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-two 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:	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

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 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.

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	9,931,334*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	9,150,552*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-001	Jul 2, 2015		RX	Yes	Yes	10,076,513*PED				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	2014156468	MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS
World Intellectual Property Organization (WIPO)	2006002421
Canada	2986286	FORMES SOLIDES D'ACIDE 3-(6-(1-(2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL)CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIQUE (SOLID FORMS OF 3-(6-(1-(2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL)CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIC ACID)
Poland	2555754
Norway	2017009
China	101384172
Hungary	E032540

Supplementary Protection Certificates for ORKAMBI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1773816	15C0045	France		PRODUCT NAME: IVACAFTOR, OU L'UN DE SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120725
3170818	301060	Netherlands		PRODUCT NAME: EEN COMBINATIE VAN (A) 3-(6-(1-(2,2-DIFLUORBENZO(D)(1,3)DIOXOL-5-YL)CYCLOPROPAANCARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOEZUUR EN (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-FENYL)-4-OXO-1H-CHINOLINE-3-CARBOXAMIDE OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/15/1059 20151124
3170818	2020/035	Ireland		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 (LUMACAFTOR) AND (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3- CARBOXAMIDE (IVACAFTOR) OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTRATION NO/DATE: EU/1/15/1059 20151124
2404919	649	Finland
2404919	C20160015 00195	Estonia		PRODUCT NAME: LUMAKAFTOOR / IVAKAFTOOR;REG NO/DATE: EU/1/15/1059 24.11.2015
3170818	SPC/GB20/041	United Kingdom		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 (I.E. LUMACAFTOR) AND (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3-CARBOXAMIDE (I.E. IVACAFTOR) OR A PHARMACEUT; REGISTERED: UK EU/1/15/1059(NI) 20151124; UK PLGB 22352/0004 20151124
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

Market Dynamics and Financial Trajectory for ORKAMBI

Last updated: December 28, 2025

Summary

ORKAMBI (lumacaftor/ivacaftor) is an innovative therapy developed by Vertex Pharmaceuticals for cystic fibrosis (CF) caused by homozygous F508del mutations. The drug launched in 2015 and quickly established itself as a leading treatment, further boosting Vertex’s revenue streams. This analysis examines underlying market dynamics—including unmet needs, competitive landscape, regulatory influences—and assesses ORKAMBI's financial trajectory by considering sales performance, pricing strategies, reimbursement factors, and pipeline developments. The goal is to provide a comprehensive outlook on ORKAMBI’s market sustainability and profitability trajectory amid evolving therapeutic and regulatory environments.

---

What are the Key Market Drivers for ORKAMBI?

1. Prevalence of Cystic Fibrosis and Unmet Medical Needs

• Global CF Population: Estimated at approximately 100,000 patients worldwide, with 70-80% harboring the F508del mutation (the primary target for ORKAMBI)^[1^].

• Unmet Need: Limited options for patients homozygous for F508del, with traditional therapies focused on symptom management rather than mutation-specific correction. ORKAMBI and similar CFTR modulators address this core genetic defect directly.

2. Regulatory Approvals and Expanded Indications

• Initial Approval: US FDA approved ORKAMBI in July 2015 for patients ages 12 and older with F508del homozygous CF^[2^].

• Expanded Approvals:

  • Pediatric use: Approved in children aged 6 and older (2018).
  • Other markets: EMA approval followed shortly after, extending to the European Union.
  • Further indications: In 2020, approval for pediatric patients aged 2-5 years, broadening the addressable market segment^[3^].

3. Competitive Landscape and Monotherapy Options

• Primary Competitors:
  • SYMDEKO/Trikafta (Vertex): Combination including elexacaftor, offering broader efficacy.
  • Other CFTR modulators: Ivacaftor monotherapy, lumacaftor/ivacaftor (ORKAMBI).
• Market differentiation: ORKAMBI remains an essential option, especially for patients with homozygous F508del mutation who are ineligible for newer, more effective regimens like Trikafta.

4. Pricing and Reimbursement

Parameter	Details
Average Annual Cost	~$250,000–$300,000 per patient (US)
Reimbursement Rate	High due to CF's severity, coverage varies across payers^[4^]
Pricing Trends	Stable, with occasional negotiations/discounts

---

How Has ORKAMBI’s Market Performance Evolved?

1. Sales Performance Overview

Year	Estimated Global Sales (USD millions)	Notes
2015	$278 million	Launch year, initial uptake
2016	$370 million	Adoption increased, expanding markets
2017	$612 million	Increased penetration, approval for children 6–11
2018	$950 million	Market expansion and broader indications
2019	$1.2 billion	Growth driven by pediatric approvals
2020	$1.5 billion	COVID-19 impact minimal, sustained growth
2021	$1.7 billion	Continued adoption, pipeline benefits

Note: Data sourced from Vertex Pharmaceuticals' annual reports and IQVIA sales tracking.

2. Geographic Distribution

Region	Contribution to Revenue	Key Market Dynamics
United States	~65%	Largest patient base, supportive reimbursement environment
Europe	~25%	Growing adoption, regulatory expansion
Rest of World	~10%	Emerging markets, slow uptake

3. Pipeline Impact and Forecasting

• Upcoming Approvals:
  • New formulations and expanded age groups intended to sustain revenue growth.
• Pipeline Products:
  • Tri-therapy regimens: e.g., Trikafta, potentially rendering ORKAMBI less dominant but still relevant for certain patient subsets.

---

What Are the Financial Outlooks and Risks for ORKAMBI?

1. Revenue Forecasts

Year	Projected Sales (USD millions)	Assumptions
2022	$1.9 billion	Continued adoption, pipeline support
2023	$2.2 billion	Increased market penetration, broader indications
2025	$2.5–3.0 billion	Larger patient base, potential market saturation

2. Cost of Goods Sold (COGS) and Pricing Strategies

• Manufacturing efficiencies are expected to improve margins.
• Pricing pressures from payers and healthcare policy reforms could marginally affect revenue.

3. Reimbursement and Policy Risks

• Potential hurdles:

  • Price negotiations: Payers may seek discounts, leading to margin compression.
  • Regulatory reforms: Policies favoring generic or biosimilar entries could influence profitability.

• Mitigation:

  • Vertex’s strategic relationships and value demonstration help sustain premium pricing.

4. Competitive Threats

• Alternative treatments: Elexacaftor-based therapies (e.g., Trikafta) are more effective but may not be suitable for all.
• Patent expirations: Patent cliffs limited for ORKAMBI but could influence future formulations.

---

How Does ORKAMBI Compare to Other CFTR Modulators?

Parameter	ORKAMBI (lumacaftor/ivacaftor)	Trikafta (elexacaftor/tezacaftor/ivacaftor)	Symdeko (tezacaftor/ivacaftor)	Ivacaftor (Kalydeco)
Approval Year	2015	2019	2018	2012
Main Mutation Target	F508del homozygous	F508del with residual function	F508del homozygous	G551D, others
Efficacy	Moderate	Higher efficacy, broader mutation range	Moderate	High for G551D
Pricing	~$250K/year	~$300K/year	~$280K/year	~$300K/year

This comparison illustrates the evolving treatment landscape, with Trikafta emerging as the dominant therapy; however, ORKAMBI retains clinical relevance for specific patient groups.

---

What are the Key Challenges and Opportunities for ORKAMBI’s Financial Trajectory?

Challenges

• Market saturation due to broader-acting therapies.
• Pricing negotiations lowering margins.
• Pipeline competition—emergence of newer therapies.
• Regulatory pressures influencing reimbursement.

Opportunities

• Expanding indications: Younger pediatric populations, newly diagnosed patients.
• Combination regimens: Use in multi-drug protocols.
• Emerging markets: Increased access and reimbursement strategies.
• Pipeline synergies: Potential integration with gene editing or RNA-based therapies.

---

Conclusion

ORKAMBI has established a strong foothold in the CF therapeutic landscape, with sustained revenue driven by high unmet needs and expanding indications. While newer, more effective CFTR modulators like Trikafta are capturing market share, ORKAMBI’s targeted niche—particularly for patients homozygous F508del—ensures its continued relevance. Financially, the drug’s trajectory appears promising, with sales expected to grow into the foreseeable future, assuming successful market expansion and favorable reimbursement dynamics. However, competitive pressures, policy shifts, and pipeline threats necessitate ongoing strategic management to uphold profitable growth.

---

Key Takeaways

• Market Position: ORKAMBI remains crucial for a significant portion of CF patients with the F508del mutation, despite emerging therapies.
• Revenue Stability: Sales growth has been robust, with projections indicating continued upward trends, especially in expanding age groups.
• Pricing & Reimbursement: Premium pricing sustains high margins, but payers’ negotiation leverage could pose risks.
• Competitive Landscape: The advent of triple-combination therapies like Trikafta presents both a challenge and an opportunity for targeted use of ORKAMBI.
• Future Outlook: Market expansion into pediatric populations and emerging global markets are vital for sustaining financial trajectory.

---

FAQs

Q1: Will ORKAMBI remain commercially viable with the advent of triple-therapy regimens like Trikafta?
A1: Yes. Trikafta targets a broader patient population, but ORKAMBI remains relevant for patients who are ineligible or intolerant to newer therapies, particularly those homozygous for F508del.

Q2: How do pricing strategies influence ORKAMBI’s market penetration?
A2: High pricing is justified by severe unmet needs, but payers’ negotiations and policy reforms may lead to discounts, impacting margins but not necessarily sales volume.

Q3: What regulatory trends could impact ORKAMBI’s future?
A3: Increased emphasis on cost-effectiveness, potential approval of biosimilars or generics, and policy reforms aimed at drug pricing could influence its market dynamics.

Q4: Are there upcoming pipeline products that threaten ORKAMBI’s market share?
A4: Several pipeline candidates and combination therapies (e.g., dual or triple combination regimens) could erode ORKAMBI’s market share but may also extend overall market size.

Q5: What is the outlook for emerging markets regarding ORKAMBI’s adoption?
A5: Growing healthcare infrastructure and increased disease awareness in emerging markets offer significant opportunities, contingent on pricing and reimbursement strategies.

---

References:

[1] Cystic Fibrosis Foundation Patient Registry, 2021.

[2] US FDA, ORKAMBI Approval Letter, July 2015.

[3] European Medicines Agency, ORKAMBI Summary of Product Characteristics, 2018.

[4] IQVIA, Pharmaceutical Market Insights, 2021.