Pirfenidone

Summary

Pirfenidone (CHEMBL1256391) is an approved small-molecule non-narcotic analgesic (ATC L04AX05); indicated across 33 conditions including immune system disorder and idiopathic pulmonary fibrosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L04AX05
• Indications: 33 conditions
• Clinical trials: 118
• Chemistry: 185.22 Da · C12H11NO

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1256391
Name	Pirfenidone
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	40632
ChEBI	CHEBI:32016
ATC	L04AX05
Molecular formula	C12H11NO
Molecular weight	185.22
InChIKey	ISWRGOKTTBVCFA-UHFFFAOYSA-N

SMILES: CC1=CN(C(=O)C=C1)C2=CC=CC=C2

IUPAC name: 5-methyl-1-phenylpyridin-2-one

ChEBI definition: A pyridone that is 2-pyridone substituted at positions 1 and 5 by phenyl and methyl groups respectively. An anti-inflammatory drug used for the treatment of idiopathic pulmonary fibrosis.

Pharmacological roles (ChEBI): non-narcotic analgesic, non-steroidal anti-inflammatory drug, antipyretic, antifibrotic agent.

Also known as: AMR-69, Esbriet, NSC-748456, Pirespa, Pirfenidona, Pirfenidone, Pirfenidone component of esbriet, Pirfenidone viatris, SID11113908, SID50104718, SID90341749, SID85231182

Parent form; salt/anhydrous children: CHEMBL5095176

Patent coverage: 4,862 distinct patent families (16,728 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 7 (assay-derived). Sample: Lysine-specific demethylase 4E, RecQ-like DNA helicase BLM, 15-hydroxyprostaglandin dehydrogenase [NAD(+)], Cytochrome P450 1A2, Aldehyde dehydrogenase 1A1, 3-hydroxyacyl-CoA dehydrogenase type-2, Transforming growth factor beta-1 proprotein/Mothers against decapentaplegic homolog 3.

Bioactivity

ChEMBL activities: 3 potent at pChembl ≥ 5 of 10 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
BLM	7.15	Potency	70.8	nM	CHEMBL_ACT_4742031
BLM	7.15	Potency	70.8	nM	CHEMBL_ACT_4917794
P04202	5.84	IC50	1430	nM	CHEMBL_ACT_19102518

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

4 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
immune system disorder	4	MONDO:0005046	EFO:0000540
idiopathic pulmonary fibrosis	4	MONDO:0800504	EFO:0000768
respiratory system disorder	4	MONDO:0005087	EFO:0000684
lung disorder	4	MONDO:0005275	EFO:0003818

27 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
keloid	3	MONDO:0005348	EFO:0004212
pneumonia	3	MONDO:0005249	EFO:0003106
severe acute respiratory syndrome	3	MONDO:0005091	MONDO:0100096
acute respiratory distress syndrome	3	MONDO:0006502	EFO:1000637
pneumoconiosis	3	MONDO:0015926	MONDO:0015926
systemic sclerosis	2	MONDO:0005100	EFO:0000717
interstitial lung disease	2	MONDO:0015925	EFO:0004244
bronchiolitis obliterans syndrome	2	MONDO:0015265	EFO:0007183
hypertrophic cardiomyopathy	2	MONDO:0005045	EFO:0000538
uterine corpus leiomyoma	2	MONDO:0007886	EFO:0000731
focal segmental glomerulosclerosis	2	MONDO:0100313	EFO:0004236
nephrotic syndrome	2	MONDO:0005377	EFO:0004255
proteinuria	2	MONDO:0003634	HP:0000093
pulmonary fibrosis	2	MONDO:0002771	EFO:0009448
burn	2	MONDO:0043519	EFO:0009516
kidney failure	2	MONDO:0001106	EFO:1002048
chronic kidney disease	2	MONDO:0005300	EFO:0003884
heart failure	2	MONDO:0005252	EFO:0003144
hypersensitivity pneumonitis	2	MONDO:0017853	MONDO:0017853
pancreatitis	2	MONDO:0004982	EFO:0000278
neurofibromatosis type 1	2	MONDO:0018975	MONDO:0018975
acute lung injury	2	MONDO:0015796	EFO:0004610
lung neoplasm	2	MONDO:0021117	MONDO:0008903
head and neck squamous cell carcinoma	2	MONDO:0010150	EFO:0000181
acute pancreatitis	2	MONDO:0006515	EFO:1000652
radiation pneumonitis	2	MONDO:0043919	MONDO:0043919
multiple sclerosis	1	MONDO:0005301	MONDO:0005301

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 118.

Phase distribution

Phase	Trials
PHASE2	43
PHASE1	19
PHASE3	18
Not specified	16
PHASE4	11
PHASE1/PHASE2	6
PHASE2/PHASE3	5

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07325357	PHASE4	NOT_YET_RECRUITING	Effect of Pirfenidone on TA Fibrosis
NCT02579603	PHASE4	COMPLETED	Safety, Tolerability and PK of Nintedanib in Combination With Pirfenidone in IPF
NCT02598193	PHASE4	COMPLETED	Safety and Tolerability Study of Pirfenidone in Combination With Nintedanib in Participants With Idiopathic Pulmonary Fibrosis (IPF)
NCT02606877	PHASE4	COMPLETED	A Study to Compare the Amount of Nintedanib and Pirfenidone in the Blood When Nintedanib and Pirfenidone Are Given Separately or in Combination
NCT02821689	PHASE4	UNKNOWN	Pirfenidone in Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis
NCT03260556	PHASE4	UNKNOWN	Pirfenidone for Progressive Fibrotic Sarcoidosis
NCT03939520	PHASE4	COMPLETED	Management of Progressive Disease in Idiopathic Pulmonary Fibrosis
NCT04856111	PHASE4	UNKNOWN	Pirfenidone vs. Nintedanib for Fibrotic Lung Disease After Coronavirus Disease-19 Pneumonia
NCT04928586	PHASE4	UNKNOWN	Immunosuppressant Combined With Pirfenidone in CTD-ILD
NCT05505409	PHASE4	UNKNOWN	Efficacy and Safety of Pirfenidone in CTD-ILD
NCT06224790	PHASE4	COMPLETED	Perfenidone in Type 2 Diabetic Patients With Diabetic Neuropathy
NCT05075161	PHASE3	RECRUITING	Pirfenidone to Prevent Fibrosis in Ards.
NCT05288179	PHASE3	RECRUITING	Efficacy and Safety of Pirfenidone Capsules in the Treatment of Pneumoconiosis
NCT06928272	PHASE3	RECRUITING	Long Covid (LC)-REVITALIZE - A Long Covid Repurposed Drug Study
NCT07082842	PHASE3	RECRUITING	Confirmatory Clinical Study of HEC585 Tablets in Patients With IPF
NCT07284602	PHASE3	NOT_YET_RECRUITING	Trial to Evaluate the Efficacy and Safety of LYT-100 (Deupirfenidone) Compared to Pirfenidone in Adults With Idiopathic Pulmonary Fibrosis (IPF)
NCT07388680	PHASE2/PHASE3	RECRUITING	Pirfenidone Capsules in the Treatment of Radiation-induced Lung Injury With or Without Immune Pneumonia
NCT00287716	PHASE3	COMPLETED	Three-Arm Study of the Safety and Efficacy of Pirfenidone in Patients With Idiopathic Pulmonary Fibrosis
NCT00287729	PHASE3	COMPLETED	Safety and Efficacy of Pirfenidone in Patients With Idiopathic Pulmonary Fibrosis
NCT00662038	PHASE3	COMPLETED	Open-Label Study of the Long Term Safety of Pirfenidone in Patients With Idiopathic Pulmonary Fibrosis (IPF)
NCT01366209	PHASE3	COMPLETED	Efficacy and Safety of Pirfenidone in Patients With Idiopathic Pulmonary Fibrosis (IPF)
NCT02222376	PHASE3	COMPLETED	Effect of Topic Pirfenidone in Diabetic Ulcers
NCT02262299	PHASE2/PHASE3	COMPLETED	European Trial of Pirfenidone in BOS, A European Multi-center Study
NCT02496182	PHASE2/PHASE3	UNKNOWN	Pirfenidone in the Chronic Hypersensitivity Pneumonitis Treatment
NCT02689778	PHASE3	COMPLETED	Effect of Pirfenidone on Glomerular Filtration Rate and Albuminuria in Patients With Diabetic Nephropathy
NCT02823236	PHASE3	UNKNOWN	Efficacy of Intralesional Triamcinolone and 8% Topical Pirfenidone for Treatment of Keloid Scars
NCT03068234	PHASE2/PHASE3	UNKNOWN	Pirfenidone as Treatment of Skin Fibrosis in Systemic Sclerosis
NCT03208933	PHASE3	COMPLETED	Open-label Study to Assess the Effectiveness of Pirfenidone in Participants With Idiopathic Pulmonary Fibrosis (IPF).
NCT03856853	PHASE3	UNKNOWN	Efficacy and Safety of Pirfenidone in Patient With Systemic Sclerosis-associated Interstitial Lung Disease
NCT03857854	PHASE3	UNKNOWN	Efficacy and Safety of Pirfenidone in Patient With Dermatomyositis Interstitial Lung Disease (Dm-ILD)
NCT04282902	PHASE3	UNKNOWN	A Study to Evaluate the Efficacy and Safety of Pirfenidone With Novel Coronavirus Infection
NCT05713292	PHASE3	COMPLETED	Pirfenidone in Adult Hospitalized Patients With COVID-19
NCT06585319	PHASE2/PHASE3	COMPLETED	Pirfenidone LP or Collagen-polyvinylpyrrolidone in COVID-19
NCT06909812	PHASE3	COMPLETED	Silicone vs Pirfenidone in the Treatment of Hypertrophic Scars and Keloids
NCT03109288	PHASE1/PHASE2	RECRUITING	Targeting Residual Activity By Precision, Biomarker-Guided Combination Therapies of Multiple Sclerosis (TRAP-MS)
NCT05060822	PHASE2	ACTIVE_NOT_RECRUITING	Phase ll Study of HEC585 in Patients With IPF
NCT05321420	PHASE2	ACTIVE_NOT_RECRUITING	LYT-100 in Patients With Idiopathic Pulmonary Fibrosis (IPF)
NCT05350371	PHASE1/PHASE2	RECRUITING	Safety and Tolerability of Pirfenidone in Acute Pancreatitis
NCT05704166	PHASE2	RECRUITING	PirfenidoneVsPlacebo as Prophylaxis Against Acute Radiation-induced Lung Injury Following HFRT in Breast Cancer Patients
NCT06241560	PHASE2	RECRUITING	A Study in People With Idiopathic Pulmonary Fibrosis to Test Whether Pirfenidone Influences the Amount of BI 1015550 in the Blood
NCT06253117	PHASE2	RECRUITING	Evaluation of Pirfenidone as a Novel Therapeutic Strategy Against Recurrent Acute Pancreatitis.
NCT06484153	PHASE1/PHASE2	NOT_YET_RECRUITING	Fruquintinib and Pirfenidone in Combination With Anti-PD-1 Antibody in Advanced or Metastatic pMMR/MSS Colorectal Carcinoma
NCT00001596	PHASE2	COMPLETED	Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
NCT00001959	PHASE2	COMPLETED	Pirfenidone to Treat Kidney Disease (Focal Segmental Glomerulosclerosis)
NCT00011076	PHASE2	COMPLETED	Pirfenidone to Treat Hypertrophic Cardiomyopathy
NCT00063583	PHASE1/PHASE2	COMPLETED	Pirfenidone: A New Drug to Treat Kidney Disease in Patients With Diabetes
NCT00076102	PHASE2	COMPLETED	Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas
NCT00080223	PHASE2	COMPLETED	Safety Study of Oral Pirfenidone in Patients With Pulmonary Fibrosis/Idiopathic Pulmonary Fibrosis
NCT00332033	PHASE2	COMPLETED	Development of a Non-Invasive Treatment for Uterine Leiomyoma (Fibroids)
NCT00754780	PHASE2	COMPLETED	Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
NCT01504334	PHASE2	UNKNOWN	Safety and Efficacy Study of Pirfenidone to Treat Idiopathic Pulmonary Fibrosis(IPF)
NCT01872689	PHASE2	COMPLETED	A Study of Lebrikizumab in Participants With Idiopathic Pulmonary Fibrosis (IPF)
NCT01890265	PHASE2	COMPLETED	Evaluate the Safety and Efficacy of FG-3019 (Pamrevlumab) in Participants With Idiopathic Pulmonary Fibrosis (IPF)
NCT01933334	PHASE2	COMPLETED	Safety and Tolerability of Pirfenidone in Participants With Systemic Sclerosis-Related Interstitial Lung Disease (SSc-ILD) (LOTUSS)
NCT02136992	PHASE2	COMPLETED	Clinical Efficacy and Safety of Pirfenidone in Patients With Idiopathic Pulmonary Fibrosis
NCT02161952	PHASE2	COMPLETED	Pirfenidone, an Antifibrotic and Antiinflammatory Drug
NCT02296281	PHASE2	UNKNOWN	Safety and Efficacy Study of Pirfenidone to Treat Grade 2 or Above Radiation-induced Lung Injury
NCT02408744	PHASE1/PHASE2	COMPLETED	Utility of Prolonged-release Pirfenidone in the Progression of Chronic Kidney Disease
NCT02707640	PHASE2	COMPLETED	A Study to Assess the Safety and Tolerability of N-Acetylcysteine When Administered With Pirfenidone to Participants With Idiopathic Pulmonary Fibrosis (IPF)
NCT02808871	PHASE2	COMPLETED	Phase ll Study of Pirfenidone in Patients With RAILD (TRAIL1)
NCT02932566	PHASE2	COMPLETED	The Efficacy and Safety of Pirfenidone in Patients With Heart Failure and Preserved Left Ventricular Ejection Fraction
NCT02951429	PHASE2	COMPLETED	Efficacy, Safety, and Tolerability Study of Pirfenidone in Combination With Sildenafil in Participants With Advanced Idiopathic Pulmonary Fibrosis (IPF) and Intermediate or High Probability of Group 3 Pulmonary Hypertension
NCT02958917	PHASE2	TERMINATED	Study of Efficacy and Safety of Pirfenidone in Patients With Fibrotic Hypersensitivity Pneumonitis
NCT03099187	PHASE2	COMPLETED	A Study of Pirfenidone in Patients With Unclassifiable Progressive Fibrosing Interstitial Lung Disease
NCT03221257	PHASE2	COMPLETED	Scleroderma Lung Study III - Combining Pirfenidone With Mycophenolate
NCT03359863	PHASE2	COMPLETED	Pirfenidone for Restrictive Chronic Lung Allograft Dysfunction
NCT03385668	PHASE2	COMPLETED	Pilot Study of Pirfenidone in Pulmonary Fibrosis With Anti-myeloperoxydase Antibodies
NCT03473340	PHASE2	TERMINATED	Studying the Treatment Effect of Pirfenidone in Chronic Lung Allograft Dysfunction (STOP-CLAD)
NCT03530150	PHASE2	COMPLETED	Pirfenidone and Its Role in Burn Wound Healing
NCT03902509	PHASE2	COMPLETED	A Study About Safety and Efficacy of Pirfenidone to Treat Grade 2 or Grade3 Radiation-induced Lung Injury.
NCT04099407	PHASE2	COMPLETED	Pirfenidone and Advanced Liver Fibrosis.
NCT04193592	PHASE2	UNKNOWN	Efficacy and Safety of Pirfenidone Treatment in HPS-ILD
NCT04258397	PHASE2	UNKNOWN	Trial of Pirfenidone to Prevent Progression in Chronic Kidney Disease
NCT04461587	PHASE2	COMPLETED	Examination of Pirfenidone (Esbriet®) Therapy in Coal Workers’ Pneumoconiosis With Pulmonary Fibrosis
NCT04607928	PHASE2	UNKNOWN	Pirfenidone Compared to Placebo in Post-COVID19 Pulmonary Fibrosis COVID-19
NCT04675619	PHASE2	COMPLETED	Evaluation of the Efficacy of Pirfenidone in Progressive Chronic Hypersensitivity Pneumonitis
NCT05118256	PHASE2	UNKNOWN	Pirfenidone for the Reduction of Metabolic, Inflammatory and Fibrogenic Activity in Complicated Silicosis
NCT05531955	PHASE2	UNKNOWN	Pirfenidone Treat Myocardial Fibrosis After Acute Myocardial Infarction
NCT05542615	PHASE2	UNKNOWN	Prolonged Release Pirfenidone for Advanced Residual Liver Fibrosis (MINERVA).
NCT05801133	PHASE2	UNKNOWN	Pifenidone Prophylactic Therapy for Radiation Lung Injury in Patients Who Have Previously Received Immune Checkpoint Inhibitors
NCT06142318	PHASE2	UNKNOWN	Pirfenidone as a Radiosensitizer in the Treatment of Head and Neck Squamous Cell Carcinoma
NCT06267794	PHASE2	COMPLETED	Prolonged Release Pirfenidone Versus Placebo in Compensated Cirrhosis.
NCT06871904	PHASE1/PHASE2	COMPLETED	Fibrosis-Modulating Effects of Metformin and Pirfenidone in Oral Submucous Fibrosis
NCT07229716	PHASE1	RECRUITING	Study on the Drug Interactions of HRS-9813, Pirfenidone and Nintedanib in Healthy Subjects
NCT07454291	PHASE1	RECRUITING	A Study to Evaluate Pharmacokinetics and Drug-drug Interactions of ENV-101 (Taladegib) in Healthy Participants
NCT00053937	PHASE1	COMPLETED	Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
NCT02525484	PHASE1	COMPLETED	A Bioequivalence Study Comparing Pirfenidone Tablet and Capsule Dosage Forms in Healthy Adult Participants
NCT02648048	PHASE1	COMPLETED	A Study of Oral Vismodegib in Combination With Pirfenidone in Participants With Idiopathic Pulmonary Fibrosis
NCT03177291	PHASE1	COMPLETED	Pirfenidone Combined With Standard First-Line Chemotherapy in Advanced-Stage Lung NSCLC
NCT03981094	PHASE1	COMPLETED	A Study of the Pharmacokinetic Interaction Between Pirfenidone and BMS-986278 in Healthy Participants
NCT04126538	PHASE1	UNKNOWN	Pirfenidone Capsule in Patients With Chronic Kidney Disease G2 and G3a Study on Safety and Pharmacokinetics
NCT04888715	PHASE1	COMPLETED	To Evaluate Drug-drug Interactions Between DWN12088 and Pirfenidone or Nintedanib in Healthy Volunteers
NCT04971746	PHASE1	COMPLETED	Drug-drug Interaction Study with GLPG4716 and Nintedanib and Pirfenidone in Healthy Subjects
NCT05280873	PHASE1	UNKNOWN	Pirfenidone Combined With Methylprednisolone Versus Methylprednisolone in the Treatment of CIP
NCT05383131	PHASE1	COMPLETED	To Evaluate Drug-drug Interactions Between HEC585 and Pirfenidone or Nintedanib in Healthy Volunteers
NCT06070610	PHASE1	COMPLETED	A Study in Healthy Men to Test Whether BI 1015550 Influences the Amount of Nintedanib and Pirfenidone in the Blood
NCT06569381	PHASE1	COMPLETED	Comparative Pharmacokinetic Study of Pirfenidone Modified-Release Tablets in Healthy Subjects Under Fasting and Fed Conditions
NCT06588517	PHASE1	COMPLETED	Comparative Pharmacokinetic(PK) Study of Pirfenidone Modified-Release Tablets and Pirfenidone Tablets in Healthy Subjects Under Fed Conditions
NCT06589921	PHASE1	COMPLETED	Comparative Pharmacokinetic Study of Pirfenidone Modified-Release Tablets and Pirfenidone Tablets in Healthy Subjects Under Fed Conditions
NCT07015398	PHASE1	COMPLETED	A Study of the Pharmacokinetic Interaction Between Pirfenidone, Nintedanib, and Nalbuphine Extended Release (NAL ER) in Healthy Participants

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 2 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).