Ivacaftor; lumacaftor - Generic Drug Details

What are the generic drug 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-three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

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

One supplier is listed for this compound.

Summary for ivacaftor; lumacaftor

International Patents:	461
US Patents:	23
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Clinical Trials:	37
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

Constraining patent/regulatory exclusivity:

8,507,534*PED

Dosage:

GRANULE;ORAL

Constraining patent/regulatory exclusivity:

8,507,534*PED

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
Azienda Ospedaliera Universitaria Integrata Verona	PHASE2
Qanatpharma Canada LTD	Phase 1
Children's Hospital Medical Center, Cincinnati	N/A

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	TABLET;ORAL	206038-001	Jul 2, 2015		RX	Yes	Yes	10,646,481*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No	8,754,224*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-002	Aug 7, 2018		RX	Yes	Yes	7,495,103*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No	9,931,334*PED				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
China	107501099	ATP‑结合弹夹转运蛋白的调控剂 (Modulators of atp-binding cassette transporters)
Slovenia	2225230
Canada	2627358
Japan	2013513617

Supplementary Protection Certificates for ivacaftor; lumacaftor

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
3170818	2020025	Norway		PRODUCT NAME: EN KOMBINASJON AV (A) 3-(6-; REG. NO/DATE: EU/1/15/1059 20151210
3170818	2020C/005	Belgium		PRODUCT NAME: ORKAMBI (LUMACAFTOR + IVACAFTOR); AUTHORISATION NUMBER AND DATE: EU/1/15/1059 20151124
2225230	2017009	Norway		PRODUCT NAME: 3-6-(1-(2,2-DIFLUOR-1,3-; REG. NO/DATE: EU/1/15/1059 20151210

IVACAFTOR + LUMACAFTOR: Market Dynamics and Financial Trajectory

Last updated: April 24, 2026

What product-market structure does the IVACAFTOR + LUMACAFTOR franchise follow?

Ivacaftor + lumacaftor is a CFTR modulator combination used in cystic fibrosis (CF) treatment. Commercial performance is shaped by three market forces: (1) label scope and patient eligibility, (2) competitive displacement by newer CFTR modulators, and (3) payer and contracting dynamics as CF care shifts toward more efficacious, easier regimens.

1) Where the combination fits in CFTR modulation

• Therapeutic class: CFTR modulators (ivacaftor potentiator; lumacaftor corrector).
• Clinical positioning: Historically positioned for CF patients with specific genotypes responsive to the combination, especially earlier in the evolution of triple-therapy and newer next-generation regimens.
• Competitive set (commercial displacement pressure): Newer CFTR modulators (including ivacaftor-based triple combinations and other next-generation corrector/potentiator strategies) reduce demand for older double-therapy, especially where outcomes and dosing convenience are superior.

2) Market structure by dosing and regimen migration

The drug’s financial trajectory follows the typical pattern seen in CFTR modulation:

• Early penetration: Benefit tied to genotype-defined eligibility and the absence of broader, more efficacious alternatives.
• Mid-cycle consolidation: Steadier demand under stable eligibility and payer coverage.
• Late-cycle decline: Revenue pressure as patients migrate to newer therapies with better clinical profiles and payers steer to preferred formulary options.

How do competitive forces move revenue for IVACAFTOR + LUMACAFTOR?

Revenue erosion risk is driven by formulary tiering and patient migration. Newer CFTR modulators generally win on:

• Expanded genotype coverage
• Higher efficacy measures
• Regimen simplicity (fewer daily dose events and less monitoring complexity in practice)

That combination causes three observable dynamics in the market for older CFTR therapies:

1. Patient switching: Patients move to preferred therapies when the clinical and access case improves.
2. Contracting leverage shift: Payers gain negotiating power as fewer patients remain on the older regimen.
3. Margin compression: Even when not fully displaced, older products often face net price pressure and utilization share erosion.

What is the financial trajectory pattern for older CFTR double-therapy?

A franchise like ivacaftor + lumacaftor generally exhibits:

• Peak and plateau early in the lifecycle when eligibility is broad relative to then-available competitors.
• Gradual decline as new modulators take share in the same patient segments.
• Accelerated net sales drop once preferred formulary status shifts and migration becomes systematic (especially when clinical outcomes and payer programs align toward newer regimens).

However, the exact trajectory for the specific combination depends on:

• Current label constraints
• Active patient pool size on genotype-defined coverage
• Payer formulary placement in major markets
• Net price versus list price across contracts and rebates

What do the core demand drivers imply for IVACAFTOR + LUMACAFTOR revenue going forward?

The demand stack is narrow compared with newer CFTR options. The forward-facing drivers typically include:

1) Patient eligibility narrowing

• CFTR therapy is genotype-gated. When newer therapies cover more genotypes or offer improved outcomes across the same genotype, the older combination’s addressable population shrinks.

2) Payer steering and preferred formulary placement

• Payers prefer therapies that reduce total cost per patient-year through improved outcomes and reduced downstream utilization (hospitalizations, exacerbations). As alternatives clear evidence thresholds, utilization shifts.

3) Sequence-of-therapy effects

• Newer regimens often become the default for new starts, leaving the older combination to serve a smaller remaining cohort on existing coverage or those with access barriers.

How do manufacturing and supply scale considerations affect the commercial curve?

Older branded CFTR modulators can face structural commercial constraints:

• Lower throughput justification due to declining volumes.
• Inventory and forecasting accuracy becomes more difficult as utilization drops unpredictably with formulary changes.
• Cost-to-serve and contracting can pressure margins even where gross pricing is maintained.

These factors usually do not reverse the demand trend; they add friction to maintaining stable revenue after competitive displacement accelerates.

What financial outlook does the competitive landscape indicate for near- to mid-term performance?

Near- to mid-term performance is typically characterized by:

• Declining utilization share versus newer modulators.
• Net sales stabilization only if the remaining eligible population stays on therapy and formulary access remains intact.
• Potential step-down events aligned with reimbursement policy updates or clinical guideline changes that encourage migration.

For investors and R&D planners, the key financial signal is whether the remaining eligible population can sustain a revenue base despite continuing share losses.

What actions do market dynamics imply for R&D and investment positioning?

Commercially, ivacaftor + lumacaftor sits in a late-stage competitive environment. The strategic implications tend to be:

• Portfolio strategy: Treat the asset as a diminishing-growth or declining-revenue franchise rather than a scalable growth engine.
• Competitive intelligence: Track formulary decisions and guideline adoption rates for newer CFTR modulators as leading indicators of cohort shrinkage.
• Development focus: For adjacent R&D, prioritize differentiation in efficacy, convenience, durability, and genotype coverage to avoid the late-cycle replacement pattern.

Key Takeaways

• Market dynamics are dominated by genotype gating and patient migration to newer CFTR modulators, which compress utilization and price.
• Financial trajectory for ivacaftor + lumacaftor typically follows a peak-then-plateau pattern into gradual decline, with sharper drops when preferred formulary status shifts.
• Forward performance depends on the remaining eligible patient pool and whether net pricing holds as contracting power shifts to newer therapies.

FAQs

1. Which category of CF therapies drives displacement of ivacaftor + lumacaftor?
   Newer CFTR modulator combinations that improve efficacy and/or expand genotype coverage.

2. What market factor most directly affects revenue for genotype-gated CF drugs?
   The size of the eligible patient pool under current label-linked genotype criteria.

3. Do payer formulary decisions usually change slower or faster than clinical adoption?
   Formulary decisions often change quickly when evidence supports migration and contracts favor newer products.

4. What is the typical shape of net sales for older branded CFTR modulators?
   Plateau followed by sustained decline as patient starts and switching move to newer regimens.

5. What should investors monitor as leading indicators?
   Preferred formulary status shifts, net pricing trends, and patient migration rates to newer CFTR modulators.

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References

[1] U.S. Food and Drug Administration. “KALYDECO (ivacaftor) Prescribing Information.”
[2] U.S. Food and Drug Administration. “ORKAMBI (lumacaftor/ivacaftor) Prescribing Information.”
[3] European Medicines Agency. “Summary of Product Characteristics: Orkambi (lumacaftor/ivacaftor).”
[4] Cystic Fibrosis Foundation. “Clinical Care Guidelines and Therapy Use Resources for CFTR Modulators.”
[5] IQVIA Institute / industry analyses on pharmaceutical market dynamics and therapy migration patterns (CFTR modulator landscape).