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Cystic Fibrosis

Rare Diseases
138
Pipeline Programs
30
Companies
50
Clinical Trials
1 recruiting
10
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
27
10
45
2
27
27
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
440%
Small Molecule
330%
RNA Therapeutic
220%
Peptide
110%
+ 379 programs with unclassified modality

Cystic Fibrosis is a ~$73M niche rare-disease market with established therapies and consolidating competition around inhalation antibiotics and CFTR modulators.

N/A — unable to attribute marketMature→ Stable30 products14 companies

Key Trends

  • Dominance of inhaled antibiotics (Cayston, Tobi) and chloride channel potentiators (Kalydeco) in revenue generation
  • Multiple patent cliffs approaching (Tobi Podhaler LOE 2030), creating generic erosion risk
  • Strong trial activity (451 trials) with emphasis on Phase 2–3 pipeline innovation

Career Verdict

Cystic Fibrosis offers stable, specialized career opportunities in rare-disease commercial and clinical roles, but limited revenue scale and near-term patent erosion mean growth potential is constrained.

AI-generated market analysis based on FDA, CMS, ClinicalTrials.gov, and hiring data

Market Leaders

#1CAYSTONDeclining
$34M
Gilead Sciences·LOE_APPROACHING
#2KALYDECOStable
$25M
#3TOBI PODHALERDeclining
$11M
Viatris·PEAK4.5yr
#4PULMOZYMEStable
$2M
Roche·LOE_APPROACHING

Drug Class Breakdown

Inhaled antibacterials (aztreonam, tobramycin)
$45M(62%)

patent cliffs accelerating erosion

Chloride channel potentiators (ivacaftor)
$25M(34%)

sustained demand, peak lifecycle

Mucolytics and osmotic agents (dornase, mannitol)
$2.1M(3%)

modest, supporting role

Other antibiotics (ofloxacin, other)
$0.2M(1%)

minimal market presence

Career Outlook

Stable

Cystic Fibrosis is a mature but specialized market offering stable employment in niche, high-impact roles. Career growth depends on pipeline advancement (Phase 3 trials suggest 3–5 year horizon for new launches) and willingness to specialize in rare-disease commercial infrastructure, patient education, and genetic therapies. Patent cliffs in inhaled antibiotics will accelerate consolidation and create merger-driven recruitment cycles.

Breaking In

Enter via clinical operations or medical affairs entry roles at Vertex, Gilead, or Roche; deep disease knowledge and patient-centricity are valued more than size of prior portfolio.

For Experienced Professionals

Experienced rare-disease professionals should prioritize roles at Vertex (CFTR leader) or prepare for consolidation/restructuring at Viatris and others facing 2030 patent cliffs; gene therapy and next-gen modulator launches offer 5–7 year career runway.

In-Demand Skills

Rare-disease commercial strategy and payer negotiationsPatient advocacy and outcomes managementInhalation drug delivery and formulation scienceCFTR modulator pharmacologyRegulatory expertise in orphan designations

Best For

Medical Science Liaison (MSL) — pulmonologists, specialistsRare-disease Brand ManagerPatient outcomes/health economics managerClinical trial recruiter/coordinatorRegulatory affairs specialist

Hiring Landscape

$112K–$307K

Cystic Fibrosis hiring is concentrated in commercial and clinical operations (1,392 commercial roles, 407 clinical operations roles across pharma), with strong representation from AbbVie, Johnson & Johnson, and Roche. Salaries are competitive ($255K average commercial, $307K clinical operations) but volumes are modest relative to larger therapeutic areas. Vertex Pharmaceuticals and Gilead Sciences—CF leaders—actively recruit for medical affairs and commercial development roles.

7,848
Open Roles
4
Companies Hiring
4
Departments

Top Hiring Companies

1495Growing
1034Growing

By Department

Commercial(18%)
$255K
Clinical Operations(5%)
$307K
Manufacturing(8%)
$112K
Medical Affairs(3%)
$302K

Cystic Fibrosis roles are well-compensated in clinical and medical affairs; however, limited market size and rare-disease focus mean fewer openings than oncology or metabolic areas.

On Market (10)

Approved therapies currently available

AbbVie
CREONApproved
pancrelipase
AbbVie
oral2009
1.5B Part D
Vertex Pharmaceuticals
KALYDECOApproved
ivacaftor
Vertex Pharmaceuticals
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC]oral2015
25M Part D
AbbVie
PERTZYEApproved
pancrelipase
AbbVie
oral2012
7M Part D
AbbVie
VIOKACEApproved
pancrelipase
AbbVie
oral2012
3M Part D
Vertex Pharmaceuticals
ORKAMBIApproved
lumacaftor and ivacaftor
Vertex Pharmaceuticals
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC]oral2018
1M Part D
Vertex Pharmaceuticals
TRIKAFTA (COPACKAGED)Approved
elexacaftor, tezacaftor, and ivacaftor
Vertex Pharmaceuticals
oral2023
Vertex Pharmaceuticals
SYMDEKO (COPACKAGED)Approved
tezacaftor and ivacaftor
Vertex Pharmaceuticals
oral2018
Vertex Pharmaceuticals
ALYFTREKApproved
vanzacaftor, tezacaftor, and deutivacaftor
Vertex Pharmaceuticals
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC]oral2024
Novartis
RECLASTApproved
zoledronic acid
Novartis
intravenous2007
Novartis
TOBREXApproved
tobramycin
Novartis
Aminoglycoside Antibacterial [EPC]ophthalmic1984

Competitive Landscape

75 companies ranked by most advanced pipeline stage

Vertex Pharmaceuticals
121 programs
1
Connected Devices for 3 monthsN/A1 trial
ELX/TEZ/IVAN/A1 trial
ELX/TEZ/IVAN/A1 trial
Energy Balance and Weight Gain With Ivacaftor TreatmentN/A1 trial
Gastrointestinal Study at Orkambi Therapy in CF PatientsN/A1 trial
+116 more programs
Active Trials
NCT03304028Terminated36Est. Jun 2020
NCT04058210Approved For Marketing
NCT04702360Approved For Marketing
+117 more trials
Sandoz
17 programs
3
2
5
4
OmalizumabPhase 4Monoclonal Antibody
Tobramycin Inhalation PowderPhase 4
Tobramycin Inhalation PowderPhase 4
Zoledronic acidPhase 4
Comprehensive Adherence ProgramPhase 3
+12 more programs
MSD
12 programs
1
6
3
2
Imipenem/Cilastatin/RelebactamPhase 41 trial
Tedizolid POPhase 41 trial
Denufosol TetrasodiumPhase 31 trial
Denufosol tetrasodium Inhalation SolutionPhase 31 trial
denufosol tetrasodiumPhase 31 trial
+7 more programs
Active Trials
NCT00034515Completed63Est. Aug 2002
NCT02170025Terminated21Est. Sep 2017
NCT00056147Completed90Est. Feb 2004
+9 more trials
Sharp Therapeutics
1
6
3
2
Imipenem/Cilastatin/RelebactamPhase 4
Tedizolid POPhase 4
Denufosol TetrasodiumPhase 3
Denufosol tetrasodium Inhalation SolutionPhase 3
denufosol tetrasodiumPhase 3
+7 more programs
Novartis
NovartisBASEL, Switzerland
6 programs
1
TOBI™ Podhaler™ 112 mg inhaled twice dailyN/A1 trial
TOBREX(Tobramycin)PHASE_15 trials
sodium chloridePHASE_11 trial
Comprehensive Adherence ProgramPHASE_3
OmalizumabPHASE_4Monoclonal Antibody5 trials
+1 more programs
Active Trials
NCT02113397Terminated1Est. Aug 2016
NCT02712983Completed107Est. Mar 2019
NCT02015663Terminated32Est. Dec 2014
+10 more trials
Chiesi
6 programs
2
2
2
Mannitol Inhalant ProductPhase 41 trial
tobramycinPhase 41 trial
tobramycinPhase 31 trial
tobramycin / BramitobPhase 31 trial
Bramitob® administered by PARI LC® PLUS nebulizerPhase 11 trial
+1 more programs
Active Trials
NCT01116089Completed25Est. Oct 2011
NCT04010799Completed68Est. Mar 2021
NCT01111383Completed209Est. May 2011
+3 more trials
ViiV Healthcare
4 programs
1
1
1
ceftazidimePhase 4
SB656933Phase 2
SB656933Phase 1
Fluticasone or placeboN/A
AbbVie
AbbVieNORTH CHICAGO, IL
3 programs
1
2
CREONPhase 41 trial
PancrelipasePhase 41 trial
ABBV-3067Phase 21 trial
Active Trials
NCT03969888Completed78Est. Jun 2022
NCT05069597Completed30Est. Aug 2023
NCT03924947Completed36Est. Jul 2022
Baxter
2 programs
1
ceftazidimePhase 4
MN 4000N/A
Shionogi
1 program
1
CefiderocolPhase 41 trial
Active Trials
NCT05314764Completed10Est. Dec 2024
Chiesi USA
1 program
1
Mannitol Inhalant ProductPhase 4
Cumberland Pharmaceuticals
1
Telavancin InjectionPhase 41 trial
Active Trials
NCT03172793Completed18Est. Apr 2019
Kite Pharma
22 programs
6
4
8
AZLIPhase 3
AZLIPhase 3
AZLIPhase 3
AZLI 75 mg three times a dayPhase 3
AZLI 75 mg three times dailyPhase 3
+17 more programs
Colorado Therapeutics
1
3
1
Once-daily insulin detemirPhase 31 trial
AquADEKs-2Phase 21 trial
Dulaglutide 0.75Mg/0.5Ml Inj PenPhase 2Peptide1 trial
RifampinPhase 21 trial
AquADEKPhase 1/21 trial
+13 more programs
Active Trials
NCT00788359Completed123Est. Jul 2014
NCT02372383Completed31Est. Jun 2016
NCT02211235Completed146Est. May 2018
+15 more trials
Syntara
9 programs
1
4
3
Inhaled mannitolPhase 31 trial
MannitolPhase 31 trial
inhaled mannitolPhase 31 trial
Inhaled MannitolPhase 21 trial
Inhaled mannitolPhase 21 trial
+4 more programs
Active Trials
NCT00779077Completed25Est. Feb 2009
NCT00792714Completed18Est. Sep 2009
NCT01883531Completed95Est. Oct 2015
+6 more trials
Amgen
AmgenTHOUSAND OAKS, CA
2 programs
1
1
MP-376Phase 31 trial
MP-376Phase 11 trial
Active Trials
NCT00840333Completed27Est. Dec 2009
NCT01270347Completed282Est. Jun 2013
Resolution Therapeutics
1
inhaled mannitolPhase 3
Algipharma
6 programs
1
1
2
2
OligoG Dry powder for inhalationPhase 2/31 trial
alginate oligosaccharidePhase 2/32 trials
OligoG DPIPhase 21 trial
Radiolabelled OligoG CF-5/20 DPIPhase 21 trial
OligoG CF-5/20Phase 1/21 trial
+1 more programs
Active Trials
NCT00970346Completed26Est. Nov 2009
NCT01465529Completed26Est. Nov 2013
NCT03822455Terminated15Est. Jul 2021
+4 more trials
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
4 programs
1
2
AZD1236Phase 21 trial
AZD9668Phase 21 trial
AZD5634 for inhalationPhase 11 trial
Non-Invasive Biomarkers in Cystic FibrosisN/A1 trial
Active Trials
NCT00413140Completed100Est. May 2005
NCT02679729Completed63Est. Oct 2016
NCT00812045Withdrawn44Est. Aug 2010
+1 more trials
Parion Sciences
4 programs
1
1
2
P-1037Phase 2
VX-371 + HSPhase 2
552-02Phase 1/21 trial
inhaled hypertonic salinePhase 11 trial
Active Trials
NCT02141191Completed12Est. Oct 2015
NCT00274313Completed40Est. Aug 2006
UNION therapeutics
1
1
DiponecaftorPhase 21 trial
BMD003Phase 11 trial
CeftazidimeN/A
Active Trials
NCT07485543Not Yet Recruiting24Est. Dec 2027
NCT06468527Completed41Est. Jun 2025
Enterprise Therapeutics
1
1
ETD001Phase 21 trial
ETD001 single dosePhase 11 trial
Active Trials
NCT04926701Completed98Est. Mar 2022
NCT06478706Completed57Est. Nov 2025
SpliSense
2 programs
1
1
SPL84Phase 21 trial
SPL84Phase 11 trial
Active Trials
NCT06217952Completed32Est. Aug 2023
NCT06429176Recruiting24Est. Dec 2025
Avanti Polar Lipids
2
Lym-X-Sorb powderPhase 21 trial
lipid absorption enhancerPhase 2
Active Trials
NCT00406536Completed110Est. Jun 2012
4D Molecular Therapeutics
1 program
1
4D-710Phase 21 trial
Active Trials
NCT05248230Recruiting30Est. Jun 2031
Armata Pharmaceuticals
1
AP-PA02Phase 22 trials
Active Trials
NCT05616221Completed48Est. Aug 2024
NCT04596319Completed29Est. Dec 2022
Arcturus Therapeutics
1
ARCT-032Phase 22 trials
Active Trials
NCT06747858Recruiting12Est. Dec 2025
NCT05712538Completed39Est. Jul 2024
Kamada
1 program
1
Aerosolized, human, plasma-derived Alpha-1 AntitrypsinPhase 21 trial
Active Trials
NCT00499837Completed21Est. Jul 2008
Bayer
BayerLEVERKUSEN, Germany
1 program
1
CiprofloxacinPhase 2Small Molecule
NovaBiotics
1 program
1
CysteaminePhase 21 trial
Active Trials
NCT03000348Completed91Est. Apr 2018

+45 more companies

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
ChiesiMannitol Inhalant Product
MSDImipenem/Cilastatin/Relebactam
ShionogiCefiderocol
AbbVieCREON
Paratek PharmaceuticalsOmadacycline Injection [Nuzyra]
AbbViePancrelipase
Cumberland PharmaceuticalsTelavancin Injection
Vertex PharmaceuticalsLUM/IVA
Vertex PharmaceuticalsLUM/IVA
MSDTedizolid PO
NovartisTobramycin
NovartisZoledronic acid
NovartisTobramycin
NovartisOmalizumab
Chiesitobramycin

Showing 15 of 48 trials with date data

Clinical Trials (50)

Total enrollment: 6,421 patients across 50 trials

NCT05740618ChiesiMannitol Inhalant Product

Inhaled Mannitol on Mucociliary Clearance in Moderate to Severe Cystic Fibrosis

Start: Mar 2023Est. completion: Jan 202514 patients
Phase 4Completed
NCT05561764MSDImipenem/Cilastatin/Relebactam

Imipenem/Cilastatin/Relebactam Pharmacokinetics, Safety, and Outcomes in Adults and Adolescents With Cystic Fibrosis

Start: Jan 2023Est. completion: Dec 20244 patients
Phase 4Terminated

Cefiderocol Pharmacokinetics in Adult Patients With Cystic Fibrosis

Start: Jun 2022Est. completion: Dec 202410 patients
Phase 4Completed

Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon

Start: Oct 2021Est. completion: Aug 202330 patients
Phase 4Completed
NCT04460586Paratek PharmaceuticalsOmadacycline Injection [Nuzyra]

Pharmacokinetics of Omadacycline in Cystic Fibrosis

Start: Jul 2021Est. completion: Dec 20239 patients
Phase 4Completed
NCT03924947AbbViePancrelipase

A Study to Compare US Marketed Creon Manufactured With a Modernized Process at an Alternate Manufacturing Site and Manufactured With the Approved Manufacturing Process at an Alternate Active Pharmaceutical Ingredient Site, in Participants With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

Start: Oct 2019Est. completion: Jul 202236 patients
Phase 4Completed

Telavancin Pharmacokinetics in Cystic Fibrosis Patients

Start: Aug 2017Est. completion: Apr 201918 patients
Phase 4Completed

A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation

Start: Sep 2016Est. completion: Oct 201770 patients
Phase 4Completed

A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation

Start: Jun 2016Est. completion: Aug 201724 patients
Phase 4Terminated
NCT02444234MSDTedizolid PO

Pharmacokinetics of Tedizolid Phosphate in Cystic Fibrosis

Start: Jul 2015Est. completion: Dec 201711 patients
Phase 4Completed

Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles

Start: Jan 2014Est. completion: Dec 201432 patients
Phase 4Terminated
NCT01702415NovartisZoledronic acid

Zoledronic Acid in Cystic Fibrosis

Start: Oct 2013Est. completion: Dec 20150
Phase 4Withdrawn

Ease of Use and Microbial Contamination of Tobramycin Inhalation Powder (TIP) Versus Nebulised Tobramycin Inhalation Solution (TIS) and Nebulised Colistimethate (COLI)

Start: Aug 2013Est. completion: Oct 201560 patients
Phase 4Completed

Predicting the Clinical Response to Omalizumab With Anti-Immunoglobulin E (IgE) Ab Response or Syk Expression in Basophils

Start: Feb 2013Est. completion: Aug 201617 patients
Phase 4Completed

Tobramycin 300 mg Once-a-day (o.d.) Aerosol in Adults With Cystic Fibrosis

Start: Jan 2012Est. completion: Jul 201210 patients
Phase 4Completed

Long Term Safety of Tobramycin Inhalation Powder in Patients With Cystic Fibrosis

Start: Jan 2012Est. completion: Jan 2014157 patients
Phase 4Completed

An Exploratory Study to Assess Multiple Doses of Omalizumab in Patients With Cystic Fibrosis Complicated by Acute Bronchopulmonary Aspergillosis (ABPA)

Start: Nov 2008Est. completion: Jul 201014 patients
Phase 4Terminated

A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis

Start: Jun 2008Est. completion: May 20093 patients
Phase 4Terminated

Omalizumab Use and Asthma-Related Quality of Life in Patients With Severe Persistent Allergic Asthma

Start: Nov 2007Est. completion: Apr 2010116 patients
Phase 4Completed

Safety and Tolerability of Omalizumab in Patients With Mild to Moderate Asthma

Start: Jul 200732 patients
Phase 4Completed

A Trial of Pulmozyme Withdrawal on Exercise Tolerance in Cystic Fibrosis Subjects With Severe Lung Disease (TOPIC)

Start: Mar 2007Est. completion: Nov 200827 patients
Phase 4Terminated

Safety and Efficacy Study of Omalizumab Given Prior to Immunotherapy

Start: May 2006Est. completion: Oct 200714 patients
Phase 4Completed

A Compassionate Access Protocol to Assess the Safety of XolairTM (Omalizumab) in Patients (≥ 6 Years Old) With Severe Allergic Asthma Who Remain Symptomatic Despite Optimal Therapy

Start: Jan 2005Est. completion: Dec 201148 patients
Phase 4Completed
NCT00333385GSKceftazidime

Continuous Versus Short Infusions of Ceftazidime in Cystic Fibrosis

Start: Oct 2001Est. completion: Apr 2004120 patients
Phase 4Terminated
NCT00005112GenentechHuman recombinant growth hormone

Growth Hormone Use in Cystic Fibrosis - a Multicenter Study

Phase 3Completed
NCT01315691InsmedLiposomal amikacin for inhalation

Study to Evaluate Arikace™ in CF Patients With Chronic Infection Due to Pseudomonas Aeruginosa

0
Phase 3Withdrawn

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older

Start: Nov 2024Est. completion: Sep 202750 patients
Phase 3Active Not Recruiting

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

Start: Aug 2023Est. completion: Jul 2029174 patients
Phase 3Enrolling By Invitation

Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age

Start: Jun 2023Est. completion: Sep 202570 patients
Phase 3Completed

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

Start: Nov 2022Est. completion: Apr 2027297 patients
Phase 3Active Not Recruiting

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

Start: Nov 2022Est. completion: Oct 2026822 patients
Phase 3Active Not Recruiting

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Start: Jun 2022Est. completion: Jun 2030210 patients
Phase 3Recruiting

Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation

Start: May 2022Est. completion: Jul 2023307 patients
Phase 3Completed

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

Start: Jan 2022Est. completion: Jan 202671 patients
Phase 3Completed

A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF)

Start: Jan 2022Est. completion: Dec 202286 patients
Phase 3Completed

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

Start: Oct 2021Est. completion: Nov 2023597 patients
Phase 3Completed

A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)

Start: Oct 2021Est. completion: Jul 202282 patients
Phase 3Completed

A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)

Start: Sep 2021Est. completion: Nov 2023435 patients
Phase 3Completed

A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)

Start: Jan 2021Est. completion: Jul 202269 patients
Phase 3Completed

A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes

Start: Jan 2021Est. completion: Mar 2023120 patients
Phase 3Completed

Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years

Start: Nov 2020Est. completion: Jun 202283 patients
Phase 3Completed

A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes

Start: Jun 2020Est. completion: May 2021121 patients
Phase 3Completed

A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy

Start: May 2020Est. completion: Dec 2022172 patients
Phase 3Completed

Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

Start: Feb 2020Est. completion: Aug 202352 patients
Phase 3Completed

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older

Start: Feb 2020Est. completion: Feb 202464 patients
Phase 3Completed

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Start: Dec 2019Est. completion: Dec 2022251 patients
Phase 3Completed

A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del

Start: Oct 2019Est. completion: Jul 2020176 patients
Phase 3Completed

A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Start: Aug 2019Est. completion: Jun 2020271 patients
Phase 3Completed

A Study Evaluating the Long-term Safety of VX-445 Combination Therapy

Start: Aug 2019Est. completion: Dec 2022458 patients
Phase 3Completed

A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Start: Oct 2018Est. completion: Jan 2023507 patients
Phase 3Completed

Related Jobs in Rare Diseases

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

27 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 6,421 patients
30 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.