Cystic Fibrosis

Rare Diseases
211
Pipeline Programs
30
Companies
50
Clinical Trials
1 recruiting
13
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
38
9
58
0
82
24
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
233%
Monoclonal Antibody
233%
Small Molecule
233%
+ 218 programs with unclassified modality

On Market (13)

Approved therapies currently available

VP
ALYFTREKApproved
vanzacaftor, tezacaftor, and deutivacaftor
Vertex Pharmaceuticals
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC]oral2024
U
CEFTAZIDIMEApproved
ceftazidime
Unknown Company
injection2010
Bayer
CIPROApproved
ciprofloxacin hydrochloride
Bayer
oral1987
U
IVACAFTORApproved
ivacaftor
Unknown Company
oral
VP
KALYDECOApproved
ivacaftor
Vertex Pharmaceuticals
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC]oral2015
VP
ORKAMBIApproved
lumacaftor and ivacaftor
Vertex Pharmaceuticals
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC]oral2015
U
PRANDINApproved
repaglinide
Unknown Company
oral1997
Novartis
RECLASTApproved
zoledronic acid
Novartis
intravenous2007
VP
SYMDEKO (COPACKAGED)Approved
tezacaftor and ivacaftor
Vertex Pharmaceuticals
oral2018
Novartis
TOBREXApproved
tobramycin
Novartis
Aminoglycoside Antibacterial [EPC]ophthalmic1981
VP
TRIKAFTA (COPACKAGED)Approved
elexacaftor, tezacaftor, and ivacaftor
Vertex Pharmaceuticals
oral2019
U
ZOLEDRONIC ACIDApproved
zoledronic acid
Unknown Company
intravenous2023
Novartis
ZOMETAApproved
zoledronic acid
Novartis
iv (infusion)2002

Competitive Landscape

32 companies ranked by most advanced pipeline stage

VP
121 programs
21
2
22
60
2
LUM/IVAPhase 41 trial
LUM/IVAPhase 41 trial
ELX/TEZ/IVAPhase 31 trial
ELX/TEZ/IVAPhase 31 trial
ELX/TEZ/IVAPhase 31 trial
+116 more programs
Active Trials
NCT03304028Terminated36Est. Jun 2020
NCT04702360Approved For Marketing
NCT04058210Approved For Marketing
+117 more trials
Genentech
6 programs
2
2
Dornase alfaPhase 41 trial
Dornase alfaPhase 41 trial
Comprehensive Adherence ProgramPhase 3
Human recombinant growth hormonePhase 31 trial
Oral Glucose tolerance testN/A1 trial
+1 more programs
Active Trials
NCT00287443Withdrawn0Est. Feb 2006
NCT00758771Completed38Est. Dec 2012
NCT00005112Completed
+2 more trials
Novartis
NovartisBASEL, Switzerland
6 programs
2
1
2
OmalizumabPhase 4Monoclonal Antibody5 trials
Zoledronic acidPhase 41 trial
Comprehensive Adherence ProgramPhase 31 trial
TOBREX(Tobramycin)Phase 15 trials
sodium chloridePhase 11 trial
+1 more programs
Active Trials
NCT02113397Terminated1Est. Aug 2016
NCT02712983Completed107Est. Mar 2019
NCT02015663Terminated32Est. Dec 2014
+11 more trials
M&
Merck & Co.RAHWAY, NJ
3 programs
1
2
Imipenem/Cilastatin/RelebactamPhase 41 trial
Tedizolid POPhase 41 trial
denufosol tetrasodiumPhase 25 trials
Active Trials
NCT01181622Completed25Est. Oct 2010
NCT00846781Terminated308Est. Feb 2011
NCT00425165Terminated6Est. May 2008
+4 more trials
AbbVie
AbbVieNORTH CHICAGO, IL
2 programs
1
1
CREONPhase 41 trial
ABBV-3067Phase 21 trial
Active Trials
NCT03969888Completed78Est. Jun 2022
NCT05069597Completed30Est. Aug 2023
Bayer
BayerLEVERKUSEN, Germany
2 programs
2
1
CIPRO(Ciprofloxacin)Phase 2Small Molecule1 trial
RiociguatPhase 2
Active Trials
NCT00645788Completed288Est. Jan 2011
Paratek Pharmaceuticals
1 program
1
Omadacycline Injection [Nuzyra]Phase 41 trial
Active Trials
NCT04460586CompletedEst. Dec 2023
Roche
RocheSTAVANGER NORWAY, Norway
1 program
1
ceftazidimePhase 41 trial
Active Trials
NCT00333385Terminated120Est. Apr 2004
GS
Gilead SciencesFOSTER CITY, CA
20 programs
5
3
8
AZLIPhase 35 trials
AZLIPhase 31 trial
AZLIPhase 31 trial
AZLI 75 mg three times dailyPhase 3
AZLI 75 mg three times dailyPhase 31 trial
+15 more programs
Active Trials
NCT00499720Approved For Marketing
NCT02110914Completed40Est. Dec 2016
NCT03607396Unknown75Est. Jul 2019
+17 more trials
Boehringer Ingelheim
Boehringer IngelheimINGELHEIM, Germany
10 programs
4
2
3
1
tiotropium Respimat® inhalerPhase 31 trial
BI 1265162Phase 21 trial
BI 1291583Phase 21 trial
BIIL 283 BSPhase 21 trial
BI 3720931Phase 1/21 trial
+5 more programs
Active Trials
NCT02976519Completed64Est. Aug 2018
NCT02269189Completed36
NCT02265679Completed45
+7 more trials
PTC Therapeutics
9 programs
4
5
AtalurenPhase 31 trial
AtalurenPhase 31 trial
AtalurenPhase 31 trial
AtalurenPhase 31 trial
AtalurenPhase 31 trial
+4 more programs
Active Trials
NCT00237380Completed24Est. May 2006
NCT00458341Completed30Est. Feb 2008
NCT00234663Completed24Est. Aug 2006
+6 more trials
Insmed
InsmedBRIDGEWATER, NJ
7 programs
1
2
2
2
Liposomal amikacin for inhalationPhase 31 trial
Liposomal amikacin for inhalationPhase 31 trial
Arikayce™Phase 21 trial
BrensocatibPhase 21 trial
ARIKACE™Phase 1/21 trial
+2 more programs
Active Trials
NCT06238856Completed18Est. Feb 2005
NCT00777296Completed66Est. Feb 2008
NCT00558844Completed41Est. Jun 2009
+4 more trials
Amgen
AmgenTHOUSAND OAKS, CA
2 programs
1
1
MP-376Phase 31 trial
MP-376Phase 11 trial
Active Trials
NCT00503490Completed40Est. Dec 2007
NCT01270347Completed282Est. Jun 2013
Novo Nordisk
2 programs
2
Insulin AspartePhase 31 trial
RepaglinidePhase 31 trial
Active Trials
NCT00072904Completed108Est. Dec 2007
NCT00662714Completed73Est. Dec 2011
Galapagos
Galapagos2800 MECHELEN, Belgium
8 programs
3
5
GLPG1837 dose 1Phase 21 trial
GLPG1837 dose 1Phase 21 trial
GLPG2222 150 mg q.d.Phase 21 trial
GLPG2222 50 mgPhase 21 trial
GLPG2737Phase 21 trial
+3 more programs
Active Trials
NCT03540524Completed10Est. Mar 2019
NCT03450720Completed6Est. Aug 2017
NCT03589313Completed6Est. Mar 2018
+5 more trials
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
2 programs
1
AZD9668Phase 25 trials
Non-Invasive Biomarkers in Cystic FibrosisN/A1 trial
Active Trials
NCT00413140Completed100Est. May 2005
NCT01214122Withdrawn0Est. Dec 2010
NCT01161355Completed6Est. Jun 2010
+3 more trials
Grifols
GrifolsNEW YORK, NY
2 programs
2
Alpha-1 HC 100 mgPhase 21 trial
Alpha1-Proteinase InhibitorPhase 21 trial
Active Trials
NCT01684410CompletedEst. Oct 2013
NCT00486837CompletedEst. Jun 2004
EP
Eloxx PharmaceuticalsIsrael - Nes Tziona
2 programs
2
ELX-02Phase 21 trial
ELX-02Phase 21 trial
Active Trials
NCT04135495CompletedEst. Oct 2022
NCT04126473CompletedEst. Apr 2022
Corbus Pharmaceuticals
2 programs
2
JBT-101Phase 21 trial
Lenabasum 20 mgPhase 21 trial
Active Trials
NCT02465450Completed85Est. Dec 2016
NCT03451045Completed447Est. Jun 2020
Cook Medical
Cook MedicalIN - Bloomington
2 programs
2
RifampinPhase 21 trial
Trimethoprim SulfamethoxazolePhase 2Small Molecule1 trial
Active Trials
NCT01349192TerminatedEst. May 2015
NCT03489629CompletedEst. Jan 2026
Avanti Polar Lipids
Avanti Polar LipidsAL - Birmingham
1 program
1
Lym-X-Sorb powderPhase 21 trial
Active Trials
NCT00406536CompletedEst. Jun 2012
GC Biopharma
1 program
1
MAG-DHAPhase 21 trial
Active Trials
NCT02518672TerminatedEst. Nov 2016
Liminal BioSciences
1 program
1
PBI4050Phase 21 trial
Active Trials
NCT02955888TerminatedEst. Feb 2018
ReCode Therapeutics
ReCode TherapeuticsCA - Menlo Park
1 program
1
RCT2100Phase 21 trial
Active Trials
NCT06237335Recruiting192Est. Dec 2026
Verona Pharma
Verona PharmaUK - London
1 program
1
RPL554Phase 21 trial
Active Trials
NCT02919995Completed10Est. Nov 2017
Swedish Orphan Biovitrum
1 program
1
rhBSSLPhase 21 trial
Active Trials
NCT00743483CompletedEst. Jun 2009
Armata Pharmaceuticals
Armata PharmaceuticalsLOS ANGELES, CA
1 program
1
AP-PA02Phase 1/21 trial
Active Trials
NCT04596319CompletedEst. Dec 2022
Spirovant Sciences
Spirovant SciencesPA - Philadelphia
1 program
1
SP-101 and doxorubicin Cohort 1Phase 1/21 trial
Active Trials
NCT06526923RecruitingEst. Dec 2026
Moderna
ModernaCAMBRIDGE, MA
1 program
1
VX-522 mRNA therapyPhase 1/2RNA Therapeutic1 trial
Active Trials
NCT05668741Recruiting39Est. May 2027
CSL Behring
1 program
1
CR002 Liquid APIPhase 11 trial
Active Trials
NCT01347190Completed25Est. Aug 2011

+2 more companies

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Merck & Co.Imipenem/Cilastatin/Relebactam
AbbVieCREON
Paratek PharmaceuticalsOmadacycline Injection [Nuzyra]
NovartisOmalizumab
NovartisOmalizumab
Vertex PharmaceuticalsLUM/IVA
Vertex PharmaceuticalsLUM/IVA
Merck & Co.Tedizolid PO
NovartisTobramycin
NovartisOmalizumab
NovartisZoledronic acid
NovartisTobramycin
NovartisOmalizumab
NovartisTobramycin
NovartisOmalizumab

Showing 15 of 48 trials with date data

Clinical Trials (50)

Total enrollment: 5,887 patients across 50 trials

NCT05561764Merck & Co.Imipenem/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

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 2023
Phase 4Completed

Behandling af Boern Med Foedevareallergi Med Omalizumab (Xolair)

Start: Nov 2019Est. completion: Mar 202220 patients
Phase 4Completed

Treatment of Idiopathic Angioedema With Xolair as Add-on Therapy

Start: Mar 2017Est. completion: Jan 202012 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

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

Study to Assess the Efficacy and Safety of Omalizumab Treatment on ICS Reduction for Severe IgE-mediated Asthma

Start: Nov 2013Est. completion: Jan 2016112 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

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

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

Evaluate the Effect of Omalizumab on Markers of Asthma Impairment in Patients With Persistent Allergic Asthma

Start: Mar 2009Est. completion: Mar 2010271 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

Efficacy of Omalizumab in Adults (18-60 Years of Age) With Moderate-Severe, Persistent Allergic Asthma, Despite Receiving Inhaled Corticosteroids and Long Acting Beta-agonists

Start: Apr 2008Est. completion: Nov 201136 patients
Phase 4Completed

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

The Effect of Xolair (Omalizumab) on Allergy Blood Cells

Start: Mar 2007Est. completion: Oct 200749 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

Effect of Omalizumab on Expression of IgE Receptors in Adults With Severe, Inadequately Controlled Allergic Asthma

Start: Dec 2006Est. completion: Mar 200831 patients
Phase 4Completed

Safety and Efficacy Study of Omalizumab Given Prior to Immunotherapy

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

Study to Evaluate the Effect of Omalizumab on Improving the Tolerability of Specific Immunotherapy in Patients With Persistent Allergic Asthma

Start: Dec 2005Est. completion: Apr 2008275 patients
Phase 4Completed

Omalizumab in Adult and Adolescent Patients With Severe Persistent Allergic Asthma

Start: Nov 2005Est. completion: Sep 2008406 patients
Phase 4Completed

Xolair in Patients With Chronic Sinusitis

Start: Jul 2005Est. completion: Jan 200914 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
NCT00333385Rocheceftazidime

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

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

82 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 5,887 patients
30 companies competing in this space