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 March 20, 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

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 North Carolina	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.

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

When does loss-of-exclusivity occur for ORKAMBI?

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

Hong Kong

Hungary

Israel

Japan

Lithuania

Mexico

New Zealand

Norway

Poland

Portugal

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 ORKAMBI around the world.

Country	Patent Number	Title	Estimated Expiration
Finland	2464337
World Intellectual Property Organization (WIPO)	2010019239
Japan	6227741
Japan	2015096539	Ｎ−［２，４−ビス（１，１−ジメチルエチル）−５−ヒドロキシフェニル］−１，４−ジヒドロ−４−オキソキノリン−３−カルボキサミドの固体形態 (SOLID FORMS OF N-[2,4-BIS(1,1-DIMETHYLETHYL)-5-HYDROXYPHENYL]-1,4-DIHYDRO-4-OXOQUINOLINE-3-CARBOXAMIDE)
Spain	2993060
South Korea	20130042034
Eurasian Patent Organization	201070698

Supplementary Protection Certificates for ORKAMBI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
1773816	C 2015 027	Romania		PRODUCT NAME: IVACAFTOR SAU O SARE ACCEPTABILA FARMACEUTIC A ACESTUIAN-(5-HIDROXI-2,4-DITERT-BUTIL-FENIL)-4-OXO-1H-CHINOLIN-3-CARBOXAMIDA SAU O SARE ACCEPTABILA FARMACEUTIC AACESTUIA; NATIONAL AUTHORISATION NUMBER: EU/1/12/782/001, EU/1/12/782/002; DATE OF NATIONAL AUTHORISATION: 20120723; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/12/782/001, EU/1/12/782/002; DATE OF FIRST AUTHORISATION IN EEA: 20120723
2404919	649	Finland
1773816	PA2015028	Lithuania		PRODUCT NAME: IVACAFTORUM; REGISTRATION NO/DATE: EU/1/12/782/001 - EU/1/12/782/002 20120723
1773816	201540032	Slovenia		PRODUCT NAME: IVACAFTOR; NATIONAL AUTHORISATION NUMBER: EU/1/12/782/001-002; DATE OF NATIONAL AUTHORISATION: 20120723; AUTHORITY FOR NATIONAL AUTHORISATION: EU
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	132016000022519	Italy		PRODUCT NAME: IVACAFTOR O UN SUO SALE FARMACEUTICAMENTE ACCETTABILE(KALYDECO); AUTHORISATION NUMBER(S) AND DATE(S): EU/12/782/001-002, 20120725

ORKAMBI: Market Dynamics and Financial Trajectory

Last updated: May 1, 2026

What is ORKAMBI’s market basis and why does it move revenue?

ORKAMBI (lumacaftor/ivacaftor) is a CF (cystic fibrosis) therapy positioned for patients with specific CFTR genotypes who meet label criteria. ORKAMBI’s revenue trajectory is dominated by three demand drivers:

1. Genotype eligibility

   • ORKAMBI’s addressable population is constrained to CF patients with the relevant CFTR mutation(s) and meeting label criteria.
   • Any downstream label narrowing or guideline shifts that prioritize newer modulator regimens reduces the conversion funnel for ORKAMBI.

2. Competitive displacement by next-generation CFTR modulators

   • In CF therapeutics, uptake moves toward regimens with improved efficacy, broader genotype coverage, or improved adherence (dose complexity and side-effect profiles).
   • As newer therapies gain share in both treatment-naïve and switch markets, ORKAMBI faces margin pressure from reduced units and net-price deterioration.

3. Formulary and payer routing

   • CF drug procurement is typically payer-driven with prior authorization and step edits.
   • When formularies add competing CFTR modulators, ORKAMBI’s net price and refill rates trend down, even if list price remains stable.

These factors determine the revenue shape: high initial penetration followed by share erosion and eventual plateauing as the market transitions to newer CFTR regimens.

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How has ORKAMBI’s financial trajectory evolved?

ORKAMBI’s financial profile is best described as declining and aging in a category that experienced successive launches of more optimized CFTR modulators.

Revenue trajectory signals (public company reporting)

• Vertex Pharmaceuticals is the primary commercial source for ORKAMBI reporting in public filings, with CFTR modulators as a major franchise.
• Over time, ORKAMBI competes against Vertex and non-Vertex CF therapies, with revenue share shifting toward newer products and off-label or guideline-favored regimens.

Net effect on ORKAMBI:

• Units trend down as eligible patients are increasingly routed to preferred options.
• Net revenue trends down faster than list price due to payer contracting, rebates, and patient switching.
• Geographic and payer variability creates periods where ORKAMBI holds up locally, but the broader franchise pattern is contraction.

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What does competitive dynamics look like in the CFTR modulator market?

The CFTR modulator market is characterized by replacement cycles. Each new regimen typically changes:

• which genotypes are targeted,
• whether initiation criteria broaden,
• dosing convenience,
• tolerability management.

That means ORKAMBI’s share is not defended by incremental improvements once a newer option becomes the default for genotype-eligible patients.

Competitive vectors that typically compress ORKAMBI’s market

• Clinical improvements that shift standard of care and payer preference
• Broader genotype coverage that expands addressable pool for newer drugs while ORKAMBI remains narrower
• Simplified regimens that improve adherence and reduce discontinuation
• Positioning that discourages switches to older combinations unless required by specific constraints

This is consistent with the real-world shift in CF modulator standards following the entry of multiple next-generation therapies over the last decade.

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Where does ORKAMBI still sell: geographies, access, and payer mechanics?

Ongoing sales tend to persist in:

• patients already established on ORKAMBI where switching barriers exist,
• regions with slower formulary updates or contract renegotiations,
• payers with narrower preferred drug lists due to managed care design.

The revenue ceiling still depends on:

• continued eligibility and treatment persistence,
• ability to maintain net pricing through contracts,
• and substitution risk when formularies update.

In CF, switching is common once a preferred option becomes available, but it is not immediate or uniform across all payer segments.

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What is the financial trajectory risk from lifecycle events (patent, exclusivity, competition)?

For legacy combination modulators like ORKAMBI, lifecycle risk generally comes from:

• loss of exclusivity (depending on compound-specific patent and regulatory exclusivity timelines in each jurisdiction),
• erosion from standard-of-care shift regardless of patent status,
• compressed pricing power when competing regimens dominate treatment decisions.

Because ORKAMBI is a product of combination CFTR modulation, the competitive landscape can shift quickly even without immediate generic entry, through payer preference and clinician switching.

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How should investors and R&D leaders interpret ORKAMBI’s market signals?

Business interpretation of ORKAMBI’s trajectory

• The product is in a decline phase shaped primarily by therapeutic displacement rather than by sudden price collapse from generic entry (unless and until exclusivity expires and generic competition materializes).
• The most actionable indicator of future revenue is not list price but payer routing and persistence among genotype-eligible patients.

Implications

• For R&D: ORKAMBI highlights that CF modulator franchises are subject to rapid regimen substitution, making late-line incremental differentiation less valuable unless it expands eligible populations or simplifies treatment substantially.
• For investment: ORKAMBI’s value is tied to how quickly guidelines and formularies re-route patients; once preferred alternatives dominate, revenue decays in a structurally predictable way.

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Market dynamics summary

Driver	Directional impact on ORKAMBI revenue	Mechanism
Genotype eligibility constraints	Downward over time	Narrow addressable pool and treatment substitution reduces conversions
Competitive displacement by newer modulators	Downward	Standard-of-care rerouting to preferred regimens
Payer formulary and prior auth	Downward	Higher barriers for non-preferred products; net price compression
Patient persistence among established users	Mixed	Some retention exists, but gradually declines as switching increases
Lifecycle events (exclusivity/patent)	Downward	Reduced pricing power; potential generic pressure after exclusivity end

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Key Takeaways

• ORKAMBI’s commercial performance is driven by limited genotype eligibility and ongoing displacement by newer CFTR modulators that shift standard of care and payer routing.
• The product’s financial trajectory trends downward as the market moves toward preferred regimens, with net revenue decay typically faster than list price stability.
• The primary forward-looking indicators are formulary preference, patient persistence, and switching rates among genotype-eligible populations, not incremental pricing actions.

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FAQs

1) Is ORKAMBI’s revenue mainly affected by competition or by exclusivity expiry?

Competition and treatment switching dominate the earlier decline phase because payer routing and guideline preference drive displacement even before exclusivity loss.

2) What determines ORKAMBI’s addressable patient population?

Label-defined CFTR genotype eligibility and label criteria determine who can be prescribed ORKAMBI, constraining long-term demand.

3) Why can ORKAMBI’s net revenue fall even if list price stays stable?

Payer contracting, rebates, and net-price adjustments usually intensify as the product becomes non-preferred.

4) What market signal best predicts ORKAMBI’s next phase of decline?

Formulary decisions that add newer CFTR modulators as preferred options and step edits that restrict ORKAMBI.

5) Does ORKAMBI retain value through patient persistence?

Yes, established patients can create short-term revenue stickiness, but persistence typically declines as switching becomes the default over time.

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References (APA)

[1] Vertex Pharmaceuticals. (n.d.). Investor relations and annual reports (product franchise disclosures for CFTR modulators). Vertex Pharmaceuticals.
[2] U.S. Food and Drug Administration. (n.d.). ORKAMBI (lumacaftor and ivacaftor) prescribing information. FDA.