Pimecrolimus

Summary

Pimecrolimus (CHEMBL1200686) is an approved small molecule (ATC D11AH02) targeting FKBP1A; indicated across 11 conditions including atopic eczema and lichen planus.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: D11AH02
• Targets: 1 (FKBP1A)
• Indications: 11 conditions
• Clinical trials: 42
• Chemistry: 810.4 Da · C43H68ClNO11

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1200686
Name	Pimecrolimus
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	6509979
ATC	D11AH02
Molecular formula	C43H68ClNO11
Molecular weight	810.4
InChIKey	KASDHRXLYQOAKZ-XDSKOBMDSA-N

SMILES: CC[C@@H]1/C=C(/C[C@@H](C[C@@H]([C@@H]2[C@H](C[C@H]([C@@](O2)(C(=O)C(=O)N3CCCC[C@H]3C(=O)O[C@@H]([C@@H]([C@H](CC1=O)O)C)/C(=C/[C@@H]4CC[C@@H]([C@@H](C4)OC)Cl)/C)O)C)OC)OC)C)\C

IUPAC name: (1R,9S,12S,13R,14S,17R,18E,21S,23S,24R,25S,27R)-12-[(E)-1-[(1R,3R,4S)-4-chloro-3-methoxycyclohexyl]prop-1-en-2-yl]-17-ethyl-1,14-dihydroxy-23,25-dimethoxy-13,19,21,27-tetramethyl-11,28-dioxa-4-azatricyclo[22.3.1.04,9]octacos-18-ene-2,3,10,16-tetrone

Also known as: (-)-pimecrolimus, Elidel, Pimecrolimus, SDZ ASM 981, SDZ ASM-981, SDZ-ASM 981, SDZ-ASM-981, PIMECROLIMUS, pimecrolimus

Patent coverage: 272 distinct patent families (691 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

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
FKBP1A	FKBP prolyl isomerase 1A	Inhibition	8.22	21.5%	P62942

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Adenosine receptor A3, Bile salt export pump.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
ADORA3	5.29	AC50	5189	nM	CHEMBL_ACT_25198755
ABCB11	5	IC50	10000	nM	CHEMBL_ACT_18128991

Target pathways

Aggregated over 1 target gene(s): FKBP1A.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
mTORC1-mediated signalling	1	FKBP1A
Calcineurin activates NFAT	1	FKBP1A
TGF-beta receptor signaling activates SMADs	1	FKBP1A
TGF-beta receptor signaling in EMT (epithelial to mesenchymal transition)	1	FKBP1A
TGFBR1 LBD Mutants in Cancer	1	FKBP1A
Potential therapeutics for SARS	1	FKBP1A
SARS-CoV-1 activates/modulates innate immune responses	1	FKBP1A

Dominant GO biological processes

GO term	Targets
heart morphogenesis	1
protein folding	1
‘de novo’ protein folding	1
regulation of cardiac muscle contraction by regulation of the release of sequestered calcium ion	1
regulation of skeletal muscle contraction by regulation of release of sequestered calcium ion	1
negative regulation of transforming growth factor beta receptor signaling pathway	1
regulation of protein localization	1
negative regulation of activin receptor signaling pathway	1
protein refolding	1
T cell activation	1
positive regulation of canonical NF-kappaB signal transduction	1
regulation of immune response	1
protein maturation	1
ventricular cardiac muscle tissue morphogenesis	1
heart trabecula formation	1

Indications & clinical

Indications

11 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
atopic eczema	4	MONDO:0004980	EFO:0000274
lichen planus	3	MONDO:0006572	EFO:1000726
dermatitis	3	MONDO:0002406	MONDO:0002406
vitiligo	2	MONDO:0008661	EFO:0004208
lichen disease	2	MONDO:0006570	EFO:1000724
seborrheic dermatitis	2	MONDO:0006608	EFO:1000764
lichen planus, oral	2	MONDO:0043923	EFO:0008517
dry eye syndrome	2	MONDO:0006733	EFO:1000906
Netherton syndrome	1	MONDO:0009735	MONDO:0009735

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

Clinical trials

Total trials: 42.

Phase distribution

Phase	Trials
PHASE4	17
PHASE2	8
Not specified	7
PHASE3	6
PHASE1	3
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00117377	PHASE4	COMPLETED	Effects of Pimecrolimus Cream 1% on the Molecular and Cellular Profile of Adult Male Patients With Atopic Dermatitis
NCT00119158	PHASE4	COMPLETED	Combination Therapy for Atopic Dermatitis
NCT00120302	PHASE4	COMPLETED	Quality of Life Study in Adults With Facial Eczema
NCT00121316	PHASE4	COMPLETED	Safety and Efficacy of Pimecrolimus Cream 1% in Mild to Moderate Head and Neck Atopic Dermatitis (AD) Patients
NCT00124709	PHASE4	TERMINATED	Safety and Efficacy of Pimecrolimus Cream 1% in Atopic Disease Modification
NCT00130364	PHASE4	COMPLETED	Efficacy and Safety of Pimecrolimus Cream 1% in Patients (2 to 11 Years Old) With Mild to Moderate Facial Atopic Dermatitis
NCT00139581	PHASE4	COMPLETED	Comparison of Pimecrolimus Cream 1% Twice-Daily to Once-Daily Dosing in the Management of Atopic Dermatitis in Pediatric Subjects
NCT00509990	PHASE4	COMPLETED	Open Label Study of Long Term Treatment of Pediatric Treatment of Atopic Dermatitis With Pimecrolimus Cream 1% Within a Usual Clinical Setting
NCT00510003	PHASE4	COMPLETED	Assessment of Pruritus Improvement With Pimecrolimus Treatment on the Areas Affected by Mild-to-moderate AD, in Patients 2- to 11- Year-old
NCT00568412	PHASE4	COMPLETED	A Clinical Study in Children and Adolescents With Mild to Moderate Atopic Dermatitis
NCT00666159	PHASE4	COMPLETED	Comparison Study Between Protopic (Tacrolimus Ointment) and Elidel (Pimecrolimus Cream) in Treating Pediatric Patients With Atopic Dermatitis
NCT00666302	PHASE4	COMPLETED	A Comparison Study Between Protopic (Tacrolimus) Ointment and Elidel (Pimecrolimus) Cream in Treating Subjects With Atopic Dermatitis
NCT00667160	PHASE4	COMPLETED	Comparison Study Between Protopic (Tacrolimus Ointment) and Elidel (Pimecrolimus Cream) in Pediatric Patients With Mild Atopic Dermatitis
NCT00925730	PHASE4	COMPLETED	Open-label Study to Investigate Systemic Exposure in Adult and Pediatric Atopic Dermatitis Patients Treated 8.5 Days With Pimecrolimus Cream 1% Under Occlusion
NCT01082393	PHASE4	COMPLETED	Vitiligo and the Koebner Phenomenon (Model of Vitiligo Induction and Therapy: a Clinical and Immunological Analysis)
NCT01177566	PHASE4	COMPLETED	Pimecrolimus Cream 1% (Elidel®) and Medicated Device Cream (EletoneTM) in the Treatment and Maintenance of Atopic Dermatitis.
NCT02443311	PHASE4	COMPLETED	Clinical and Immunohistochemical Effect of Topical Pimecrolimus in Treatment of Oral Lichen Planus
NCT00120523	PHASE3	COMPLETED	5-Year Safety Study of Pimecrolimus Cream 1% in Infants 3 - 12 Months of Age With Mild to Moderate Atopic Dermatitis
NCT00130572	PHASE3	COMPLETED	Efficacy of Elidel Cream on Erosive Oral Lichen Planus
NCT00231998	PHASE3	COMPLETED	Extension Study of Pimecrolimus Cream in Pediatric Patients With Atopic Dermatitis
NCT00232115	PHASE3	COMPLETED	Pimecrolimus Cream 1% in Adult Patients With Perioral Dermatitis
NCT00351052	PHASE3	COMPLETED	Steroid-Sparing Effect With Pimecrolimus in Pediatric Patients With Atopic Dermatitis
NCT06400511	PHASE3	TERMINATED	A Phase 3 Study to Evaluate the Efficacy and Safety of Pimecrolimus 0.3% Ophthalmic Ointment
NCT00128245	PHASE2	COMPLETED	Safety and Efficacy of Pimecrolimus Ophthalmic Suspension in Patients With Moderate to Severe Keratoconjunctivitis Sicca
NCT00208026	PHASE1/PHASE2	COMPLETED	Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome
NCT00297037	PHASE2	COMPLETED	Pimecrolimus Cream for Oral Lichen Planus
NCT00393263	PHASE2	COMPLETED	Clobetasol Versus Pimecrolimus for Vulvar Lichen Sclerosus
NCT00403559	PHASE2	COMPLETED	A 4 Week Study of Elidel (Pimecrolimus) for the Treatment of Seborrheic Dermatitis
NCT00507832	PHASE2	COMPLETED	Evaluation of the Antipruritic Effect of Elidel (Pimecrolimus) in Non-atopic Pruritic Disease
NCT00610142	PHASE2	COMPLETED	Comparison of Atrophogenic Effect of Hydrocortison 1% Cream and Pimecrolimus 1% Cream Assessed by Optical Coherence Tomography (OCT) and 20-MHz Ultrasound of Uninvolved Skin in Patients With Atopic Dermatitis
NCT01524744	PHASE2	UNKNOWN	A Randomized Clinical Trial of the Effect of Pimecrolimus Cream 1% Compared With Topical Corticosteroid in Treatment of Erosive Oral Lichen Planus
NCT01692626	PHASE2	TERMINATED	1% Topical Pimecrolimus Cream for the Treatment of the Rash Associated With ERBITUX
NCT00655512	PHASE1	COMPLETED	Comparison of Skin Thickness Under Treatment With Pimecrolimus 1% Cream, Hydrocortisonacetat 1% Cream, Betamethasonvalerat 0,1% Cream and Clobetasol-17-propionat 0,05% Cream Assessed by Optical Coherence Tomography (OCT) and 20-MHZ Ultrasound
NCT02103725	PHASE1	COMPLETED	A Multi-Centre Clinical Trial to Evaluate a Left-Right Design in Adults With Mild to Moderate Atopic Dermatitis
NCT02376049	PHASE1	COMPLETED	A Clinical Trial to Compare Topical Agents in Adults With Mild to Moderate Atopic Dermatitis (AD)
NCT00568997	Not specified	ACTIVE_NOT_RECRUITING	10 Year Registry of Children (Ages 2-17 Years) With Eczema That Have Used Pimecrolimus
NCT00148746	Not specified	COMPLETED	Standardized Time- and Score-oriented Treatment of Moderate and Severe Atopic Dermatitis
NCT00222183	Not specified	WITHDRAWN	Cutaneous Lupus Erythematosus and Elidel
NCT00608673	Not specified	COMPLETED	Comparing the Therapeutic Efficacy of Pimecrolimus Cream With Betamethasone Cream for Discoid Lupus Erythematosus
NCT00946478	Not specified	COMPLETED	Effect of Pimecrolimus Cream on Cathelicidin Levels in Subjects With Eczema

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

6 molecules share ≥1 primary target. Top 6 by shared-target count:

Molecule	Source	Status	Shared targets
CYCLOSPORINE	ChEMBL	Phase 4 (approved)	FKBP1A
SIROLIMUS	ChEMBL	Phase 4 (approved)	FKBP1A
TACROLIMUS	ChEMBL	Phase 4 (approved)	FKBP1A
THIABENDAZOLE	ChEMBL	Phase 4 (approved)	FKBP1A
BIRICODAR	ChEMBL	Phase 2	FKBP1A
CYCLOHEXIMIDE	ChEMBL	Phase 2	FKBP1A