Cystic Fibrosis
Respiratory 2 original drugsPhase 3 Readouts Pro
8 active Phase 3 trials with confidence-graded completion dates.
Anchored on CT.gov primary completion date. Topline announcements typically precede this by 3–9 months. Confidence labels combine date type (ACTUAL/ESTIMATED) with last-update freshness.
Treatments by Mechanism
Top 10 mechanisms across 25 industry trials with a known mechanism of action.
MoA derived from FDA pharmClassEpc when intervention matches an approved drug. Codenamed clinical-stage assets without an approved counterpart show "—" and aren't grouped here — they're still in the phase tables below.
Clinical Pipeline by Phase
313 industry-sponsored trials across 88 sponsors
| Sponsor | Asset | MoA | Status | Primary Completion | NCT |
|---|---|---|---|---|---|
| AbbVie | CREON | amylases/lipases/proteases | COMPLETED | 2023-07-07 | NCT05069597 |
| AbbVie | Pancrelipase | — | COMPLETED | 2022-07-11 | NCT03924947 |
| Mylan Inc. | Placebo | — | COMPLETED | 2019-03-19 | NCT03502070 |
| Anthera Pharmaceuticals | Liprotamase | — | COMPLETED | 2018-03 | NCT02823964 |
| Vertex Pharmaceuticals Incorporated | LUM/IVA | — | COMPLETED | 2017-09 | NCT02875366 |
| Novartis | Tobramycin Inhalation Powder | — | COMPLETED | 2015-10 | NCT01844778 |
| Novartis | Placebo | — | COMPLETED | 2015-02 | NCT02178540 |
| Novartis | TBM100 | — | COMPLETED | 2014-01 | NCT01519661 |
| Roche | dornase alfa [Pulmozyme®] | — | COMPLETED | 2013-06 | NCT01712334 |
| Forest Laboratories | Panzytrat® 25,000 | — | COMPLETED | 2012-05 | NCT01327703 |
| Forest Laboratories | EUR-1008 (APT-1008) | — | COMPLETED | 2011-12 | NCT01131507 |
| Forest Laboratories | EUR-1008 (APT-1008) | — | COMPLETED | 2010-12 | NCT01100606 |
| Vertex Pharmaceuticals Incorporated | LUM/IVA | — | TERMINATED | 2017-08 | NCT02823470 |
| Novartis | TIS or TIP | — | TERMINATED | 2017-04-10 | NCT02248922 |
| Novartis | Tobramycin Inhalation Powder | — | TERMINATED | 2014-12 | NCT02015663 |
| Novartis | Omalizumab | Anti-IgE [EPC] | TERMINATED | 2010-07 | NCT00787917 |
| Digestive Care, Inc. | Pancrelipase | — | TERMINATED | 2010-05 | NCT00744250 |
| Roche | Dornase alfa | Recombinant Human Deoxyribonuclease 1 [EPC] | TERMINATED | 2009-05 | NCT00680316 |
| Sponsor | Asset | MoA | Status | Primary Completion | NCT |
|---|---|---|---|---|---|
| Haisco Pharmaceutical Group Co., Ltd. | HSK31858, tablet | — | RECRUITING | 2026-11-28 | NCT06660992 |
| Vertex Pharmaceuticals Incorporated | VX-121/TEZ/D-IVA | — | RECRUITING | 2030-06-30 | NCT05422222 |
| Vertex Pharmaceuticals Incorporated | VX-121/TEZ/D-IVA | — | ACTIVE NOT RECRUITING | 2026-10-30 | NCT05444257 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | ACTIVE NOT RECRUITING | 2027-04-06 | NCT05331183 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | ACTIVE NOT RECRUITING | 2027-09-30 | NCT06460506 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2026-01-29 | NCT05153317 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2025-09-04 | NCT05882357 |
| Insmed Incorporated | Brensocatib 10 mg | — | COMPLETED | 2024-10-28 | NCT04594369 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2024-02-24 | NCT04183790 |
| Vertex Pharmaceuticals Incorporated | IVA | — | COMPLETED | 2023-10-02 | NCT03277196 |
| Vertex Pharmaceuticals Incorporated | LUM/IVA | — | COMPLETED | 2023-08-22 | NCT04235140 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2023-07-05 | NCT05274269 |
| Vertex Pharmaceuticals Incorporated | VX-121/TEZ/D-IVA | — | COMPLETED | 2023-05-18 | NCT05076149 |
| Vertex Pharmaceuticals Incorporated | VX-121/TEZ/D-IVA | — | COMPLETED | 2023-05-12 | NCT05033080 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2023-03-24 | NCT04545515 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2023-01-09 | NCT03525574 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2022-12-21 | NCT04362761 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2022-12-20 | NCT05111145 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2022-12-16 | NCT04058366 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2022-12-14 | NCT04043806 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2022-07-26 | NCT04969224 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2022-07-14 | NCT04599465 |
| Vertex Pharmaceuticals Incorporated | IVA | — | COMPLETED | 2022-06-28 | NCT02725567 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2022-06-03 | NCT04537793 |
| Vertex Pharmaceuticals Incorporated | LUM | — | COMPLETED | 2021-10-29 | NCT03601637 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2021-05-17 | NCT04353817 |
| Zambon SpA | CMS | — | COMPLETED | 2021-04-09 | NCT03093974 |
| Vertex Pharmaceuticals Incorporated | TEZ/IVA | — | COMPLETED | 2020-10-28 | NCT03537651 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2020-08-07 | NCT03691779 |
| Savara Inc. | Vancomycin inhalation powder | — | COMPLETED | 2020-07-28 | NCT03181932 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2020-07-24 | NCT04105972 |
| Vertex Pharmaceuticals Incorporated | ELX/TEZ/IVA | — | COMPLETED | 2020-06-12 | NCT04058353 |
| Vertex Pharmaceuticals Incorporated | LUM/IVA | — | COMPLETED | 2019-07-17 | NCT03125395 |
| Vertex Pharmaceuticals Incorporated | TEZ/IVA | — | COMPLETED | 2019-05 | NCT02565914 |
| Vertex Pharmaceuticals Incorporated | VX-445/TEZ/IVA | — | COMPLETED | 2019-04-24 | NCT03525444 |
| Vertex Pharmaceuticals Incorporated | VX-659/TEZ/IVA | — | COMPLETED | 2019-02-05 | NCT03447249 |
| Vertex Pharmaceuticals Incorporated | VX-445/TEZ/IVA | — | COMPLETED | 2018-12-28 | NCT03525548 |
| Vertex Pharmaceuticals Incorporated | TEZ/IVA | — | COMPLETED | 2018-12-21 | NCT03559062 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2018-12-18 | NCT03068312 |
| Vertex Pharmaceuticals Incorporated | VX-659/TEZ/IVA | — | COMPLETED | 2018-09-26 | NCT03460990 |
| Vertex Pharmaceuticals Incorporated | TEZ | — | COMPLETED | 2018-09 | NCT02953314 |
| Vertex Pharmaceuticals Incorporated | Tezacaftor/Ivacaftor | — | COMPLETED | 2018-08-09 | NCT03150719 |
| Vertex Pharmaceuticals Incorporated | LUM/IVA | — | COMPLETED | 2018-08 | NCT02544451 |
| AO GENERIUM | Tigerase® | — | COMPLETED | 2018-05-16 | NCT04468100 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2017-09 | NCT02412111 |
| Vertex Pharmaceuticals Incorporated | LUM/IVA | — | COMPLETED | 2017-09 | NCT02797132 |
| Syntara | Inhaled mannitol | — | COMPLETED | 2017-02 | NCT02134353 |
| Vertex Pharmaceuticals Incorporated | VX-661/Ivacaftor | — | COMPLETED | 2017-02 | NCT02392234 |
| Vertex Pharmaceuticals Incorporated | VX-661 Plus Ivacaftor Combination | — | COMPLETED | 2017-01-20 | NCT02347657 |
| Anthera Pharmaceuticals | Liprotamase Powder for Oral Solution | — | COMPLETED | 2016-12 | NCT02734810 |
| PTC Therapeutics | Ataluren (PTC124®) | — | COMPLETED | 2016-11 | NCT02139306 |
| Anthera Pharmaceuticals | Liprotamase | — | COMPLETED | 2016-10 | NCT02279498 |
| Vertex Pharmaceuticals Incorporated | Lumacaftor | — | COMPLETED | 2016-10 | NCT02390219 |
| Vertex Pharmaceuticals Incorporated | VX-809 | — | COMPLETED | 2016-09 | NCT02514473 |
| Aradigm Corporation | Ciprofloxacin dispersion for inhalation | — | COMPLETED | 2016-08-17 | NCT01515007 |
| Aradigm Corporation | Ciprofloxacin dispersion for inhalation | — | COMPLETED | 2016-08-11 | NCT02104245 |
| Vertex Pharmaceuticals Incorporated | VX-661 plus ivacaftor combination | — | COMPLETED | 2016-06-07 | NCT02516410 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2016-04 | NCT01707290 |
| Vertex Pharmaceuticals Incorporated | Lumacaftor Plus Ivacaftor Combination | — | COMPLETED | 2016-04 | NCT01931839 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2015-12 | NCT01946412 |
| Vertex Pharmaceuticals Incorporated | Lumacaftor | — | COMPLETED | 2015-10 | NCT01897233 |
| Insmed Incorporated | Liposomal amikacin for inhalation | — | COMPLETED | 2015-07-16 | NCT01316276 |
| Novartis | TOBI | Aminoglycoside Antibacterial [EPC] | COMPLETED | 2015-06 | NCT01082367 |
| Gilead | AZLI | — | COMPLETED | 2015-01 | NCT01641822 |
| Abbott | Creon® | — | COMPLETED | 2014-08 | NCT02137382 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2014-05 | NCT01117012 |
| Vertex Pharmaceuticals Incorporated | Lumacaftor Plus Ivacaftor Combination | — | COMPLETED | 2014-04 | NCT01807949 |
| Vertex Pharmaceuticals Incorporated | Lumacaftor Plus Ivacaftor Combination | — | COMPLETED | 2014-04 | NCT01807923 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2014-03 | NCT01705145 |
| Forest Laboratories | EUR-1008 25,000 Units | — | COMPLETED | 2014-02 | NCT01641393 |
| PTC Therapeutics | Ataluren | — | COMPLETED | 2013-12-02 | NCT01140451 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2013-10 | NCT01614457 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2013-10 | NCT01614470 |
| Gilead | AZLI | — | COMPLETED | 2013-04 | NCT01404234 |
| Amgen | MP-376 (Levofloxacin Solution for Inhalation) | — | COMPLETED | 2012-10 | NCT01270347 |
| Amgen | Aeroquin | — | COMPLETED | 2012-05-07 | NCT01180634 |
| Boehringer Ingelheim | tiotropium Respimat® inhaler | — | COMPLETED | 2012-03 | NCT01179347 |
| PTC Therapeutics | Ataluren | — | COMPLETED | 2011-11-12 | NCT00803205 |
| Novartis | Tobramycin inhalation powder | — | COMPLETED | 2011-10 | NCT00982930 |
| Gilead | AZLI | — | COMPLETED | 2011-09 | NCT01059565 |
| Chiesi Farmaceutici S.p.A. | tobramycin | Aminoglycoside Antibacterial [EPC] | COMPLETED | 2011-05 | NCT01111383 |
| Novartis | Tobramycin Inhalation Powder | — | COMPLETED | 2011-05 | NCT00918957 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2010-11 | NCT00909727 |
| Merck | Denufosol Tetrasodium (INS37217) Inhalation Solution | — | COMPLETED | 2010-10 | NCT00625612 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2010-07 | NCT00909532 |
| Gilead | Aztreonam for Inhalation Solution (AZLI) | — | COMPLETED | 2010-05 | NCT00757237 |
| Syntara | inhaled mannitol | — | COMPLETED | 2010-04 | NCT00630812 |
| Chiesi Farmaceutici S.p.A. | tobramycin / Bramitob | — | COMPLETED | 2010-04 | NCT00885365 |
| Forest Laboratories | Ultrase® MT12 | — | COMPLETED | 2009-11 | NCT00880100 |
| Gilead | AZLI 75 mg three times daily (TID) | — | COMPLETED | 2009-06 | NCT00712166 |
| Solvay Pharmaceuticals | Pancrelipase Delayed Release | — | COMPLETED | 2009-06 | NCT00775528 |
| Johnson & Johnson Pharmaceutical Research & Development, L.L.C. | Pancrease MT 10.5, or MT 21 | — | COMPLETED | 2009-02 | NCT00662675 |
| Solvay Pharmaceuticals | Pancrelipase Delayed Release | — | COMPLETED | 2008-12 | NCT00690820 |
| AstraZeneca | Benralizumab | Interleukin-5 Receptor alpha-directed Cytolytic Antibody [EPC] | TERMINATED | 2024-04-16 | NCT05006573 |
| Zambon SpA | CMS | — | TERMINATED | 2022-03-15 | NCT03460704 |
| Vertex Pharmaceuticals Incorporated | VX-659/TEZ/IVA | — | TERMINATED | 2020-09-09 | NCT03447262 |
| Gilead | AZLI | — | TERMINATED | 2020-05-27 | NCT03219164 |
| Vertex Pharmaceuticals Incorporated | VX-659/TEZ/IVA | — | TERMINATED | 2019-01-18 | NCT03633526 |
| Vertex Pharmaceuticals Incorporated | ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | TERMINATED | 2017-08 | NCT02742519 |
| PTC Therapeutics | Ataluren | — | TERMINATED | 2017-06-05 | NCT02107859 |
| Sponsor | Asset | MoA | Status | Primary Completion | NCT |
|---|---|---|---|---|---|
| ReCode Therapeutics | RCT2100 | — | RECRUITING | 2026-08-30 | NCT06237335 |
| Vertex Pharmaceuticals Incorporated | VX-522 mRNA therapy | — | RECRUITING | 2026-12-23 | NCT05668741 |
| Spirovant Sciences, Inc. | — | — | RECRUITING | 2026-12-31 | NCT06526923 |
| Arcturus Therapeutics, Inc. | ARCT-032 | — | RECRUITING | 2027-06 | NCT06747858 |
| 4D Molecular Therapeutics | 4D-710 | — | RECRUITING | 2027-06 | NCT05248230 |
| Anagram Therapeutics, Inc. | ANG003 Dose A | — | RECRUITING | 2027-07 | NCT07450547 |
| Verona Pharma, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA | Nebulized Ensifentrine Suspension; 3 mg | — | RECRUITING | 2027-09-24 | NCT06559150 |
| SpliSense Ltd. | SPL84 | — | RECRUITING | 2027-10-01 | NCT06429176 |
| Sanofi | SAR445399 | — | RECRUITING | 2027-10-29 | NCT07547436 |
| Roche | GDC-6988 | — | RECRUITING | 2027-11-15 | NCT06603246 |
| CSL Behring | CSL787 | — | RECRUITING | 2028-02-28 | NCT07048262 |
| Respirion Pharmaceuticals Pty Ltd | RSP-1502 | — | RECRUITING | 2026-04 | NCT06016088 |
| BiomX Ltd | BX004 | — | RECRUITING | 2026-03 | NCT06998043 |
| Sionna Therapeutics Inc. | SION-719 | — | ACTIVE NOT RECRUITING | 2026-07 | NCT07108153 |
| Thirty Respiratory Limited | RESP302 | — | ACTIVE NOT RECRUITING | 2028-02-05 | NCT06663176 |
| Boehringer Ingelheim | — | — | ACTIVE NOT RECRUITING | 2040-10-29 | NCT06962852 |
| S-INFINITY Pharmaceuticals Co., Ltd | — | — | NOT YET RECRUITING | 2027-01-08 | NCT06981091 |
| Aimmune Nestlé Health Science US R&D, LLC | NHS7108 | — | NOT YET RECRUITING | 2027-06 | NCT07559175 |
| Clarametyx Biosciences, Inc. | CMTX-101 | — | COMPLETED | 2025-11-14 | NCT06159725 |
| Enterprise Therapeutics Ltd | ETD001 | — | COMPLETED | 2025-11-14 | NCT06478706 |
| Boehringer Ingelheim | BI 1291583 | — | COMPLETED | 2024-10-07 | NCT05865886 |
| Armata Pharmaceuticals, Inc. | AP-PA02 | — | COMPLETED | 2024-07-17 | NCT05616221 |
| Haisco Pharmaceutical Group Co., Ltd. | HSK31858 | — | COMPLETED | 2024-03-29 | NCT05601778 |
| BiomX, Inc. | BX004-A | — | COMPLETED | 2024-03-10 | NCT05010577 |
| Renovion, Inc. | ARINA-1 | — | COMPLETED | 2024-02-01 | NCT05495243 |
| Entero Therapeutics | adrulipase | — | COMPLETED | 2023-06-20 | NCT05719311 |
| Armata Pharmaceuticals, Inc. | AP-PA02 | — | COMPLETED | 2022-12-14 | NCT04596319 |
| Insmed Incorporated | Brensocatib | DPP1 | COMPLETED | 2022-11-01 | NCT05090904 |
| Eloxx Pharmaceuticals, Inc. | ELX-02 | — | COMPLETED | 2022-10-03 | NCT04135495 |
| AbbVie | ABBV-3067 | — | COMPLETED | 2022-06-09 | NCT03969888 |
| Eloxx Pharmaceuticals, Inc. | ELX-02 | — | COMPLETED | 2022-04-06 | NCT04126473 |
| Sanofi | MRT5005 | — | COMPLETED | 2022-03-15 | NCT03375047 |
| Laurent Pharmaceuticals Inc. | LAU-7b | — | COMPLETED | 2021-09-15 | NCT03265288 |
| AzurRx SAS | MS1819-SD | — | COMPLETED | 2021-06-03 | NCT04302662 |
| Entero Therapeutics | MS1819 | — | COMPLETED | 2021-04-08 | NCT04375878 |
| Santhera Pharmaceuticals | POL6014 | — | COMPLETED | 2020-12-30 | NCT03748199 |
| Vertex Pharmaceuticals Incorporated | LUM/IVA | — | COMPLETED | 2020-10-09 | NCT03625466 |
| Vertex Pharmaceuticals Incorporated | VX-561 | — | COMPLETED | 2020-08-20 | NCT03911713 |
| Corbus Pharmaceuticals Inc. | Lenabasum 20 mg | — | COMPLETED | 2020-06-17 | NCT03451045 |
| Proteostasis Therapeutics, Inc. | PTI-808 | — | COMPLETED | 2019-12-23 | NCT03251092 |
| Insmed Incorporated | Brensocatib 10 mg | — | COMPLETED | 2019-12-12 | NCT03218917 |
| Vertex Pharmaceuticals Incorporated | VX-121 | — | COMPLETED | 2019-12-10 | NCT03912233 |
| Entero Therapeutics | MS1819 | — | COMPLETED | 2019-07-13 | NCT03746483 |
| Spyryx Biosciences, Inc. | SPX-101 | — | COMPLETED | 2019-06-20 | NCT03229252 |
| Vertex Pharmaceuticals Incorporated | VX-121 (Suspension) | — | COMPLETED | 2019-05-03 | NCT03768089 |
| Novartis | TIP | — | COMPLETED | 2019-03-20 | NCT02712983 |
| Proteostasis Therapeutics, Inc. | PTI-808 | — | COMPLETED | 2019-03-13 | NCT03500263 |
| Proteostasis Therapeutics, Inc. | PTI-428 | — | COMPLETED | 2019-02-18 | NCT03591094 |
| Vertex Pharmaceuticals Incorporated | Tezacaftor/Ivacaftor | — | COMPLETED | 2018-07 | NCT02730208 |
| Novartis | QBW276 | — | COMPLETED | 2018-04-24 | NCT02566044 |
| Galapagos NV | GLPG2737 | — | COMPLETED | 2018-04-10 | NCT03474042 |
| NovaBiotics Ltd. | Cysteamine | — | COMPLETED | 2018-04 | NCT03000348 |
| Vertex Pharmaceuticals Incorporated | IVA | — | COMPLETED | 2018-03-27 | NCT03227471 |
| Vertex Pharmaceuticals Incorporated | VX-659 | — | COMPLETED | 2018-02-28 | NCT03224351 |
| Vertex Pharmaceuticals Incorporated | VX-152 | — | COMPLETED | 2018-01 | NCT02951195 |
| Proteostasis Therapeutics, Inc. | PTI-428 | — | COMPLETED | 2017-11-28 | NCT02718495 |
| Verona Pharma, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA | RPL554 | — | COMPLETED | 2017-11-03 | NCT02919995 |
| Galapagos NV | GLPG2222 50 mg | — | COMPLETED | 2017-10-19 | NCT03119649 |
| ProQR Therapeutics | QR-010 | — | COMPLETED | 2017-09-14 | NCT02532764 |
| Vertex Pharmaceuticals Incorporated | LUM/IVA | — | COMPLETED | 2017-09-06 | NCT03061331 |
| Parion Sciences | VX-371 + HS | — | COMPLETED | 2017-09 | NCT02709109 |
| Galapagos NV | GLPG2222 150 mg q.d. | — | COMPLETED | 2017-08-11 | NCT03045523 |
| Vertex Pharmaceuticals Incorporated | TEZ | — | COMPLETED | 2017-08 | NCT02951182 |
| Vertex Pharmaceuticals Incorporated | Tezacaftor/Ivacaftor | — | COMPLETED | 2017-06 | NCT02508207 |
| AlgiPharma AS | Alginate oligosaccharide | — | COMPLETED | 2017-03 | NCT02453789 |
| AlgiPharma AS | alginate oligosaccharide | — | COMPLETED | 2017-01 | NCT02157922 |
| Corbus Pharmaceuticals Inc. | JBT-101 (lenabasum) | — | COMPLETED | 2016-12-28 | NCT02465450 |
| Nivalis Therapeutics, Inc. | Cavosonstat | — | COMPLETED | 2016-12 | NCT02589236 |
| Galapagos NV | GLPG1837 dose 1 | — | COMPLETED | 2016-11 | NCT02707562 |
| Galapagos NV | GLPG1837 dose 1 | — | COMPLETED | 2016-08 | NCT02690519 |
| Vertex Pharmaceuticals Incorporated | VX-661 | — | COMPLETED | 2016-05-27 | NCT02070744 |
| Parion Sciences | P-1037 | — | COMPLETED | 2016-02 | NCT02343445 |
| Beyond Air Inc. | Nitric oxide | Vasodilator [EPC] | COMPLETED | 2015-11 | NCT01958944 |
| Syntara | Inhaled Mannitol | — | COMPLETED | 2015-10 | NCT01883531 |
| Abbott | Creon IR | — | COMPLETED | 2015-07 | NCT02415959 |
| Savara Inc. | Vancomycin hydrochloride inhalation powder | — | COMPLETED | 2014-11 | NCT01746095 |
| Humanigen, Inc. | KB001-A | — | COMPLETED | 2014-11 | NCT01695343 |
| BDD Pharma Ltd | Radiolabelled OligoG CF-5/20 DPI | — | COMPLETED | 2014-09 | NCT01991028 |
| Nordmark Arzneimittel GmbH & Co. KG | Burlulipase | — | COMPLETED | 2014-07 | NCT01710644 |
| Vertex Pharmaceuticals Incorporated | Lumacaftor | — | COMPLETED | 2014-04 | NCT01225211 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2014-04 | NCT01685801 |
| Vertex Pharmaceuticals Incorporated | VX-661 | — | COMPLETED | 2014-03 | NCT01531673 |
| Grifols Therapeutics LLC | Alpha-1 HC 100 mg | — | COMPLETED | 2013-10 | NCT01684410 |
| AlgiPharma AS | OligoG CF-5/20 | — | COMPLETED | 2013-10 | NCT01465529 |
| Gilead | Aztreonam for Inhalation Solution (AZLI) | — | COMPLETED | 2013-05 | NCT01375049 |
| Vertex Pharmaceuticals Incorporated | VX-770 | — | COMPLETED | 2013-02 | NCT01161537 |
| Vertex Pharmaceuticals Incorporated | Ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2011-11 | NCT01262352 |
| Aradigm Corporation | Ciprofloxacin for Inhalation | — | COMPLETED | 2011-04 | NCT00889967 |
| Bayer | Ciprofloxacin (Cipro Inhale, BAYQ3939) | — | COMPLETED | 2011-01 | NCT00645788 |
| GSK | SB656933 | — | COMPLETED | 2010-12-29 | NCT00903201 |
| Insmed Incorporated | Arikayce™ | — | COMPLETED | 2010-11-02 | NCT03905642 |
| Merck | denufosol tetrasodium Inhalation Solution | — | COMPLETED | 2010-10 | NCT01181622 |
| Boehringer Ingelheim | Tiotropium bromide 5 mcg | — | COMPLETED | 2010-04 | NCT00737100 |
| Gilead | FTI, AZLI | — | COMPLETED | 2010-02 | NCT00794586 |
| Vertex Pharmaceuticals Incorporated | VX-809 | — | COMPLETED | 2009-12 | NCT00865904 |
| AstraZeneca | AZD9668 | — | COMPLETED | 2009-08 | NCT00757848 |
| Amgen | MP-376 | — | COMPLETED | 2009-06 | NCT00677365 |
| Insmed Incorporated | Arikayce™ 560 mg | — | COMPLETED | 2009-06 | NCT00558844 |
| Swedish Orphan Biovitrum | rhBSSL | — | COMPLETED | 2009-06 | NCT00743483 |
| Humanigen, Inc. | KB001 | — | COMPLETED | 2009-03 | NCT00638365 |
| Sponsor | Asset | MoA | Status | Primary Completion | NCT |
|---|---|---|---|---|---|
| Sionna Therapeutics Inc. | SION-451 | — | RECRUITING | 2026-06 | NCT07035990 |
| Reistone Biopharma Company Limited | RSS0343 Tablets | — | RECRUITING | 2026-09 | NCT07289464 |
| Vertex Pharmaceuticals Incorporated | VX-581 | — | RECRUITING | 2026-11-19 | NCT07283770 |
| Krystal Biotech, Inc. | KB407 (Nebulization) | — | RECRUITING | 2026-12 | NCT05504837 |
| Vertex Pharmaceuticals Incorporated | VX-272 | — | RECRUITING | 2027-03-23 | NCT07437105 |
| Vertex Pharmaceuticals Incorporated | VX-828 | — | RECRUITING | 2026-04-23 | NCT06154447 |
| Cystetic Medicines, Inc. | — | — | RECRUITING | 2025-12 | NCT05802264 |
| Vertex Pharmaceuticals Incorporated | VNZ/TEZ/D-IVA | — | COMPLETED | 2026-02-28 | NCT07349394 |
| Kither Biotech Srl | KIT2014 | — | COMPLETED | 2025-07-14 | NCT06659757 |
| Vertex Pharmaceuticals Incorporated | VX-828 | — | COMPLETED | 2025-05-05 | NCT06861413 |
| Arcturus Therapeutics, Inc. | ARCT-032 | — | COMPLETED | 2024-07-29 | NCT05712538 |
| Vertex Pharmaceuticals Incorporated | VNZ/TEZ/D-IVA | — | COMPLETED | 2024-05-23 | NCT06299709 |
| Vertex Pharmaceuticals Incorporated | VX-118 | — | COMPLETED | 2024-05-04 | NCT06312787 |
| Vertex Pharmaceuticals Incorporated | VNZ/TEZ/D-IVA | — | COMPLETED | 2024-04-16 | NCT06299696 |
| SpliSense Ltd. | SPL84 | — | COMPLETED | 2023-08-06 | NCT06217952 |
| Vertex Pharmaceuticals Incorporated | Vanzacaftor | — | COMPLETED | 2023-06-14 | NCT05867147 |
| Vertex Pharmaceuticals Incorporated | VX-121/TEZ/D-IVA | — | COMPLETED | 2023-03-16 | NCT05437120 |
| CSL Behring | CSL787 | — | COMPLETED | 2023-03-12 | NCT04643587 |
| Vertex Pharmaceuticals Incorporated | VX-121/TEZ/D-IVA | — | COMPLETED | 2022-11-09 | NCT05535959 |
| Nabriva Therapeutics AG | Lefamulin | — | COMPLETED | 2022-09-20 | NCT05225805 |
| Enterprise Therapeutics Ltd | ETD001 single dose | — | COMPLETED | 2022-03-22 | NCT04926701 |
| Calithera Biosciences, Inc | CB-280 | — | COMPLETED | 2021-11-23 | NCT04279769 |
| Chiesi Farmaceutici S.p.A. | CHF 6333 | — | COMPLETED | 2021-03-08 | NCT04010799 |
| Ionis Pharmaceuticals, Inc. | IONIS-ENaCRx | — | COMPLETED | 2020-10-13 | NCT03647228 |
| Neupharma Srl | Teicoplanin Sandoz 200 mg powder and solvent for solution for injection or infusion or oral solution. | — | COMPLETED | 2020-09-16 | NCT04176328 |
| Proteostasis Therapeutics, Inc. | PTI-801 | — | COMPLETED | 2020-02-27 | NCT03140527 |
| Galapagos NV | GLPG2451 dose regimen A | — | COMPLETED | 2019-03-11 | NCT03540524 |
| Proteostasis Therapeutics, Inc. | PTI-428 | — | COMPLETED | 2018-10-03 | NCT03258424 |
| Boehringer Ingelheim | BI 443651 | — | COMPLETED | 2018-08-29 | NCT02976519 |
| Galapagos NV | GLPG3067 single dose | — | COMPLETED | 2018-03-26 | NCT03589313 |
| Galapagos NV | GLPG2737 single dose | — | COMPLETED | 2017-08-16 | NCT03450720 |
| Spyryx Biosciences, Inc. | SPX-101 | — | COMPLETED | 2017-08-02 | NCT03056989 |
| Vertex Pharmaceuticals Incorporated | VX-659 | — | COMPLETED | 2017-08 | NCT03029455 |
| Chiesi Farmaceutici S.p.A. | CHF6333 (Part 1 - SAD) | — | COMPLETED | 2017-07 | NCT03056326 |
| Nivalis Therapeutics, Inc. | Cavosonstat | — | COMPLETED | 2016-12 | NCT02934139 |
| AstraZeneca | AZD5634 for inhalation | — | COMPLETED | 2016-10-24 | NCT02679729 |
| Vertex Pharmaceuticals Incorporated | VX-440 | — | COMPLETED | 2016-09-14 | NCT03486236 |
| ProQR Therapeutics | QR-010 | — | COMPLETED | 2016-09 | NCT02564354 |
| Protalix | AIR DNase | — | COMPLETED | 2016-02 | NCT02605590 |
| Savara Inc. | Molgramostim | — | COMPLETED | 2015-10 | NCT02468908 |
| Nivalis Therapeutics, Inc. | N91115 | — | COMPLETED | 2015-07 | NCT02275936 |
| Nivalis Therapeutics, Inc. | N91115 | — | COMPLETED | 2015-01 | NCT02227888 |
| Nivalis Therapeutics, Inc. | N6022 | — | COMPLETED | 2014-04 | NCT01746784 |
| Vertex Pharmaceuticals Incorporated | Lumacaftor | — | COMPLETED | 2014-03 | NCT01910415 |
| Vertex Pharmaceuticals Incorporated | ivacaftor | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] | COMPLETED | 2014-02 | NCT02015507 |
| Pari Pharma GmbH | Vantobra (tobramycin) | — | COMPLETED | 2013-11 | NCT01953367 |
| Vertex Pharmaceuticals Incorporated | Lumacaftor | — | COMPLETED | 2013-09 | NCT01768663 |
| Vertex Pharmaceuticals Incorporated | lumacaftor | — | COMPLETED | 2013-08 | NCT01899105 |
| Gilead | GS-5737 | — | COMPLETED | 2013-07 | NCT01840735 |
| Pulmatrix Inc. | PUR118 | — | COMPLETED | 2012-12 | NCT01543191 |
| Savara Inc. | AeroVanc | — | COMPLETED | 2012-03 | NCT01537666 |
| Chiesi Farmaceutici S.p.A. | Bramitob® administered by PARI LC® PLUS nebulizer | — | COMPLETED | 2011-10 | NCT01116089 |
| CSL Behring | CR002 Liquid API | — | COMPLETED | 2011-08 | NCT01347190 |
| Vertex Pharmaceuticals Incorporated | VX-809 | — | COMPLETED | 2011-05 | NCT01216046 |
| Vertex Pharmaceuticals Incorporated | VX-770 | — | COMPLETED | 2010-12 | NCT01208285 |
| Vertex Pharmaceuticals Incorporated | VX-770 | — | COMPLETED | 2010-08 | NCT01153542 |
| Vertex Pharmaceuticals Incorporated | VX-770 | — | COMPLETED | 2010-03 | NCT01018368 |
| Vertex Pharmaceuticals Incorporated | VX-770 | — | COMPLETED | 2010-03 | NCT01060566 |
| Gilead | GS-9411 | — | COMPLETED | 2010-02 | NCT00999531 |
| Amgen | MP-376 (Levofloxacin solution for Inhalation) | — | COMPLETED | 2009-12 | NCT00840333 |
| Vertex Pharmaceuticals Incorporated | VX-809 | — | COMPLETED | 2009-12 | NCT00966602 |
| AlgiPharma AS | OligoG CF-5/20 | — | COMPLETED | 2009-10 | NCT00970346 |
| Syntara | Mannitol | Osmotic Diuretic [EPC] | COMPLETED | 2009-09 | NCT00792714 |
| Gilead | GS-9411 | — | COMPLETED | 2009-09 | NCT00951522 |
| Gilead | GS-9411 | — | COMPLETED | 2009-02 | NCT00800579 |
| GSK | SB656933 | — | COMPLETED | 2008-12 | NCT00605761 |
| Boehringer Ingelheim | BI 1323495 | — | TERMINATED | 2021-12-20 | NCT04656275 |
| Alaxia SAS | ALX-009 | — | TERMINATED | 2021-12 | NCT02598999 |
| Synspira, Inc. | Inhaled SNSP113 | — | TERMINATED | 2017-12-18 | NCT03309358 |
| Gilead | GS-5737 | — | TERMINATED | 2013-06 | NCT01793649 |
| Krystal Biotech, Inc. | KB407 (Nebulization) | — | WITHDRAWN | 2024-07-01 | NCT05095246 |
| Gilead | GS-9411 | — | WITHDRAWN | 2010-03 | NCT01025713 |
Novel Drugs by Company (New molecular entities)
Reformulations (8 drugs) Click to expand
Reformulations are FDA-approved versions of existing molecules in new dosage forms (e.g., oral solution vs tablet, extended-release vs immediate-release). They require a new NDA but use an already-proven active ingredient.
Drug Targets
Generic Drugs (3 ANDA approved) Click to expand
Generic drugs contain the same active ingredient as the brand-name drug and are approved via ANDA (Abbreviated New Drug Application).
Drug Categories:
- Novel Drugs: NDA Type 1/2 (new molecular entity) or original BLA
- Reformulations: NDA Type 3/5 (new dosage form of existing molecule)
- Biosimilars: BLA-approved biologics highly similar to reference products
- Generics: ANDA-approved copies of small molecule drugs