ORKAMBI Drug Patent Profile

When do Orkambi patents expire, 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 ten 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
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ORKAMBI

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 8,507,534.

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
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No

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
Argentina	081069
Argentina	081760
Australia	2011237494
Australia	2011237601
Brazil	112012026255
Brazil	112012026257
Canada	2795748

Supplementary Protection Certificates for ORKAMBI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3170818	LUC00172	Luxembourg		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; AUTHORISATION NUMBER AND DATE: EU/1/15/1059 20151124
2404919	SPC/GB16/026	United Kingdom		PRODUCT NAME: LUMACAFTOR, OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, OR AN ESTER PRODRUG THEREOF; REGISTERED: UK EU/1/15/1059/001 20151124; UK EU/1/15/1059/002 20151124
3170818	PA2020525,C3170818	Lithuania		PRODUCT NAME: (A) 3-(6-(1-(2,2-DIFLUORBENZO(D)(1,3)DIOKSOL-5-IL)CIKLOPROPANKARBOKSAMIDO)-3-METILPIRIDIN-2-IL)BENZENKARBOKSIRUGSTIES IR (B) N-(5-HIDROKSI-2,4-DITERT-BUTILFENIL)-4-OKSO-1H-CHINOLIN-3-KARBOKSAMIDO ARBA FARMACINIU POZIURIU PRIIMTINOS N-(5-HIDROKSI-2,4-DITERT-BUTIL-FENIL)-4-OKSO-1H-CHINOLIN-3-KARBOKSAMIDO DRUSKOS DERINYS; REGISTRATION NO/DATE: EU/1/15/1059 20151119
3170818	PA2020525	Lithuania		PRODUCT NAME: (A) 3-(6-(1-(2,2-DIFLUORBENZO(D)(1,3)DIOKSOL-5-IL)CIKLOPROPANKARBOKSAMIDO)-3-METILPIRIDIN-2-IL)BENZENKARBOKSIRUGSTIES IR (B) N-(5-HIDROKSI-2,4-DITERT-BUTIL-FENIL)-4-OKSO-1H-CHINOLIN-3-KARBOKSAMIDO ARBA JO FARMACINIU POZIURIU PRIIMTINOS DRUSKOS DERINYS; REGISTRATION NO/DATE: EU/1/15/1059 20151119
2404919	C20160015 00195	Estonia		PRODUCT NAME: LUMAKAFTOOR / IVAKAFTOOR;REG NO/DATE: EU/1/15/1059 24.11.2015
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
1773816	300748	Netherlands		PRODUCT NAME: IVACAFTOR, OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120725

ORKAMBI (lumacaftor/ivacaftor): Investment Scenario and Fundamentals

Last updated: April 24, 2026

What is ORKAMBI and where does it sit in the cystic fibrosis landscape?

ORKAMBI is a fixed-dose combination of lumacaftor (CFTR corrector) and ivacaftor (CFTR potentiator) for treatment of cystic fibrosis (CF) in patients aged 2 years and older who have two copies of the F508del mutation. It targets the most common CF genotype and has been a key commercial bridge after earlier CFTR modulator entries.

Core label positioning

• Indication (US): CF in patients aged 2 years and older with two F508del mutations.
• Mechanistic class: CFTR corrector + potentiator.
• Form: Oral combination therapy (brand-manufactured product).

Market context

• CFTR modulator competition accelerated after ORKAMBI’s launch, shifting market share toward newer regimens that address broader mutation sets and in many cases improved clinical profiles. ORKAMBI remains a major therapy for the F508del/F508del subgroup, but long-term growth depends on maintaining access, pricing, and adherence versus newer F508del-targeting combinations.

What do the fundamentals say about efficacy differentiation and patient selection?

Clinical differentiation in CF is measured through lung function outcomes (FEV1), pulmonary exacerbations, and safety/tolerability. For ORKAMBI, patient selection is mutation-restrictive, which tightens the addressable population to F508del homozygotes (and overlaps with payer eligibility criteria).

Efficacy and endpoints used in modulator value cases

• Baseline lung function improvement versus placebo in F508del homozygotes.
• Reduction in pulmonary exacerbations and stabilization of respiratory parameters.
• Benefits and tradeoffs tied to dose adherence and adverse event management.

Patient selection and real-world utilization constraints

• Eligibility anchored to genotype testing and confirmed F508del/F508del status.
• Use often constrained by:
  • age-specific labeling boundaries in each jurisdiction,
  • payer step edits and prior authorization,
  • tolerance and adherence driven by safety profile.

How is ORKAMBI performing as a cash-flow asset versus newer CFTR regimens?

From an investment lens, ORKAMBI behaves like a mature CFTR modulator: earlier mover advantages in F508del homozygotes, followed by margin pressure from newer entrants and label expansions elsewhere in the CF modulators ecosystem.

Commercial reality

• ORKAMBI’s addressable segment shrinks at the margin as newer regimens capture patients who would have been candidates historically.
• The therapy’s durability depends on whether payers continue to cover it as a standard-of-care for F508del/F508del, and whether prescribers keep it in treatment algorithms.

Investment takeaway

• Upside is primarily operational: pricing, contracting, retention in F508del/F508del, and channel management.
• Downside is structural: treatment substitution by newer modulator standards and narrowing of formularies.

What are the patent and exclusivity levers that matter for valuation?

For valuation, investors typically focus on: 1) patent term survival by key jurisdictions, 2) exclusivity (where applicable), 3) strength of line-of-therapy barriers in formularies.

ORKAMBI is a combination product. Patent landscapes for combinations tend to depend on:

• composition claims and formulation claims,
• method-of-use claims tied to mutation-defined treatment,
• specific dosing regimens and therapeutic effects.

However, this analysis does not provide jurisdiction-by-jurisdiction expiration, ownership, or exclusivity expiry dates beyond what is included in the cited regulatory and product documentation. Without explicit expiration dates and legal status data for the relevant claims, the only defensible conclusion is at a high level: the valuation case hinges on regulatory/market exclusivity and patent barrier strength in major markets.

What are the regulatory fundamentals and label risk factors investors monitor?

Regulatory label anchors (US)

• Indication: CF, age 2+, with two F508del mutations.
• Regulatory product documentation: ORKAMBI is registered with dosing and safety labeling maintained by the FDA.

Key investor-relevant label dynamics

• Safety and tolerability: CFTR modulator classes carry known class considerations that can influence prescribing behavior and discontinuation rates.
• Drug-drug interactions and co-medications: CF patients often use multiple therapies; interaction management affects adherence and persistence.
• Pediatric use: labeling down to age 2 affects TAM (total addressable market) but also ties to tighter monitoring regimes.

Source anchor: FDA label information for ORKAMBI. [1]

How should investors underwrite TAM for ORKAMBI in the current CF modulator era?

A practical TAM build for ORKAMBI typically uses:

• CF prevalence in treated geographies,
• genotype distribution (F508del homozygote share),
• age band coverage (2 years and older),
• payer adoption (formulary placement),
• persistence and switching dynamics.

TAM driver hierarchy

1. Genotype eligibility: F508del/F508del share.
2. Coverage: formulary inclusion and prior authorization criteria.
3. Switching pressure: substitution by newer modulator regimens as standards evolve.
4. Persistence: adherence and adverse-event-driven discontinuations.

Base-case expectation

• Without explicit market share data in this response, the investment expectation is that ORKAMBI remains meaningful in the subset where it is a payer-accepted option and where patients remain on it due to clinical response or transition friction.

What does the competitive set imply for pricing power and gross margin?

ORKAMBI faces competition inside CFTR modulators and, over time, competition in the specific regimen selection for F508del patients.

Competitive forces investors price in

• Formulary substitution: newer regimens reduce ORKAMBI’s share if payers prefer them.
• Contracting leverage: manufacturers with broader label coverage can negotiate stronger system-level contracts.
• Net price erosion: expected from periodic payer renegotiations and volume-based contracting dynamics across mature specialty franchises.

Investment implication

• If ORKAMBI is retained in formularies as an option, it can hold pricing better than fully displaced brands.
• If it becomes a “second-line” or restricted-access option, the gross margin and revenue trajectory usually compress.

What are the downside and upside scenarios investors should model?

Base case

• Continued revenue contribution from F508del/F508del patients in geographies where ORKAMBI remains covered.
• Gradual share pressure from newer regimens, partly offset by ongoing pediatric and treatment-naïve starts where allowed.

Bear case

• Faster formulary displacement in major markets.
• Higher discontinuation rates due to tolerability dynamics and class-related adverse event management.
• Net price erosion from contract renegotiations and payer pressure.

Bull case

• ORKAMBI maintains or improves formulary access for F508del/F508del.
• Better persistence from patient support programs and improved monitoring routines.
• Contracting preserves net pricing through volume commitments.

Key deal-relevant diligence items investors should pull for ORKAMBI

Even for a brand with mature regulatory status, the cash-flow profile hinges on operational details. The diligence checklist for ORKAMBI typically includes:

• Formulary placement and access rules by geography (including step edits and prior authorization).
• Patient persistence and treatment switching rates out of ORKAMBI to alternative CFTR regimens.
• Net pricing drivers: rebates, discounts, and outcomes-based contract terms if used.
• Pediatric uptake pipeline: new starts in ages 2 to <18, subject to local label adoption and practice patterns.
• Safety pharmacovigilance that could tighten labeling or impact persistence.

What is the investment thesis in one line?

ORKAMBI is a mature CFTR modulator franchise with value anchored to F508del/F508del eligibility and formulary access, where the primary investment risk is share erosion from newer CFTR regimens and the primary upside is durable payer coverage and patient persistence in the eligible subgroup.

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Key Takeaways

• ORKAMBI is a lumacaftor/ivacaftor CFTR corrector-potentiator for CF patients aged 2+ with two F508del mutations. [1]
• The addressable market depends on genotype distribution and continued payer/formulary acceptance in F508del/F508del.
• The investment model is driven by net price, persistence, and switching pressure from newer CFTR regimens.
• Patent valuation must be based on specific claim survival and exclusivity timelines by jurisdiction, paired with the regulatory and formulary realities that determine commercial persistence.

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FAQs

1) What patient group does ORKAMBI target?

Patients with cystic fibrosis aged 2 years and older with two F508del mutations. [1]

2) Is ORKAMBI a corrector, a potentiator, or both?

It is a combination of a CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor). [1]

3) What is the main market risk for ORKAMBI?

Loss of formulary share and treatment switching as payers and clinicians favor newer CFTR regimens for overlapping patient groups.

4) What determines ORKAMBI revenue durability?

Payer access for F508del/F508del, net pricing and contracting, plus persistence versus switching.

5) What regulatory document is the primary source for label details?

The FDA prescribing information/labeling for ORKAMBI. [1]

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References

[1] U.S. Food and Drug Administration. (n.d.). ORKAMBI (lumacaftor and ivacaftor) prescribing information. FDA. https://www.accessdata.fda.gov/