CLINICAL TRIALS PROFILE FOR IVACAFTOR

All Clinical Trials for Ivacaftor

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00457821 ↗	Safety Study of Ivacaftor in Subjects With Cystic Fibrosis	Completed	Cystic Fibrosis Foundation	Phase 2	2007-05-01	The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.
NCT00457821 ↗	Safety Study of Ivacaftor in Subjects With Cystic Fibrosis	Completed	Cystic Fibrosis Foundation Therapeutics	Phase 2	2007-05-01	The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.
NCT00457821 ↗	Safety Study of Ivacaftor in Subjects With Cystic Fibrosis	Completed	Vertex Pharmaceuticals Incorporated	Phase 2	2007-05-01	The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.
NCT00909532 ↗	Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation	Completed	Cystic Fibrosis Foundation	Phase 3	2009-06-01	The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.
NCT00909532 ↗	Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation	Completed	Cystic Fibrosis Foundation Therapeutics	Phase 3	2009-06-01	The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.

Clinical Trial Conditions for Ivacaftor

Condition Name

Condition Name for Ivacaftor
Intervention	Trials
Cystic Fibrosis	110
Chronic Obstructive Pulmonary Disease	3
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation	2
Diabetes	2

Condition MeSH

Condition MeSH for Ivacaftor
Intervention	Trials
Cystic Fibrosis	113
Fibrosis	106
Lung Diseases	5
Pulmonary Disease, Chronic Obstructive	3

Clinical Trial Locations for Ivacaftor

Trials by Country

Trials by Country for Ivacaftor
Location	Trials
Canada	96
United Kingdom	83
Australia	65
Germany	61
France	55

Trials by US State

Trials by US State for Ivacaftor
Location	Trials
Massachusetts	55
California	53
Missouri	52
Ohio	51
Pennsylvania	50

Clinical Trial Progress for Ivacaftor

Clinical Trial Phase

Clinical Trial Phase for Ivacaftor
Clinical Trial Phase	Trials
Phase 4	14
Phase 3	59
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Ivacaftor
Clinical Trial Phase	Trials
Completed	76
Recruiting	17
Active, not recruiting	12

Clinical Trial Sponsors for Ivacaftor

Sponsor Name

Sponsor Name for Ivacaftor
Sponsor	Trials
Vertex Pharmaceuticals Incorporated	93
Cystic Fibrosis Foundation	14
Cystic Fibrosis Foundation Therapeutics	13

Sponsor Type

Sponsor Type for Ivacaftor
Sponsor	Trials
Industry	105
Other	76
NIH	5

Ivacaftor: Clinical Trials, Market Analysis, and Projections

Introduction

Ivacaftor, marketed under the brand name Kalydeco®, is a groundbreaking medication for the treatment of cystic fibrosis (CF), particularly in patients with specific CFTR (cystic fibrosis transmembrane conductance regulator) mutations. Here, we delve into the recent clinical trials, market analysis, and future projections for this drug.

Clinical Trials Overview

Trials in Various Age Groups

Several clinical trials have been conducted to evaluate the safety and efficacy of ivacaftor in different age groups and with various CFTR mutations.

• Infants and Young Children: Studies such as the Vertex VX15-770-124 and VX-770-108 trials have focused on infants and young children (ages 12-24 months and 2-5 years, respectively) with CFTR gating mutations. These trials have shown promising results in terms of safety and effectiveness, with significant improvements in lung function and other clinical outcomes[1].
• Children and Adolescents: The ENVISION trial (Trial 2) involved children aged 6 to <11 years with the G551D mutation, demonstrating significant improvements in lung function over a 48-week period[4].
• Adults: Trials such as the VX-770-111 (KONNECTION) study have included adults with non-G551D CFTR gating mutations, showing long-term safety and efficacy[1].

Key Trial Findings

• FEV1 Improvement: Many trials have reported substantial improvements in forced expiratory volume in 1 second (FEV1) percent predicted, a key indicator of lung function in CF patients[1][4].
• Long-term Safety: Long-term safety studies, such as the PERSIST trial, have confirmed that ivacaftor is well-tolerated over extended periods, with minimal adverse effects[1].
• Quality of Life: Studies have also shown that ivacaftor significantly improves quality-adjusted life-years (QALYs) and life expectancy, bringing health outcomes closer to those of the non-CF population[3].

Market Analysis

Current Market Size and Growth

The global cystic fibrosis market, driven in part by ivacaftor and other CFTR modulators, was valued at $8.2 billion in 2020. This market is projected to grow at a CAGR of more than 4% from 2021 to 2025, driven by the continued uptake of high-priced treatments and label expansions[5].

Ivacaftor API Market

The ivacaftor API (Active Pharmaceutical Ingredient) market is expected to experience significant growth, with a projected CAGR of 6.4% from 2024 to 2032. This growth is fueled by the increasing prevalence of cystic fibrosis, advancements in pharmaceutical manufacturing, and rising healthcare expenditures[2].

Key Players

The ivacaftor API market is dominated by several key players, including Vertex Pharmaceuticals Incorporated, Teva Pharmaceutical Industries Ltd., Cipla Limited, Mylan N.V., and Hetero Drugs Limited. These companies are driving innovation and competition in the market[2].

Market Projections

Future Growth Drivers

• Expanding Patient Population: As more patients become eligible for ivacaftor due to label expansions and new indications, the market is expected to grow.
• New Formulations and Combinations: The launch of new formulations, such as triple combination therapies like VX-121 + tezacaftor + VX-561 (deutivacaftor), will further drive market growth[5].
• Geographical Expansion: The market is expected to expand in regions such as North America, Europe, Asia-Pacific, and other emerging markets[2].

Cost and Budget Impact

Despite the high cost of ivacaftor, its budget impact is relatively limited. A study estimated that the incremental lifetime cost of ivacaftor is significant, but the budget impact per member per month is minimal, at $0.087[3].

Competitive Landscape

The competitive landscape of the ivacaftor API market is characterized by extensive competitive intelligence, with companies focusing on product type, end-user applications, and regional market share. The market is segmented by oral dosage form, injectable dosage form, and other forms, with cystic fibrosis being the primary application[2].

Economic and Clinical Impact

Life-Years and QALYs

Ivacaftor has been associated with significant improvements in life-years and QALYs. A study estimated that ivacaftor adds approximately 18.25 additional life-years and 15.03 additional QALYs compared to usual care alone[3].

Cost-Effectiveness

While ivacaftor is costly, its long-term benefits in terms of improved health outcomes and reduced morbidity and mortality make it a cost-effective option for CF patients with specific mutations.

Key Takeaways

• Clinical Efficacy: Ivacaftor has demonstrated significant improvements in lung function and quality of life across various age groups and CFTR mutations.
• Market Growth: The ivacaftor API market is projected to grow at a CAGR of 6.4% from 2024 to 2032, driven by increasing prevalence and advancements in pharmaceuticals.
• Cost and Budget Impact: Despite high costs, ivacaftor has a relatively limited budget impact and is considered cost-effective in the long term.
• Future Drivers: Expanding patient populations, new formulations, and geographical expansion will drive future market growth.

FAQs

What is ivacaftor used for?

Ivacaftor is used to treat cystic fibrosis in patients with specific CFTR gating mutations, such as G551D, G178R, and others.

What are the key clinical trial findings for ivacaftor?

Clinical trials have shown significant improvements in lung function (FEV1), long-term safety, and quality of life for patients with CF treated with ivacaftor.

How is the ivacaftor API market expected to grow?

The ivacaftor API market is projected to grow at a CAGR of 6.4% from 2024 to 2032, driven by increasing prevalence of CF, advancements in pharmaceutical manufacturing, and rising healthcare expenditures.

Who are the key players in the ivacaftor API market?

Key players include Vertex Pharmaceuticals Incorporated, Teva Pharmaceutical Industries Ltd., Cipla Limited, Mylan N.V., and Hetero Drugs Limited.

What is the budget impact of ivacaftor?

Despite its high cost, ivacaftor has a relatively limited budget impact, estimated at $0.087 per member per month.

Sources

1. Clinical Trials Finder - Find a Cystic Fibrosis Care Center. Cystic Fibrosis Foundation.
2. Ivacaftor API Market to Set an Explosive Growth in Near Future. OpenPR.
3. Forecasting US ivacaftor outcomes and cost in cystic fibrosis. European Respiratory Journal.
4. Clinical Trials for Patients Age 6 Years or Older. KalydecoHCP.
5. Cystic Fibrosis - Global Drug Forecast and Market Analysis to 2030. GlobalData.