Ivacaftor; lumacaftor - Generic Drug Details

What are the generic sources for ivacaftor; lumacaftor and what is the scope of patent protection?

Ivacaftor; lumacaftor is the generic ingredient in one branded drug marketed by Vertex Pharms Inc and is included in two NDAs. There are twenty-two patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Ivacaftor; lumacaftor has four hundred and sixty-six patent family members in thirty-six countries.

One supplier is listed for this compound.

Summary for ivacaftor; lumacaftor

International Patents:	466
US Patents:	22
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Clinical Trials:	36
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ivacaftor; lumacaftor
DailyMed Link:	ivacaftor; lumacaftor at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for ivacaftor; lumacaftor

Dosage:

GRANULE;ORAL

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 ivacaftor; lumacaftor

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 ivacaftor; lumacaftor clinical trials

Pharmacology for ivacaftor; lumacaftor

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

Anatomical Therapeutic Chemical (ATC) Classes for ivacaftor; lumacaftor

R07AX	Other respiratory system products
R07A	OTHER RESPIRATORY SYSTEM PRODUCTS
R07	OTHER RESPIRATORY SYSTEM PRODUCTS
R	Respiratory system

R07AX	Other respiratory system products
R07A	OTHER RESPIRATORY SYSTEM PRODUCTS
R07	OTHER RESPIRATORY SYSTEM PRODUCTS
R	Respiratory system

US Patents and Regulatory Information for ivacaftor; lumacaftor

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	211358-001	Aug 7, 2018		RX	Yes	No
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-002	Sep 28, 2016		RX	Yes	No					Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-003	Sep 2, 2022		RX	Yes	No					Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-001	Jul 2, 2015		RX	Yes	Yes					Y

EU/EMA Drug Approvals for ivacaftor; lumacaftor

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 ivacaftor; lumacaftor

Country	Patent Number	Title	Estimated Expiration
Canada	2736545	UNITES POSOLOGIQUES D'ACIDE 3-(6-(1-(2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL)CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIQUE (DOSAGE UNITS OF 3-(6-(1-(2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL)CYCLOPROPAANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIC ACID)
Israel	180224	MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS AND PHARMACEUTICAL COMPOSITIONS CONTAINING THE SAME
Russian Federation	2525115	СПОСОБ МОДУЛЯЦИИ ТРАНСПОРТЕРОВ АТФ-СВЯЗЫВАЮЩЕЙ КАССЕТЫ (METHOD OF MODULATING TRANSPORTERS OF ATP-BINDING CASSETTE)
South Korea	20120061875

Supplementary Protection Certificates for ivacaftor; lumacaftor

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2404919	C 2016 015	Romania		PRODUCT NAME: LUMACAFTOR SAU O SARE ACCEPTABILA FARMACEUTIC A ACESTUIA, SAU UN PROMEDICAMENT ESTER AL ACESTUIA; NATIONAL AUTHORISATION NUMBER: EU/1/12/1059/001-002; DATE OF NATIONAL AUTHORISATION: 20151119; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/12/1059/001-002; DATE OF FIRST AUTHORISATION IN EEA: 20151119
1773816	15C0045	France		PRODUCT NAME: IVACAFTOR, OU L'UN DE SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120725
2404919	PA2016015,C2404919	Lithuania		PRODUCT NAME: 3-(6-((1-(2,2-DIFLUOR-1,3-BENZODIOKSOL-5-IL)CIKLOPROPANKARBONIL)AMINO)-3-METILPIRIDIN-2-IL)BENZOINE RUGSTIS ARBA JOS FARMACINIU POZIURIU PRIIMTINA DRUSKA ARBA ESTERIS; REGISTRATION NO/DATE: EU/1/15/1059 20151119
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

Market Dynamics and Financial Trajectory for the Pharmaceutical Drugs: Ivacaftor and Lumacaftor

Last updated: July 30, 2025

Introduction

The advent of targeted therapies in cystic fibrosis (CF) treatment has revolutionized patient outcomes, with Ivacaftor and Lumacaftor emerging as pivotal medications. Both drugs, developed by Vertex Pharmaceuticals, target specific genetic mutations responsible for CF. As their market penetration deepens, understanding the evolving market dynamics and financial trajectories of these drugs is critical for stakeholders, including pharmaceutical companies, investors, healthcare providers, and policymakers.

Overview of Ivacaftor and Lumacaftor

Ivacaftor (Kalydeco) was approved by the FDA in 2012 as the first CFTR modulator effective for specific gating mutations in CFTR proteins, such as G551D. It enhances chloride transport across the CFTR channels, restoring some function disrupted by CF-causing mutations.

Lumacaftor (Orkambi), approved in 2015, synergizes with Ivacaftor to treat patients homozygous for the F508del mutation, the most prevalent CF mutation. Lumacaftor functions as a corrector, aiding proper folding and trafficking of CFTR proteins, thereby increasing their presence at the cell surface.

Market Dynamics

1. Increasing Adoption Driven by Genetic Precision Medicine

The global CF population, estimated at approximately 100,000 patients primarily in North America, Europe, and some select regions of Asia, is a key driver of the market. The therapies' efficacy in genetically defined subgroups fosters a tailor-made approach, bolstering market expansion.

Regulatory approvals extend the access scope—after initial approvals in highly developed markets, SFDA (China), EMA, and other agencies have approved these drugs, driving adoption in emerging markets. The ongoing expansion of genetic testing capabilities enhances diagnostic precision, enabling more patients to qualify for mutation-specific therapies, thus fueling demand.

2. Market Penetration and Competition

Despite being pioneers, Ivacaftor and Lumacaftor face increasing competition from newer CFTR modulators. Trikafta (elexacaftor/tezacaftor/ivacaftor), approved in 2019, has demonstrated significant efficacy across a broader mutation spectrum, including F508del, eroding market share for earlier-generation treatments like Lumacaftor.

However, in some markets, Ivacaftor maintains a strong foothold among patients with gating mutations, and Lumacaftor remains relevant for specific genetic profiles. The gradual shift toward combination therapies necessitates pharmaceutical companies to innovate, yet also promises lucrative revenues for existing drugs due to clinical inertia and existing physician familiarity.

3. Pricing and Reimbursement Landscape

Pricing for Ivacaftor and Lumacaftor remains high—approximately $300,000 annually per patient—reflecting the high R&D costs and the orphan drug status of CF medications (per the FDA). Reimbursement policies, notably in the U.S. through payers like CMS and private insurers, impact market access.

Pricing negotiations and the implementation of value-based reimbursement models impose pressure on pharmaceutical margins but also incentivize differentiation based on clinical outcomes, which can sustain revenue streams.

4. Patent Expiry and Biosimilar Threats

Patent expirations influence long-term financial trajectories. Although both drugs are protected by patents, respective patent cliffs could lead to biosimilar competition, particularly if biosimilar versions gain regulatory approval in pivotal markets. This could compress market share and pricing.

However, current patent protections and market exclusivity, bolstered by regulatory data exclusivity and orphan drug protections, delay biosimilar entry, allowing sustained revenues for the incumbent drugs.

Financial Trajectory

1. Revenue Performance

Recent financial reports from Vertex confirm robust sales. For instance, Ivacaftor's global sales exceeded $1.8 billion in 2022, with steady growth driven by expanded indications and market penetration. Lumacaftor, primarily in combination with Ivacaftor, contributed approximately $900 million globally in 2022, reflecting its targeted patient population.

Growth trajectories for these drugs are projected to stabilize or moderately grow in the near term, supported by pipeline expansions and new approvals, but may plateau as competition intensifies.

2. R&D Investment and Pipeline Prospects

Vertex continues investing heavily in CF pipeline development, with promising next-generation CFTR modulators like Elexacaftor, Tezacaftor, and innovative gene therapies poised to redefine the market landscape. These developments threaten the incremental revenue gains of existing drugs but also provide new opportunities for revenue diversification.

Financial forecasts project that as newer therapies gain market dominance, sales of Ivacaftor and Lumacaftor could decline over the next 5-10 years, though residual revenues for established markets may persist due to existing patient treatments and extended orphan drug protections.

3. Impact of Market Expansion and Patient Criteria

Expanding indications for existing combinations and the potential inclusion of pediatric populations will sustain positive growth trajectories. Moreover, initiatives to increase testing and diagnosis, especially in under-diagnosed populations, will enhance market size.

Conversely, pricing pressures, reimbursement challenges, and the patent expiry timeline present financial risks, mandating strategic adaptation through pipeline diversification and geographic expansion.

Key Market Factors Affecting Financial Outcomes

• Regulatory approvals: Expanding approvals in emerging markets underpin revenue growth.
• Market competition: Emerging modulators and potential biosimilars threaten to compress revenues.
• Pricing and reimbursement policies: Reimbursement dynamics are critical in revenue realization.
• Pipeline innovations: The advent of gene therapy and next-generation modulators may redefine future financial trajectories.
• Patient access initiatives: Efforts to improve early diagnosis and treatment adherence underpin long-term revenues.

Conclusion

The market for Ivacaftor and Lumacaftor remains dynamic, characterized by significant therapeutic success and evolving competitive landscapes. While current revenues remain robust, impending patent expiries and the advent of novel therapies necessitate strategic innovation and diversification for sustaining financial growth.

Stakeholders should monitor regulatory developments, demographic shifts, and pipelines of next-generation modulators to optimize investment and market strategies. The coming decade promises considerable transformation, aligning clinical advancements with financial opportunities in a highly specialized niche.

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Key Takeaways

• The initial market for Ivacaftor and Lumacaftor has been driven by advances in precision medicine and genetic testing.
• Competition from newer CFTR modulators, especially Trikafta, is reshaping market shares.
• High drug prices and reimbursement policies are pivotal in revenue generation but face ongoing scrutiny.
• Patent protection delays biosimilar entry, sustaining revenues; however, patent expiries could lead to compression.
• Continuous pipeline development and geographic expansion are essential for long-term financial sustainability.

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FAQs

1. How do Ivacaftor and Lumacaftor differ in their mechanism of action?
Ivacaftor acts as a potentiator, enhancing the gating function of CFTR channels at the cell surface. Lumacaftor is a corrector that helps misfolded CFTR proteins reach the cell surface. Combined, they target different defects caused by F508del mutations, improving chloride transport.

2. What is the impact of emerging therapies like Trikafta on the market?
Trikafta, which includes an additional corrector, has demonstrated superior efficacy across multiple CF mutations and has begun to dominate the market, reducing the sales share of Ivacaftor and Lumacaftor for certain indications.

3. Are there safety concerns associated with combination therapies?
Clinical trials have shown that Ivacaftor/Lumacaftor are generally well-tolerated, though potential side effects include respiratory events and elevated liver enzymes. Ongoing pharmacovigilance ensures safety profiles remain acceptable.

4. How do patent protections influence the financial stability of these drugs?
Patent protections confer market exclusivity, allowing sustained pricing and revenue streams. Future patent expiries could introduce biosimilar competition, exerting price pressure and reducing profitability.

5. What legislative or policy developments could affect the future market trajectory?
Changes in drug pricing regulations, patent laws, and reimbursement policies, especially in emerging markets, will influence market access and revenue. Uptake of value-based care models may also impact pricing strategies.

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References

[1] U.S. Food and Drug Administration. (2012). Kalydeco (Ivacaftor) Approval.
[2] European Medicines Agency. (2015). Orkambi (Lumacaftor/ivacaftor) approval.
[3] Vertex Pharmaceuticals. (2022). Annual Revenue Reports.
[4] Cystic Fibrosis Foundation. (2022). Patient Population and Market Analysis.
[5] MarketWatch. (2023). CFTR Modulator Market Outlook and Trends.