Drug discovery
142
drugs
With orphan designations
Overview
Idiopathic pulmonary fibrosis (IPF) is a progressive, fatal interstitial lung disease characterized by irreversible lung scarring (usual interstitial pneumonia pattern) and declining lung function [1][6]. Median survival is 2–5 years post-diagnosis, with respiratory failure as the primary cause of death [6][16]. Diagnosis requires exclusion of secondary causes and confirmation via HRCT or histopathology [6][16].
Categories: rare respiratory diseases, rare transplant-related disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
tazarotene | small molecules | FDA | 2026-06-17 | — | GRI Bio Operations, Inc. |
tadalafil | small molecules | FDA | 2026-03-23 | — | Shenzhen Hanhui Pharmaceutical Technology Co., Ltd |
tranilast lysate | small molecules | FDA | 2026-03-09 | — | Nuformix Technologies Limited |
small molecule inhibitor of ARG-1 and ARG-2 | small molecules | FDA | 2026-03-09 | — | AstraZeneca Pharmaceuticals LP |
deupirfenidone | small molecules | FDA | 2026-02-19 | — | PureTech LYT 100, Inc., a subsidiary of PureTech Health plc |
isoquinoline derivative containing diesters that inhibits BCL-xL | small molecules | FDA | 2026-01-15 | — | Nanjing Reju Therapeutics Co., Ltd. |
Phe-Thr-Thr-Phe-Thr-Val-Thr | peptides | EMA | 2026-01-09 | — | Scendea (NL) B.V. |
Deupirfenidone | small molecules | EMA | 2026-01-09 | — | Granzer Regulatory Consulting & Services GmbH |
humanised monoclonal antibody-drug conjugate delivering an mTOR inhibitor payload | antibodies | FDA | 2025-12-09 | — | Bionevix Ltd |
Adeno-associated virus vector serotype 6.2 containing human TERT gene | gene therapies | EMA | 2025-12-09 | — | Telomere Therapeutics S.L. |
zampilimab | antibodies | FDA | 2025-11-25 | — | Chiesi Farmaceutici S.p.A. |
Orvepitant maleate | small molecules | EMA | 2025-11-21 | — | Granzer Regulatory Consulting & Services GmbH |
kynurenic acid | small molecules | FDA | 2025-11-14 | — | BirchBioMed Inc. |
a humanized anti-S100A4 monoclonal antibody of immunoglobulin G4 isotype | antibodies | FDA | 2025-10-24 | — | Calluna Pharma AS |
roflumilast | small molecules | FDA | 2025-09-03 | — | Transpire Bio Inc |
Admilparant | small molecules | EMA | 2025-08-22 | — | Bristol-Myers Squibb Pharma EEIG |
taladegib | small molecules | FDA | 2025-06-18 | — | Endeavor Biomedicines |
Taladegib | small molecules | EMA | 2025-05-22 | — | Orphix Consulting GmbH |
Tranilast | small molecules | EMA | 2025-05-22 | — | Boyd Consultants Limited |
orvepitant | small molecules | FDA | 2025-04-04 | — | NeRRe Therapeutics Ltd |
selective CXCR7 agonist | small molecules | FDA | 2025-02-12 | — | iLeadBMS Co., Ltd. |
an autologous cell product derived from the human airway basal cells without any gene modification | cell therapies | FDA | 2025-02-04 | — | Regend Therapeutics Co., Ltd. |
artesunate | small molecules | FDA | 2024-11-18 | — | Artasome Therapeutics, LLC |
fluoro methyl gallocatechin gallate | small molecules | FDA | 2024-07-18 | — | Avanti Biosciences, Inc. |
Humanized immunoglobulin G1 monoclonal antibody directed against WNT1-inducible signaling pathway protein-1 | antibodies | FDA | 2024-07-08 | — | Mediar Therapeutics, Inc. |
Nerandomilast | small molecules | EMA | 2024-06-28 | — | Boehringer Ingelheim International GmbH |
Zinc (2R,3S,4R,5R)-2,3,4,5,6-pentahydroxyhexanoate and (3S,9aS)-3-(1H-imidazol-5-ylmethyl)-octahydro-1H-pyrido[1,2-a]piperazine-1,4-dione | small molecules | FDA | 2024-05-30 | — | NovMetaPharma Co., Ltd. |
lixudebart | antibodies | FDA | 2024-05-24 | — | Alentis Therapeutics AG |
2-(Piperazin-1-yl)ethyl 3-fluoro-5-(2-(3-(6-methylpyridin-2-yl)-4-(quinolin-4-yl)-1H-pyrazol-1-yl)acetamido)benzoate | small molecules | FDA | 2024-05-15 | — | AgomAb Spain, S.L. |
immunomodulatory inhibitor of high mobility group box 1 | antibodies | FDA | 2024-02-22 | — | Spark Biopharma, Inc. |
Bersiporocin | small molecules | EMA | 2023-12-13 | — | Propharma Group The Netherlands B.V. |
Pyridine derivative containing pyridazine that inhibits lysophospholipase | small molecules | FDA | 2023-11-08 | — | Boehringer Ingelheim Pharmaceuticals, Inc. |
zelasudil | small molecules | FDA | 2023-08-14 | — | Redx Pharma Plc |
Human Umbilical Cord-derived Mesenchymal Stem Cell Injection | cell therapies | FDA | 2023-08-09 | — | Wuhan Optics Valley Vcanbiopharma Co., Ltd. |
(R)-1-[4'-(5-chloro-3-{[(1-phenylethoxy)carbonyl] amino}thiophen-2-yl)-2'-methoxy-[1,1'-biphenyl]-4-yl]cyclopropanecarboxylic acid | small molecules | FDA | 2023-06-12 | — | HiLung, Inc. |
a humanized, immunoglobulin G subtype 4 anti-Amphiregulin monoclonal antibody | antibodies | FDA | 2023-02-16 | — | Pulmongene (Hong Kong) Co., Limited |
imidazole derivative | small molecules | FDA | 2023-02-01 | — | InSilico Medicine Hong Kong Limited |
Piperidine-containing autotaxin inhibitor | small molecules | FDA | 2023-01-19 | — | NextGen Bioscience |
Ivaltinostat | small molecules | FDA | 2023-01-03 | — | Machaon Biotherapeutics, Inc. |
(H-L-Arginyl-L-Valyl-L-Isoleucyl-L-Arginyl-L-Alanyl-L-Cysteinyl-L-Leucyl-Glycyl-L-Valyl-Glycyl-L-Leucyl-L-Leucyl-Glycyl-L-Asparaginyl-L-Leucyl-D-Alanyl-Glycyl-L-Lysyl-amino-PEG12-propionic acid)2 (Disulfide bond at Cys) | proteins | FDA | 2022-12-13 | — | CohBar, Inc. |
(2S)-4-[2-methoxyethyl-[4-(5,6,7,8-tetrahydro-1,8-naphthyridin-2-yl)butyl]amino]-2-(quinazolin-4-ylamino)butanoic acid | small molecules | EMA | 2022-12-09 | — | Pharma Gateway AB |
ifenprodil | small molecules | FDA | 2022-12-01 | — | Algernon Pharmaceuticals Inc. |
garadacimab | antibodies | FDA | 2022-08-16 | — | CSL Behring |
Ex-vivo conditioned medium from cultured adult human mesenchymal stem cells | other | FDA | 2022-08-15 | — | Summa Bio-Solutions Inc. |
4'-chloro-2'-cyano-N-(trans-4-hydroxy-4-methylcyclohexyl)-biphenyl-4-sulfonamide | small molecules | FDA | 2022-08-15 | — | Modern Biosciences Ltd |
nerandomilast [Jascayd] | small molecules | FDA | 2022-08-15 | 2025-10-07 | Boehringer Ingelheim Pharmaceuticals, Inc. |
Selective Colony Stimulating Factor-1 Receptor kinase inhibitor | antibodies | FDA | 2022-07-27 | — | Elixiron Immunotherapeutics Inc. |
Pirfenidone | small molecules | EMA | 2022-06-24 | — | Regintel Limited |
(6S,9aS)-N-benzyl-6-(4-hydroxybenzyl)-4,7-dioxo-2-((3-(pyridin-2-yl)isoxazol-5-yl)methyl)-8-(quinolin-5-ylmethyl)hexahydro-2H-pyrazino[2,1-c][1,2,4]triazine-1(6H)-carboxamide methane sulfonic acid | small molecules | FDA | 2022-06-07 | — | 3Plus2 Pharma, LLC |
Chimeric peptide of human glucagon-like peptide-1, glucagon and gastric inhibitory polypeptide analogues linked to a human immunoglobulin Fc fragment | peptides | EMA | 2022-05-16 | — | JVM Europe B.V. |
N-(1¿,2-dihydroxy-[1,2¿-binapthalen]-4¿-yl)-4-methoxybenzenesulfonamide | small molecules | FDA | 2022-04-19 | — | Tvardi Therapeutics, Inc. |
Treprostinil sodium | small molecules | EMA | 2022-03-16 | — | Ferrer Internacional S.A. |
((S)-3-(1-Cyclopentyl-5-(2-(trifluoromethyl)phenyl)-1H-pyrazole-3-carboxamido)-5-(3,3-difluoropiperidin-1-yl)pentanoic acid hydrochloride) | small molecules | FDA | 2022-03-08 | — | APIE Therapeutics |
glucagon-like peptide-1/glucagon/gastric inhibitory polypeptide triple agonist linked to human IgG4 Fc fragment | proteins | FDA | 2021-05-10 | — | Hanmi Pharmaceutical Co., Ltd. |
axatilimab | antibodies | FDA | 2021-04-07 | — | Syndax Pharmaceuticals, Inc. |
lithium carbonate | small molecules | FDA | 2021-03-29 | — | Lispiro LLC |
Sodium-3-((2,6-dichloro-7-fluoro-1-(1-propyl-1H-pyrazol-4-yl)-1H-indol-3-yl)thio)-2-fluorobenzoate | small molecules | FDA | 2021-02-23 | — | Blade Therapeutics, Inc. |
Fc-fusion protein comprised of an anti-CXCR4 i-body tethered at its C-terminus to constant domains 2 and 3 of the Fc region of a mutated human IgG1 | proteins | FDA | 2021-02-22 | — | AdAlta Limited |
N-(N-(4-(trifluoromethoxy)phenyl) carbamimidoyl)pyrrolidine-1-carboximidamide acetate (Or N1-(4-trifluoromethoxy)phenyl-N5-pyrrolidine biguanide acetate) | small molecules | FDA | 2021-02-01 | — | ImmunoMet Therapeutics Inc. |
Treprostinil | small molecules | FDA | 2020-12-07 | — | United Therapeutics Corporation |
Caveolin-1 Scaffolding Domain 7-Mer | peptides | FDA | 2020-09-09 | — | Lung Therapeutics, Inc. |
Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-B-D-galactopyranosyl) sulfane | small molecules | FDA | 2020-08-25 | — | Galecto Biotech AB |
Olitigaltin | small molecules | EMA | 2020-08-21 | — | Galecto Biotech AB |
Sodium cromoglicate | small molecules | EMA | 2020-06-26 | — | IQVIA RDS Spain S.L. |
Benzeneacetamide, 2-[2-[2-[[2-methoxy-4-(1-methyl-4-piperidinyl)phenyl]amino]-5-(trifluoromethyl)-4-pyrimidinyl]ethyl]- | small molecules | FDA | 2020-05-18 | — | Amplia Therapeutics |
4-fluoro-5-((6-methylhexahydropyrrolo[3,4-b] pyrrol-5(1H)-yl) sulfonyl) isoquinoline | small molecules | FDA | 2020-02-05 | — | The National Institutes of Pharmaceutical R&D Co., Ltd |
drug component, inhaled nitric oxide, of the inhaled nitric oxice/INOpulse® Device combination product | small molecules | FDA | 2019-09-10 | — | Mallinckrodt Pharmaceuticals Ireland Limited |
(2R,3S)-2-(3-(4,5-dichloro-1H-benzo[d]imidazol-1-yl)propyl)piperidin-3-ol Hydrogen Chloride | small molecules | FDA | 2019-08-05 | — | Daewoong Pharmaceutical Co., Ltd. |
N-(4-amino-3,4-dioxo-1-phenylbutan-2-yl)-4-(2-fluorophenyl)-2-methyloxazole-5-carboxamide | small molecules | FDA | 2019-07-22 | — | Blade Therapeutics, Inc. |
(R)-N-(1-(1-(1H-indazol-5-yl)-3-methyl-2,4-dioxo-1,3,8-triazaspiro[4,5]decan-8-yl)-3-methyl-1-oxobutan-2-yl)-2-fluoro-5-(trifluoromethyl)benzamide | small molecules | FDA | 2019-07-18 | — | X-Rx, Inc. |
(6-(4-((4-(1H-pyrazol-4-yl)phenyl)amino)pyrimidin-2-yl)-1-methyl-1H-indol-2-yl)(3,3-difluoroazetidin-1-yl)methanone | small molecules | FDA | 2019-05-08 | — | Beijing Tide Pharmaceutical Co., Ltd |
cromolyn solution | small molecules | FDA | 2019-04-11 | — | Respivant Sciences, Inc. |
saracatinib | small molecules | FDA | 2019-03-11 | — | AstraZeneca Pharmaceuticals, LP |
5-(4-((2S,5S)-5-(4-chlorobenzyl)-2-methylmorpholino)piperidin-1-yl)-1H-1,2,4-triazol-3-amine | small molecules | FDA | 2019-02-25 | — | Molecure S.A. (previously OncoArendi S.A.) |
Autotaxin inhibitor | small molecules | FDA | 2019-01-14 | — | Bridge Biotherapeutics, Inc |
small interfering RNA agent that targets human transforming growth factor-Eszett1 messenger RNA | RNAs | FDA | 2018-12-19 | — | Toray Industries, Inc. |
15(S)-hydroxy-(5Z,8Z,11Z,13E,17Z)-eicosapentaenoic acid ethyl ester | small molecules | FDA | 2018-12-17 | — | Afimmune |
(1S,3S)-3-((2-methyl-6-(1-methyl-5-(((methyl(propyl)carbamoyl)oxy)methyl)-1H-1,2,3-triazol-4-yl)pyridin-3-yl)oxy)cyclohexane-1-carboxylic acid | small molecules | FDA | 2018-12-17 | — | Bristol-Myers Squibb Company |
5-(3,5-Dimethyl-4-hydroxybenzylamino)-3-(4-morpholin-4-yl-phenylamino)-1H-pyrazole-4-carboxylic acid amide | small molecules | FDA | 2018-12-07 | — | Metagone Biotech Inc. |
(S)-(-)-3-(4-aminophenyl)-2-methoxypropanoic acid | small molecules | EMA | 2018-08-24 | — | Nogra Pharma Limited |
Tilorone | small molecules | EMA | 2018-08-24 | — | Professor Marjukka Myllärniemi |
small molecule inhibitor of integrins avb6 and avb1 | small molecules | FDA | 2018-08-01 | — | Pliant Therapeutics, Inc. |
soidum (5-(3-cyanopyrazolo[1,5-a]pyridin-5-yl)-2-methoxypyridin-3-yl)((2,4-difluorophenyl)sulfonyl)amide | small molecules | FDA | 2018-07-09 | — | Sunshine Lake Pharma Co., Ltd |
small molecule selective inhibitor of c-Jun N-terminal kinase | small molecules | FDA | 2018-06-27 | — | Celgene Corporation |
N-(2-chloro-6-propoxypyridin-4-yl)-2-(2-hydroxyethyl)-2-(4-isopropyl-1,3-dimethyl-1H-pyrazolo[3,4-b]pyridin-6-yl)hydrazinecarboxamide | small molecules | FDA | 2018-05-23 | — | Arroyo Biosciences, LLC |
6-(4-(4-(2,3-Dicholorophenyl) piperazin-1-yl) butoxy)-2H-benzo [b] [1,4] oxazin-3(4H)-one hydrochloride | small molecules | FDA | 2018-04-04 | — | Reviva Pharmaceuticals, Inc. |
plasminogen (Human) | proteins | FDA | 2017-12-14 | — | Kedrion Biopharma Inc. |
yinfenidone | small molecules | FDA | 2017-08-10 | — | Sunshine Lake Pharma Co., Ltd |
2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-a]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile | small molecules | FDA | 2017-06-08 | — | Galapagos NV |
small molecule inhibitor of the Wnt pathway | small molecules | FDA | 2017-06-06 | — | Biosplice Therapeutics, Inc. |
nitric oxide | small molecules | FDA | 2017-01-31 | — | VERO Biotech |
3-[4-(lH-imidazol-l-ylmethyl)phenyl]-5-(2-methylpropyl) thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt | small molecules | FDA | 2017-01-25 | — | Vicore Pharma AB |
human single domain antibody-like protein inhibitor of the chemokine receptor type 4 | antibodies | FDA | 2017-01-11 | — | AdAlta Limited |
Ziritaxestat [GLPG1690] | small molecules | EMA | 2016-08-29 | — | Lakefront Biotherapeutics |
carbon monoxide | small molecules | FDA | 2016-08-16 | — | Proterris, Inc. |
3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt | small molecules | EMA | 2016-07-14 | — | Vicore Pharma AB |
monoclonal antibody targeting eotaxin-2 | antibodies | FDA | 2015-10-29 | — | ChemomAb, Ltd. |
Setogepram sodium [PBI-4050] | small molecules | EMA | 2015-10-09 | — | [INACTIVE] Prometic Pharma SMT B.V. |
tetra-substituted porphyrin derivative containing manganese (III) | small molecules | FDA | 2015-03-16 | — | Aeolus Pharmaceuticals |
lebrikizumab | antibodies | FDA | 2015-03-09 | — | Eli Lilly and Company |
3-pentylbenzenacetic acid sodium salt | small molecules | FDA | 2015-02-11 | — | Liminal BioSciences Limited |
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid | small molecules | EMA | 2014-11-19 | — | Inventiva |
tipelukast | small molecules | FDA | 2014-10-20 | — | MediciNova, Inc. |
pirfenidone | small molecules | FDA | 2014-07-31 | — | Avalyn Pharma, Inc. |
Humanised anti-alpha ν beta 6 monoclonal antibody | antibodies | EMA | 2014-07-29 | — | Biogen Netherlands B.V. |
Nintedanib esylate [Ofev] | small molecules | EMA | 2013-04-26 | — | Boehringer Ingelheim International GmbH |
Tralokinumab [CAT-354] | antibodies | EMA | 2012-11-08 | — | Medimmune Limited |
tralokinumab | antibodies | FDA | 2012-07-24 | — | MedImmune Ltd. |
Recombinant human pentraxin-2 | proteins | EMA | 2012-07-17 | — | Roche Registration GmbH |
pamrevlumab | antibodies | FDA | 2012-07-06 | — | FibroGen, Inc. |
recombinant human Pentraxin-2; recombinant human Serum Amyloid P | proteins | FDA | 2012-02-17 | — | Genentech, Inc. |
Tanzisertib [CC-930] | small molecules | EMA | 2011-12-09 | — | Celgene Europe Limited |
4-[[9-(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol | small molecules | FDA | 2011-09-23 | — | Celgene Corporation |
bispecific antibody targeting interleukin 4 (IL-4) and interleukin 13 (IL-13) | antibodies | FDA | 2011-09-14 | — | Sanofi US Services, Inc., A SANOFI COMPANY |
nintedanib [OFEV] | small molecules | FDA | 2011-06-29 | 2014-10-15 | Boehringer Ingelheim Pharmaceuticals, Inc. |
Recombinant humanized anti-LOXL2 monoclonal antibody | antibodies | FDA | 2011-04-18 | — | Gilead Sciences, Inc. |
(R)-1-phenylethyl-5-(4-biphenyl-4-cyclopropanecarboxylic acid)-3-methylisoxazole-4-yl carbamate sodium salt | small molecules | FDA | 2011-04-15 | — | Bristol-Myers Squibb Company |
Sar9, Met(O2)11-Substance P | proteins | FDA | 2011-03-16 | — | New Amsterdam Sciences |
2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione | small molecules | EMA | 2010-11-26 | — | Calliditas Therapeutics France S.A.S. |
Ambrisentan [ABS-10-001] | small molecules | EMA | 2010-10-01 | — | Gilead Sciences International Limited |
2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione | small molecules | FDA | 2010-09-21 | — | Calliditas Therapeutics AB |
humanized monoclonal antibody against human integrin alphaVbeta6 | antibodies | FDA | 2010-08-05 | — | Biogen Idec, Inc. |
Cintredekin Besudotox | proteins | FDA | 2010-04-30 | — | Insys Development Company, Inc. |
Macitentan [Opsumit] | small molecules | EMA | 2010-01-28 | — | Janssen Cilag International |
ambrisentan | small molecules | FDA | 2009-12-23 | — | Gilead Sciences, Inc. |
purified bovine type collagen | proteins | FDA | 2009-04-27 | — | Magnolia Therapeutics LLC |
macitentan | small molecules | FDA | 2009-04-14 | — | Actelion Pharmaceuticals Ltd |
bosentan | small molecules | FDA | 2008-09-30 | — | Actelion Pharmaceuticals Ltd |
Bosentan monohydrate [Tracleer] | small molecules | EMA | 2008-09-05 | — | [INACTIVE] Actelion Registration Limited |
ammonium tetrathiomolybdate | small molecules | FDA | 2008-05-05 | — | Pipex Pharmaceuticals, Inc. |
Fresolimumab | antibodies | EMA | 2008-04-01 | — | [INACTIVE] Sanofi B.V. |
carlumab | antibodies | FDA | 2007-11-08 | — | Janssen Biotech, Inc. |
Interferon gamma | proteins | EMA | 2007-10-29 | — | mondoBIOTECH Laboratories AG |
Interferon gamma | proteins | FDA | 2007-10-11 | — | mondoBIOTECH Laboratories AG |
Interferon gamma-1B | proteins | EMA | 2005-05-27 | — | Intermune UK Limited |
Acetylcysteine | small molecules | EMA | 2005-01-26 | — | Zambon Group S.p.A. |
Pirfenidone [Esbriet] | small molecules | EMA | 2004-11-16 | — | Roche Registration GmbH |
Heparin sodium [FibroCure] | other | EMA | 2004-09-02 | — | Werner Seeger |
human anti-transforming growth factor-B1,2,3 | antibodies | FDA | 2004-07-09 | — | Genzyme Corporation |
pirfenidone [ESBRIET] | small molecules | FDA | 2004-03-05 | 2014-10-15 | Legacy Pharma Inc. |
interferon gamma-1b | proteins | FDA | 2002-09-12 | — | InterMune, Inc. |
Interferon beta-1a (recombinant human) | proteins | FDA | 1999-01-07 | — | Biogen Idec, Inc. |