IVACAFTOR; IVACAFTOR, TEZACAFTOR - Generic Drug Details

What are the generic drug sources for ivacaftor; ivacaftor, tezacaftor and what is the scope of freedom to operate?

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

Ivacaftor; ivacaftor, tezacaftor has four hundred and twenty patent family members in thirty-nine countries.

One supplier is listed for this compound.

Summary for IVACAFTOR; IVACAFTOR, TEZACAFTOR

International Patents:	420
US Patents:	25
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	44
DailyMed Link:	IVACAFTOR; IVACAFTOR, TEZACAFTOR at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for IVACAFTOR; IVACAFTOR, TEZACAFTOR

Constraining patent/regulatory exclusivity:

10,206,877

Dosage:

TABLET, 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; IVACAFTOR, TEZACAFTOR

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Chiesi USA, Inc.	Phase 4
University of North Carolina, Chapel Hill	Phase 4
Emory University	Phase 4

See all IVACAFTOR; IVACAFTOR, TEZACAFTOR clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for IVACAFTOR; IVACAFTOR, TEZACAFTOR

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; IVACAFTOR, TEZACAFTOR

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	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-001	Feb 12, 2018		RX	Yes	Yes	8,754,224		Y	Y
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-001	Feb 12, 2018		RX	Yes	Yes
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-002	Jun 21, 2019		RX	Yes	No	10,206,877		Y
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-002	Jun 21, 2019		RX	Yes	No	8,415,387
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-002	Jun 21, 2019		RX	Yes	No	9,974,781		Y
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-002	Jun 21, 2019		RX	Yes	No	8,410,274		Y
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-001	Feb 12, 2018		RX	Yes	Yes	8,354,427

International Patents for IVACAFTOR; IVACAFTOR, TEZACAFTOR

Country	Patent Number	Title	Estimated Expiration
Brazil	PI0916877	composição farmacêutica e administração da mesma
Japan	2018048207	（Ｒ）−１−（２，２−ジフルオロベンゾ［ｄ］［１，３］ジオキソール−５−イル）−Ｎ−（１−（２，３−ジヒドロキシプロピル）−６−フルオロ−２−（１−ヒドロキシ−２−メチルプロパン−２−イル）−１Ｈ−インドール−５−イル）シクロプロパンカルボキサミドの固体形態 (SOLID FORM OF (R)-1-(2,2-DIFLUOROBENZO[d][1,3]DIOXOL-5-YL)-N-(1-(2,3-DIHYDROXYPROPYL)-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1H-INDOL-5-YL)CYCLOPROPANECARBOXAMIDE)
Canada	2878057	COMPOSITIONS PHARMACEUTIQUES DE (R)-1-(2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL)-N-(1-(2,3-DIHYDROXYPROPYL)-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1H-INDOL-5-YL)CYCLOPROPANE-CARBOXAMIDE ET LEUR ADMINISTRATION (PHARMACEUTICAL COMPOSITIONS OF (R)-1-(2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL)-N-(1-(2,3-DIHYDROXYPROPYL)-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1H-INDOL-5-YL) CYCLOPROPANECARBOXAMIDE AND ADMINISTRATION THEREOF)
Portugal	3131582
Mexico	366161	COMPOSICION FARMACEUTICA Y SU ADMINISTRACION. (PHARMACEUTICAL COMPOSITION AND ADMINISTRATIONS THEREOF.)
Russian Federation	2573830	ТВЕРДЫЕ ФОРМЫ (R)-1-(2,2-ДИФТОРБЕНЗО[d][1,3]ДИОКСОЛ-5-ИЛ)-N-(2,3-ДИГИДРОКСИПРОПИЛ)-6-ФТОР-2-(1-ГИДРОКСИ-2-МЕТИЛПРОПАН-2-ИЛ)-1H-ИНДОЛ-5-ИЛ)ЦИКЛОПРОПАНКАРБОКСАМИДА (SOLID FORMS OF (R)-1-(2,2-DIFLUOROBENZO[d][1,3]DIOXOL-5-YL)-N-(2,3-DIHYDROXYPROPYL)-6-FLUORO-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1H-INDOL-5-YL)CYCLOPROPANE CARBOXAMIDE)
Australia	2005258320	Modulators of ATP-Binding Cassette transporters

Supplementary Protection Certificates for IVACAFTOR; IVACAFTOR, TEZACAFTOR

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1773816	CR 2015 00038	Denmark		PRODUCT NAME: N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLIN-3-CARBOXAMID ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF; REG. NO/DATE: EU/1/12/782/001-002 20120725
1773816	C01773816/01	Switzerland		PRODUCT NAME: IVACAFTOR; REGISTRATION NO/DATE: SWISSMEDIC 62686 13.01.2014
1773816	C20150028 00162	Estonia		PRODUCT NAME: IVAKAFTOOR;REG NO/DATE: EU/1/12/782/001-002 25.07.2012
2826776	PA2021508	Lithuania		PRODUCT NAME: (A) (R)-1-(2,2-DIFLUORBENZO(D)(1,3)DIOKSOL-5-IL)-N-(1-(2,3-DIHIDROKSIPROPIL)-6-FLUOR-2-(1-HIDROKSI-2-METILPROPAN-2-IL)-1H-INDOL-5-IL) CIKLOPROPANKARBOKSAMIDO ARBA JO FARMACISKAI PRIIMTINOS DRUSKOS IR (B) N-(5-HIDROKSI-2,4-DITERT-BUTIL-FENIL)-4-OKSO-1H-CHINOLIN-3-KARBOKSAMIDO ARBA JO FARMACISKAI PRIIMTINOS DRUSKOS DERINYS; REGISTRATION NO/DATE: EU/1/18/1306 20181031
1773816	35/2015	Austria		PRODUCT NAME: N-(5-HYDROXY-2,4DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLIN-3-CARBOXAMID ODER EIN PHARMAZEUTISCH ANNEHMBARES SALZ DAVON; REGISTRATION NO/DATE: EU/1/12/782/001, EU/1/12/782/002 (MITTEILUNG) 20120725
2826776	C02826776/01	Switzerland		PRODUCT NAME: TEZACAFTOR UND IVACAFTOR; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 66742 10.05.2019
2826776	301105	Netherlands		PRODUCT NAME: EEN COMBINATIE VAN (A) (R)-1-(2,2-DIFLUOROBENZO(D)(1,3)DIOXOL-5-YL)-N-(1-(2,3-DIHYDROXYPROPYL)-6-FLUOR-2-(1-HYDROXY-2-METHYLPROPAN-2-YL)-1H-INDOL-5-YL)CYCLOPROPAANCARBOXAMIDE OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN 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/1/18/1306 20181106

IVACAFTOR; IVACAFTOR, TEZACAFTOR Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory of Ivacaftor and Ivacaftor, Tezacaftor, Elexacaftor

Introduction

Ivacaftor, along with its combination therapies such as ivacaftor, tezacaftor, and elexacaftor (marketed as Trikafta or Kaftrio), has revolutionized the treatment of cystic fibrosis (CF), a genetic disorder that affects the lungs, pancreas, and other organs. This article delves into the market dynamics and financial trajectory of these drugs, highlighting their impact, growth prospects, and economic implications.

Market Growth and Outlook

The market for ivacaftor and its combination therapies has witnessed significant growth and is poised for continued expansion. From 2023 to 2031, the ivacaftor reagent market is expected to see substantial and sustained growth, driven by emerging trends and pivotal factors steering market dynamics[1].

Drivers of Market Growth

Several factors drive the growth of this market:

Increasing Prevalence of Cystic Fibrosis

The rising prevalence of cystic fibrosis and the need for effective treatments are key drivers. CF affects thousands of people worldwide, and the demand for therapies that can improve lung function and quality of life is high.

Advancements in CFTR Modulators

Ivacaftor, tezacaftor, and elexacaftor are part of a class of drugs known as CFTR modulators, which target the underlying cause of CF by improving the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. These advancements have significantly improved treatment outcomes, driving market growth.

Regulatory Approvals

Regulatory approvals in various regions, including the U.S., Europe, and other countries, have expanded the market reach of these drugs. For instance, the approval of Trikafta in the U.S. and its equivalent, Kaftrio, in Europe has been a major boost[4].

Financial Performance

The financial performance of Vertex Pharmaceuticals, the company behind these drugs, reflects their market success.

Revenue Growth

In 2023, Vertex reported a full-year product revenue of $9.87 billion, an 11% increase compared to 2022. The revenue from Trikafta/Kaftrio was particularly significant, reaching $8,944.7 million for the full year 2023, up from $7,686.8 million in 2022[5].

Product Revenue Breakdown

• Trikafta/Kaftrio: This combination therapy accounted for the bulk of the revenue, with $2,333.3 million in the fourth quarter of 2023 alone.
• Other CF Products: While other CF products contributed to the revenue, Trikafta/Kaftrio remains the flagship product driving financial growth[5].

Cost-Effectiveness and Reimbursement

Despite the high cost of these treatments, they have been shown to be cost-effective in certain contexts.

Cost of Treatment

The treatment with Trikafta is expensive, costing approximately $306,600 per patient per year. However, studies have shown that it significantly improves lung function, reduces pulmonary exacerbations, and enhances health-related quality of life[4].

Reimbursement Recommendations

Healthcare authorities like CADTH in Canada have recommended reimbursement for Trikafta under certain conditions. These conditions include demonstrating improvements in lung function and a reduction in the number of days requiring antibiotics for pulmonary exacerbations[4].

Clinical Efficacy

The clinical efficacy of ivacaftor and its combination therapies is well-documented.

Improvement in Lung Function

Studies have shown that switching to Trikafta after initial treatment with other CFTR modulators results in statistically significant and clinically meaningful improvements in lung function, as measured by ppFEV1 (percent predicted forced expiratory volume in one second)[4].

Reduction in Pulmonary Exacerbations

Trikafta has been associated with a reduced rate of pulmonary exacerbations, including events that require IV antibiotics and/or hospitalization. This reduction significantly improves the quality of life for patients with CF[4].

Market Challenges

While the market for these drugs is growing, there are several challenges to consider:

High Cost

The high cost of these treatments is a significant barrier to access. The incremental cost-effectiveness ratio (ICER) for Trikafta is high, making it challenging for some healthcare systems to justify the cost[4].

Regulatory and Access Issues

Delayed access to these treatments due to regulatory and reimbursement issues can impact patient outcomes. Studies have projected the potential impact of delayed access, highlighting the need for timely and equitable access to these therapies[3].

Future Prospects

The future looks promising for ivacaftor and its combination therapies.

Pipeline Development

Vertex Pharmaceuticals has a broad and deep clinical-stage pipeline, with new drug applications (NDAs) expected to be submitted to the FDA by mid-2024 for both VX-548 in acute pain and the Vanzacaftor Triple in CF. This pipeline ensures continued innovation and potential expansion of the market[5].

Expanding Indications

Ongoing research and clinical trials are exploring the use of these drugs in different patient populations and with various genotypes, which could further expand their market reach.

Key Takeaways

• Market Growth: The market for ivacaftor and its combination therapies is expected to see significant growth from 2023 to 2031.
• Financial Performance: Vertex Pharmaceuticals has reported substantial revenue growth driven by these drugs.
• Clinical Efficacy: These treatments have been shown to improve lung function, reduce pulmonary exacerbations, and enhance quality of life.
• Cost-Effectiveness: Despite high costs, these treatments are cost-effective in certain contexts.
• Future Prospects: Ongoing pipeline development and expanding indications promise continued market growth.

FAQs

What is the primary use of ivacaftor and its combination therapies?

Ivacaftor and its combination therapies, such as Trikafta, are primarily used for the treatment of cystic fibrosis in patients aged 12 years and older who have at least one F508del mutation in the CFTR gene.

How expensive is the treatment with Trikafta?

The treatment with Trikafta costs approximately $306,600 per patient per year.

What are the clinical benefits of Trikafta?

Trikafta has been shown to improve lung function, reduce pulmonary exacerbations, and enhance health-related quality of life for patients with CF.

Are these treatments cost-effective?

Despite their high cost, these treatments are considered cost-effective in certain contexts, particularly when compared to the long-term benefits and reduced healthcare utilization.

What are the future prospects for these drugs?

Vertex Pharmaceuticals has a robust pipeline with new drug applications expected to be submitted to the FDA by mid-2024, ensuring continued innovation and potential market expansion.

Sources

1. Market Research Intellect: Ivacaftor Reagent Market Size, Scope And Forecast Report.
2. The BMJ: Paying twice: the “charitable” drug with a high price tag.
3. Cystic Fibrosis Journal: Projecting the impact of delayed access to elexacaftor/tezacaftor/ivacaftor.
4. CADTH: Reimbursement Recommendation - Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta).
5. Vertex Pharmaceuticals: Fourth Quarter and Full Year 2023 Financial Results.