Isotretinoin

drug
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Also known as 13 cis-retinoic acidAbsoricaAbsorica ldAccutaneAmnesteemClaravisIsotretinoinaIsotretinoineIsotrexMyorisanNSC-758156PAT-001RO-43780RoaccutaneSotretZenataneSID26753642SID26753643SID50106981

Summary

Isotretinoin (CHEMBL547) is an approved small-molecule keratolytic drug (ATC D10BA01); indicated across 51 conditions including acne and neuroblastoma.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: D10BA01 (+2 more)
  • Indications: 51 conditions
  • Clinical trials: 137
  • Chemistry: 300.4 Da · C20H28O2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL547
NameIsotretinoin
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID5282379
ChEBICHEBI:6067
ATCD10BA01, D10AD54, D10AD04
Molecular formulaC20H28O2
Molecular weight300.4
InChIKeySHGAZHPCJJPHSC-XFYACQKRSA-N

SMILES: CC1=C(C(CCC1)(C)C)/C=C/C(=C/C=C/C(=C\C(=O)O)/C)/C

IUPAC name: (2Z,4E,6E,8E)-3,7-dimethyl-9-(2,6,6-trimethylcyclohexen-1-yl)nona-2,4,6,8-tetraenoic acid

ChEBI definition: A retinoic acid that is all-trans-retinoic acid in which the double bond which is α,β- to the carboxy group is isomerised to Z configuration. A synthetic retinoid, it is used for the treatment of severe cases of acne and other skin diseases.

Pharmacological roles (ChEBI): keratolytic drug, antineoplastic agent, teratogenic agent.

Also known as: 13 cis-retinoic acid, Absorica, Absorica ld, Accutane, Amnesteem, Claravis, Isotretinoin, Isotretinoina, Isotretinoine, Isotrex, Myorisan, NSC-758156

Patent coverage: 8,634 distinct patent families (31,016 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: 17 (assay-derived). Sample: Microtubule-associated protein tau, RecQ-like DNA helicase BLM, Ferritin light chain, Peripheral myelin protein 22, 5-hydroxytryptamine receptor 2B, Alpha-2B adrenergic receptor, Thyroid hormone receptor beta, Prostaglandin G/H synthase 1, Adenosine receptor A3, Mitogen-activated protein kinase 14, Serine/threonine-protein kinase mTOR, Cruzipain, Aldehyde dehydrogenase 1A1, Mitogen-activated protein kinase 1, Olfactory receptor 51E2, Cellular retinoic acid-binding protein 1, Bile salt export pump.

Bioactivity

ChEMBL activities: 14 potent at pChembl ≥ 5 of 27 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
OR51E26.8IC50160nMCHEMBL_ACT_19491341
MAPK16.33IC50462nMCHEMBL_ACT_7750438
HTR2B6.27Ki540nMCHEMBL_ACT_7750463
HTR2B6.07IC50848nMCHEMBL_ACT_7750462
P402206.05IC50900nMCHEMBL_ACT_230760
P402206.05IC50900nMCHEMBL_ACT_875651
ADORA35.63Ki2357nMCHEMBL_ACT_7780332
PMP225.62Potency2393nMCHEMBL_ACT_4358326
MAPK145.6IC502529nMCHEMBL_ACT_7750440
ADRA2B5.52Ki3019nMCHEMBL_ACT_7780344
ADORA35.38IC504169nMCHEMBL_ACT_7780331
MTOR5.33Potency4645nMCHEMBL_ACT_4522703
PTGS15.3AC504968nMCHEMBL_ACT_25205281
ADRA2B5.18IC506612nMCHEMBL_ACT_7780343

Target pathways

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

Indications & clinical

Indications

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

IndicationPhaseMONDOEFO
acne4MONDO:0011438EFO:0003894

43 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)PhaseMONDOEFO
neuroblastoma3MONDO:0005072EFO:0000621
rosacea3MONDO:0006604EFO:1000760
medulloblastoma3MONDO:0007959EFO:0002939
head and neck cancer3MONDO:0005627EFO:0006859
ganglioneuroblastoma3MONDO:0005035EFO:0000502
brain neoplasm3MONDO:0021211EFO:0003833
central nervous system neoplasm3MONDO:0006130EFO:1000158
dysplasia of cervix3MONDO:0006736EFO:1000910
kidney cancer3MONDO:0002367MONDO:0002367
lung neoplasm3MONDO:0021117MONDO:0008903
severe acute respiratory syndrome3MONDO:0005091MONDO:0100096
breast neoplasm3MONDO:0021100MONDO:0007254
actinic keratosis2MONDO:0005173EFO:0002496
HIV infectious disease2MONDO:0005109EFO:0000180
glioblastoma2MONDO:0018177EFO:0000519
leukemia2MONDO:0005059EFO:0000565
plasma cell myeloma2MONDO:0009693EFO:0001378
focal segmental glomerulosclerosis2MONDO:0100313EFO:0004236
juvenile myelomonocytic leukemia2MONDO:0011908EFO:1000309
mycosis fungoides2MONDO:0009691EFO:1001051
tinea pedis2MONDO:0005984EFO:0007512
spermatogenic failure2MONDO:0004983EFO:0000279
pulmonary emphysema2MONDO:0004849EFO:0000464
Klinefelter syndrome2MONDO:0006823EFO:1001006
ichthyosis2MONDO:0019269MONDO:0019269
lymphoma2MONDO:0005062EFO:0000574
Hodgkins lymphoma1MONDO:0004952EFO:0000183
myelodysplastic syndrome1MONDO:0018881EFO:0000198
acute myeloid leukemia1MONDO:0018874EFO:0000222
acute promyelocytic leukemia1MONDO:0012883EFO:0000224
Burkitt lymphoma1MONDO:0007243EFO:0000309
chronic myeloid leukemia1MONDO:0011996EFO:0000339
diffuse large B-cell lymphoma1MONDO:0018905EFO:0000403
neoplasm1MONDO:0005070EFO:0000616
renal cell carcinoma1MONDO:0005086EFO:0000681
anaplastic astrocytoma1MONDO:0016684EFO:0002499
anaplastic oligodendroglioma1MONDO:0016696EFO:0002501
epidermolysis bullosa1MONDO:0006541EFO:1000690
gliosarcoma1MONDO:0016681EFO:1001465
glioma1MONDO:0021042MONDO:0003268
Alzheimer disease1MONDO:0004975MONDO:0004975
Wilms tumor1MONDO:0006058MONDO:0006058
oligodendroglioma1MONDO:0016695MONDO:0002543

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

Clinical trials

Total trials: 137.

Phase distribution

PhaseTrials
PHASE241
PHASE325
PHASE125
Not specified21
PHASE414
PHASE1/PHASE28
EARLY_PHASE13

Top trials by phase / activity

NCTPhaseStatusTitle
NCT06698263PHASE4NOT_YET_RECRUITING13-cis Retinoic Acid (Isotretinoin) and Sperm Production
NCT01139749PHASE4UNKNOWNEfficacy and Safety of Low-dose Oral Isotretinoin for Seborrhea
NCT01404130PHASE4COMPLETEDA Pilot Study of the Effects of Isotretinoin on Cognition, Learning and Memory
NCT01445028PHASE4COMPLETEDIsotretinoin for Proliferative Vitreoretinopathy
NCT02457520PHASE4COMPLETEDABSORICA in Patients With Severe Recalcitrant Nodular Acne
NCT02855138PHASE4COMPLETEDEffects of Isotretinoin on The Gonads and Hirsutism
NCT03076021PHASE4COMPLETEDEffects of Isotretinoin on CYP2D6 Activity
NCT04940767PHASE4COMPLETEDA Companion Treatment Study for Patients With Moderate to Severe Acne Vulgaris
NCT05218486PHASE4UNKNOWNEffect of Oral Isotretinoin on The Level of SerumYKL40 in Acne Vulgaris Patients
NCT05316675PHASE4UNKNOWNImpact of Isotretinoin in Different Doses on Quality of Life
NCT05529888PHASE4COMPLETEDMeasure the Serum Level of Homocysteine in Acne Patients Before and After Oral Isotretinoin.
NCT05666765PHASE4UNKNOWNComparison Between Isotretinoin, Silymarin and Both in the Treatment of Acne Vulgaris
NCT06873087PHASE4COMPLETEDChanges in PACAP, CGRP, and VIP Levels in Rosacea Patients on Systemic Isotretinoin and Their Association with Erythema
NCT07228481PHASE4COMPLETEDStudy of Two Dosages of Roaccutane in Cystic Acne
NCT00392327PHASE3ACTIVE_NOT_RECRUITINGChemotherapy and Radiation Therapy in Treating Young Patients With Newly Diagnosed, Previously Untreated, High-Risk Medulloblastoma/PNET
NCT03126916PHASE3RECRUITINGTesting the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)
NCT06172296PHASE3RECRUITINGDinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
NCT06447480PHASE3RECRUITINGClinical Trial to Compare Oral Isotretinoin to Standard of Care in Moderate Acne Skin of Color Patients
NCT00001073PHASE3COMPLETEDSafety and Effectiveness of Giving Isotretinoin to HIV-Infected Women to Treat Cervical Tumors
NCT00002737PHASE3COMPLETEDInterferon Alfa With or Without Isotretinoin in Treating Patients With Metastatic Kidney Cancer
NCT00003564PHASE3WITHDRAWNProcarbazine and Isotretinoin in Treating Patients With Recurrent Primary Malignant Gliomas
NCT00003599PHASE3WITHDRAWNVitamin E in Preventing the Side Effects of Isotretinoin in Former and Current Smokers Who Are Receiving Isotretinoin to Prevent Lung Cancer
NCT00004188PHASE3COMPLETEDCombination Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Neuroblastoma
NCT00026312PHASE3COMPLETEDIsotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma
NCT00030719PHASE3UNKNOWNCombination Chemotherapy With or Without Filgrastim Before Surgery, High-Dose Chemotherapy, and Radiation Therapy Followed by Isotretinoin With or Without Monoclonal Antibody in Treating Patients With Neuroblastoma
NCT00054561PHASE3COMPLETEDIsotretinoin, Interferon Alfa, and Vitamin E in Treating Patients With Stage III or Stage IV Head and Neck Cancer
NCT00078988PHASE3COMPLETEDHigh-Dose Chemotherapy Plus Autologous Stem Cell Transplantation Compared With Intermediate-Dose Chemotherapy Plus Autologous Stem Cell Transplantation With or Without Isotretinoin in Treating Young Patients With Recurrent High-Grade Gliomas
NCT00410631PHASE3UNKNOWNObservation, Combination Chemotherapy, Radiation Therapy, and/or Autologous Stem Cell Transplant in Treating Young Patients With Neuroblastoma
NCT00499616PHASE3COMPLETEDCombination Chemotherapy and Surgery With or Without Isotretinoin in Treating Young Patients With Neuroblastoma
NCT00567567PHASE3COMPLETEDComparing Two Different Myeloablation Therapies in Treating Young Patients Who Are Undergoing a Stem Cell Transplant for High-Risk Neuroblastoma
NCT00882531PHASE3COMPLETEDIsotretinoin in Papular-Pustular Rosacea
NCT00975143PHASE3COMPLETEDEfficacy and Safety of CIP-Isotretinoin in Patients With Severe Recalcitrant Nodular Acne
NCT01041638PHASE3COMPLETEDMonoclonal Antibody Ch14.18, Sargramostim, Aldesleukin, and Isotretinoin After Autologous Stem Cell Transplant in Treating Patients With Neuroblastoma
NCT01474590PHASE3COMPLETEDEfficacy & Safety Comparison of Epiduo With Doxycycline Versus Vehicle With Isotretinoin in the Treatment of Severe Acne
NCT03370367PHASE3COMPLETEDDouble Blind Phase III Trial of Effects of Low Dose 13-Cisretinoic Acid on Prevention of Second Primaries in Stages I-II Head and Neck Cancer
NCT04290572PHASE3UNKNOWNEfficacy and Safety of Oral Isotretinoin for the Treatment of Facial Recalcitrant Flat Warts
NCT04353180PHASE3UNKNOWNAssessment the Activity Value of Isotretinoin (13- Cis-Retinoic Acid ) in the Treatment of COVID-19 ( Isotretinoin in Treatment of COVID-19) (Randomized)
NCT06242288PHASE3COMPLETEDCorrelation Between Depression Scores and Serum NF-ĸB/NLRP3 Axis, Biotinidase, and HMGB After Treatment With Isotretinoin in Patients With Acne Vulgaris
NCT06451237PHASE3COMPLETEDAcne Remission Maintenance by Weekend Systemic Isotretinoin
NCT03786783PHASE2ACTIVE_NOT_RECRUITINGDinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma
NCT04385277PHASE2ACTIVE_NOT_RECRUITINGTreatment With Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide After Intensive Therapy for People With High-Risk Neuroblastoma (NBL)
NCT07481370PHASE2ENROLLING_BY_INVITATIONIsotretinoin vs hCG for Male Infertility Due to Low or Absent Sperm
NCT00002506PHASE2COMPLETEDIsotretinoin Plus Interferon in Treating Patients With Recurrent Cancer
NCT00002586PHASE2COMPLETED13-Cis Retinoic Acid With or Without Vitamin E for Prevention of Lung Cancer
NCT00003405PHASE2WITHDRAWNCombination Chemotherapy Plus Biological Therapy in Treating Patients With Acute Myelogenous Leukemia
NCT00003585PHASE1/PHASE2COMPLETEDBiological Therapy Plus Chemotherapy in Treating Patients With Metastatic or Recurrent Kidney Cancer
NCT00003619PHASE1/PHASE2COMPLETEDCombination Chemotherapy Followed By Peripheral Stem Cell Transplantation or Isotretinoin in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Acute Lymphocytic Leukemia
NCT00004110PHASE2COMPLETEDMonoclonal Antibody Therapy Plus Etoposide in Treating Patients With Neuroblastoma
NCT00005847PHASE2COMPLETEDChemotherapy With or Without Biological Therapy in Treating Patients With Metastatic Prostate Cancer That Has Not Responded to Hormone Therapy
NCT00017368PHASE2COMPLETEDCombination Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Neuroblastoma
NCT00021359PHASE2COMPLETEDIsotretinoin Plus Dexamethasone in Treating Patients With Multiple Myeloma
NCT00025038PHASE2COMPLETEDCombination Chemotherapy Followed By Donor Bone Marrow or Umbilical Cord Blood Transplant in Treating Children With Newly Diagnosed Juvenile Myelomonocytic Leukemia
NCT00038376PHASE2COMPLETEDPhase II Study Of Roferon and Accutane For Patients With T-Cell Malignancies
NCT00040872PHASE2COMPLETEDMultiple Therapies in Treating Patients With Advanced Neuroblastoma
NCT00062010PHASE2COMPLETEDInterferon Alfa, Isotretinoin, and Paclitaxel in Treating Patients With Recurrent Small Cell Lung Cancer
NCT00089258PHASE2COMPLETEDBiological Therapy in Treating Patients With Neuroblastoma That Has Not Responded to Previous Treatment
NCT00098020PHASE2COMPLETEDPodocyte Retinoids
NCT00112502PHASE2COMPLETEDTemozolomide Alone or in Combination With Thalidomide and/or Isotretinoin and/or Celecoxib in Treating Patients Who Have Undergone Radiation Therapy for Glioblastoma Multiforme
NCT00135135PHASE2COMPLETEDTherapy for Children With Neuroblastoma
NCT00138151PHASE2TERMINATEDIsotretinoin, Interferon Alpha-2b, and Paclitaxel in Stage IV, Recurrent, or Persistent Cervical Cancer
NCT00176527PHASE2TERMINATEDIsotretinoin, Interferon Alfa-2b, Docetaxel, and Estramustine in Treating Patients With Metastatic Prostate Cancer That Did Not Respond to Hormone Therapy
NCT00324740PHASE1/PHASE2TERMINATEDVorinostat and Isotretinoin in Treating Patients With Advanced Kidney Cancer
NCT00528437PHASE2COMPLETEDTemozolomide,Thiotepa and Carboplatin With Autologous Stem Cell Rescue Followed by 13-cis-retinoic Acid in Patients With Recurrent/Refractory Malignant Brain Tumors
NCT00555399PHASE1/PHASE2TERMINATEDVorinostat, Isotretinoin and Temozolomide in Adults With Recurrent Glioblastoma Multiforme (GBM)
NCT00701740PHASE2COMPLETEDOral Isotretinoin for Photoaging: Results of a Randomized Controlled Phase II Trial
NCT00808899PHASE2TERMINATEDNeuroblastoma Protocol 2008: Therapy for Children With Advanced Stage High Risk Neuroblastoma
NCT00842907PHASE2UNKNOWNEfficacy and Safety of Oral Isotretinoin for Cutaneous Photodamage
NCT00969722PHASE2TERMINATEDA Study of MAb-3F8 Plus Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Versus 13-cis-Retinoic Acid (RA) Plus GM-CSF in Primary Refractory Neuroblastoma Patients
NCT01183416PHASE2COMPLETEDHigh-Dose 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Myeloablative Therapy and Autologous Stem-Cell Transplantation
NCT01183429PHASE2COMPLETED3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Non-Myeloablative Therapy in Patients With High-Risk Neuroblastoma
NCT01334515PHASE2COMPLETEDBiological Therapy, Sargramostim, and Isotretinoin in Treating Patients With Relapsed or Refractory Neuroblastoma
NCT01560585PHASE1/PHASE2TERMINATEDOpen Label Study of Isotretinoin in Mild to Moderate Alzheimer’s Disease
NCT01592045PHASE1/PHASE2COMPLETEDch14.18 Pharmacokinetic Study in High-risk Neuroblastoma
NCT01701479PHASE1/PHASE2UNKNOWNLong Term Continuous Infusion ch14.18/CHO Plus s.c. Aldesleukin (IL-2)
NCT01857934PHASE2COMPLETEDTherapy for Children With Advanced Stage Neuroblastoma
NCT01969058PHASE2COMPLETEDEffect of Isotretinoin on Immune Activation Among HIV-1 Infected Subjects With Incomplete CD4+ T Cell Recovery
NCT02061384PHASE2COMPLETEDRA-2 13-cis Retinoic Acid (Isotretinoin)
NCT02278861PHASE2COMPLETEDOral Isotretinoin Versus Topical Tretinoin for Actinic Keratosis
NCT02771743PHASE2UNKNOWNResponse-based Treatment of High-risk Neuroblastoma
NCT02864082PHASE2COMPLETEDA Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis
NCT03033303PHASE2COMPLETEDA Study of the Effect of Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin in Patients in First Remission of High-Risk Neuroblastoma
NCT03323801PHASE2COMPLETEDRA-4: 13-cis Retinoic Acid for Treatment of Men With Azoospermia
NCT03471455PHASE2UNKNOWNIsotretinoin In Preventing Recurrences In Chronic Recurrent Dermatophytosis
NCT04154293PHASE2COMPLETEDA Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
NCT04594759PHASE1/PHASE2COMPLETEDWeekly Isotretinoin Therapy Study
NCT04870489PHASE2UNKNOWNCombined Treatment With CO2 Laser and Isotretinoin for Acne Scars
NCT04909515PHASE2WITHDRAWNNaxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GMCSF) and Isotretinoin for Consolidation of Patients With High-Risk Neuroblastoma in First Remission.
NCT05684861PHASE2COMPLETEDSerum TWEAK Levels in Acne Vulgaris
NCT01711554PHASE1ACTIVE_NOT_RECRUITINGLenalidomide and Dinutuximab With or Without Isotretinoin in Treating Younger Patients With Refractory or Recurrent Neuroblastoma
NCT06485505PHASE1RECRUITINGIsotretinoin Versus Excimer Light an Adjuvant in Treating Onychomycosis
NCT00000764PHASE1COMPLETEDChemoprevention of Anal Neoplasia Arising Secondary to Anogenital Human Papillomavirus Infection in Persons With HIV Infection.
NCT00005576PHASE1COMPLETEDMonoclonal Antibody Therapy With Sargramostim and Interleukin-2 in Treating Children With Neuroblastoma
NCT00014729PHASE1COMPLETEDPhase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa
NCT00070200PHASE1COMPLETEDInduction Chemotherapy Using Cyclophosphamide and Topotecan in Treating Patients Who Are Undergoing Autologous Peripheral Stem Cell Transplantation for Newly Diagnosed or Progressive Neuroblastoma
NCT00098891PHASE1COMPLETEDMS-275 and Isotretinoin in Treating Patients With Metastatic or Advanced Solid Tumors or Lymphomas
NCT00217412PHASE1COMPLETEDVorinostat With or Without Isotretinoin in Treating Young Patients With Recurrent or Refractory Solid Tumors, Lymphoma, or Leukemia
NCT00334789PHASE1COMPLETEDBelinostat and Isotretinoin in Treating Patients With Solid Tumors That Are Metastatic or That Cannot Be Removed by Surgery
NCT00416585PHASE1COMPLETEDEffects of Roaccutane on Ca, P, PTH and Vitamin D Metabolites in the Treatment of Sever Acne
NCT00867178PHASE1COMPLETEDVorinostat Combined With Isotretinoin and Chemotherapy in Treating Younger Patients With Embryonal Tumors of the Central Nervous System
NCT01020539PHASE1COMPLETEDAllogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Leukemia

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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