despite the decline, ORKAMBI will continue to contribute to Vertex's revenue stream from patients who are not eligible for or have not yet transitioned to newer therapies, or in markets where newer treatments are not yet available or reimbursed.
• Strategic Importance: While ORKAMBI's financial contribution is diminishing, it played a crucial role in establishing Vertex's leadership in CF treatment and paved the way for its more advanced therapies. Its legacy is one of significant medical advancement and market penetration in a rare disease.
• Forecasted Sales: Analyst consensus forecasts for ORKAMBI indicate a steady decline. For example, projections suggest sales falling below $1 billion annually within the next few years. Specific figures are subject to frequent revision based on market dynamics and Vertex's strategic decisions.

What Are the Key Factors Influencing ORKAMBI's Future Performance?

Several key factors will continue to influence ORKAMBI's market performance and financial returns.

• Pace of TRIKAFTA Adoption: The rate at which patients and physicians transition from ORKAMBI to TRIKAFTA is the most significant driver of ORKAMBI's revenue decline.
• Lifecycle Management and Patent Litigation: Vertex's strategy in defending its remaining patents and managing the lifecycle of ORKAMBI will impact the timing and extent of generic competition.
• Global Market Access and Reimbursement Policies: Evolving payer policies, healthcare budget constraints, and regulatory approvals in emerging markets will affect ORKAMBI's accessibility and sales volume.
• Patient Advocacy and Support Programs: The continued efforts of patient advocacy groups in securing access and affordability will remain important, particularly for patients in regions with less robust healthcare systems.
• Development of Next-Generation Therapies: While TRIKAFTA represents a significant advancement, the ongoing research into even more effective CF treatments could further marginalize older therapies like ORKAMBI.

Key Takeaways

• ORKAMBI's market exclusivity is governed by a portfolio of patents on lumacaftor, ivacaftor, and their combination, with extensions providing protection into the mid-2030s.
• The drug is a mature product with $1.30 billion in 2022 revenue, but its sales are declining due to the success of Vertex's own next-generation therapies, particularly TRIKAFTA.
• TRIKAFTA, covering approximately 90% of the CF patient population, has largely superseded ORKAMBI, leading to patient transitions and revenue erosion.
• Generic or biosimilar entry is a long-term threat, with the earliest core patent expiries in the mid-2020s, though combination patents extend further.
• ORKAMBI's future financial trajectory is characterized by continued revenue decline, with its contribution expected to significantly diminish as patent protections expire and newer treatments gain further market share.

Frequently Asked Questions

1. When will ORKAMBI face significant generic or biosimilar competition? While patents for individual components have earlier expiry dates (e.g., ivacaftor-related patents around 2025, with PTEs extending them to late 2023), patents covering the combination therapy and specific use methods extend further, into the mid-2030s. Significant generic or biosimilar competition is unlikely before the mid-to-late 2030s, depending on patent litigation outcomes and regulatory approvals.

2. What is the primary reason for ORKAMBI's declining sales? The primary driver of ORKAMBI's declining sales is the market introduction and widespread adoption of Vertex's triple-combination therapy, TRIKAFTA. TRIKAFTA offers superior efficacy and covers a broader range of CFTR mutations compared to ORKAMBI.

3. Are there any ongoing patent challenges against ORKAMBI's intellectual property? As of the most recent public filings, Vertex Pharmaceuticals actively defends its patent portfolio for its CFTR modulator therapies. While specific ongoing litigations can fluctuate, the company has historically engaged in robust defense of its intellectual property against potential challenges.

4. Will ORKAMBI remain available for patients who cannot access or tolerate TRIKAFTA? Yes, ORKAMBI is expected to remain available for patients who are not candidates for TRIKAFTA due to specific mutation profiles, contraindications, or intolerance. However, its market share within these segments will likely decrease as physicians prioritize newer therapies where appropriate.

5. What is the total addressable market for ORKAMBI, considering all eligible patients? ORKAMBI is indicated for patients with cystic fibrosis who are homozygous for the F508del mutation. Globally, this represents approximately 50% of the CF patient population, translating to tens of thousands of patients. However, the currently treated addressable market is smaller and diminishing due to the availability of TRIKAFTA.

Citations

[1] Wainwright, J. L., Redding, G. J., Konstan, M. W., Zobell, C. L., Borowitz, D., & Southern, K. W. (2016). Lumacaftor/ivacaftor in patients with cystic fibrosis homozygous for the F508del mutation: A pooled analysis of two phase 3 trials. The Journal of Cystic Fibrosis, 15(4), 486-492. doi:10.1016/j.jcf.2016.01.014

[2] Vertex Pharmaceuticals Incorporated. (2023). Vertex Pharmaceuticals Announces Fourth Quarter and Full Year 2022 Results. [Press Release]. Retrieved from https://investors.vrtx.com/news-releases/news-release-details/vertex-pharmaceuticals-announces-fourth-quarter-and-full-year-2022/

[3] Vertex Pharmaceuticals Incorporated. (2023). Vertex Pharmaceuticals Announces Third Quarter 2023 Results. [Press Release]. Retrieved from https://investors.vrtx.com/news-releases/news-release-details/vertex-pharmaceuticals-announces-third-quarter-2023-results/

[4] Vertex Pharmaceuticals Incorporated. (2024). Trikafta® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Tablets Full Prescribing Information. Retrieved from https://www.vrtx.com/sites/default/files/2024-01/trikafta-pi.pdf