Biotin

Summary

Biotin (CHEMBL857) is an approved small-molecule prosthetic group (ATC A11HA05); indicated across 11 conditions including iron deficiency anemia and anemia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A11HA05
• Indications: 11 conditions
• Clinical trials: 14
• Chemistry: 244.31 Da · C10H16N2O3S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL857
Name	Biotin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	171548
ChEBI	CHEBI:15956
ATC	A11HA05
Molecular formula	C10H16N2O3S
Molecular weight	244.31
InChIKey	YBJHBAHKTGYVGT-ZKWXMUAHSA-N

SMILES: C1[C@H]2[C@@H]([C@@H](S1)CCCCC(=O)O)NC(=O)N2

IUPAC name: 5-[(3aS,4S,6aR)-2-oxo-1,3,3a,4,6,6a-hexahydrothieno[3,4-d]imidazol-4-yl]pentanoic acid

ChEBI definition: An organic heterobicyclic compound that consists of 2-oxohexahydro-1H-thieno[3,4-d]imidazole having a valeric acid substituent attached to the tetrahydrothiophene ring. The parent of the class of biotins.

Pharmacological roles (ChEBI): prosthetic group, coenzyme, nutraceutical, cofactor.

Other ChEBI roles (chemical / environmental): human metabolite, Saccharomyces cerevisiae metabolite, Escherichia coli metabolite, mouse metabolite, fundamental metabolite.

Also known as: Bioepiderm, Biotin, Biotin component of vitaped, Biotina, Biotine, Biotin 100, D-biotin, MD-1003, NSC-63865, Ritatin, Vitamin b7, Vitamin h

Patent coverage: 192,746 distinct patent families (584,666 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: 9 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Pyruvate kinase PKM, Lethal(3)malignant brain tumor-like protein 1, Prelamin-A/C, Inositol monophosphatase 1, Insulin-degrading enzyme, Menin/Histone-lysine N-methyltransferase MLL, Ras and Rab interactor 1/Tyrosine-protein kinase ABL1, Mitogen-activated protein kinase 1.

Bioactivity

ChEMBL activities: 10 potent at pChembl ≥ 5 of 13 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
LMNA	8	Potency	10	nM	CHEMBL_ACT_3633480
TDP1	7.9	Potency	12.6	nM	CHEMBL_ACT_3934542
TDP1	7.65	Potency	22.4	nM	CHEMBL_ACT_3933820
L3MBTL1	6.45	Potency	354.8	nM	CHEMBL_ACT_4604141
ABL1	6.28	IC50	528.7	nM	CHEMBL_ACT_10221306
IDE	5.62	EC50	2373	nM	CHEMBL_ACT_7551057
MAPK1	5.6	Potency	2512	nM	CHEMBL_ACT_4533678
IDE	5.6	EC50	2526	nM	CHEMBL_ACT_6459133
TDP1	5.55	Potency	2818	nM	CHEMBL_ACT_3936823
P97697	5.25	Potency	5623	nM	CHEMBL_ACT_4415892

Target pathways

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

Indications & clinical

Indications

11 indications (4 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
iron deficiency anemia	4	MONDO:0001356	HP:0001891
anemia	4	MONDO:0002280	EFO:0004272
chronic kidney disease	4	MONDO:0005300	EFO:0003884
type 2 diabetes mellitus	4	MONDO:0005148	MONDO:0005148
multiple sclerosis	3	MONDO:0005301	MONDO:0005301
asthma	3	MONDO:0004979	MONDO:0004979
heart failure	2	MONDO:0005252	EFO:0003144
neonatal anemia	2	MONDO:0001240	MONDO:0001240
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976
Huntington disease	2	MONDO:0007739	MONDO:0007739
sickle cell disease	0	MONDO:0011382	MONDO:0011382

Clinical trials

Total trials: 14.

Phase distribution

Phase	Trials
Not specified	9
EARLY_PHASE1	2
PHASE4	1
PHASE3	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03995277	PHASE4	COMPLETED	Effect of Biotin on Routine Laboratory Values
NCT05632549	PHASE3	UNKNOWN	The Possible Efficacy and Safety of L-carnitine and Biotin as Adjunctive Therapies in Children With Moderate Persistent Asthma
NCT03427086	PHASE2	COMPLETED	Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis
NCT04476277	EARLY_PHASE1	COMPLETED	Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease
NCT05450900	EARLY_PHASE1	COMPLETED	Can Biotin Supplementation be Used to Mask hCG Abuse?
NCT00894920	Not specified	COMPLETED	Biotin Status in Pregnancy
NCT02011191	Not specified	COMPLETED	Biotin Deficiency and Restless Legs Syndrome
NCT03034707	Not specified	COMPLETED	Interference of Biotin Supplementation in Biotin-streptavidin Platforms for Hormone Testing
NCT03300440	Not specified	UNKNOWN	PROVIT The Influence of Probiotics on Body and Mind in Individuals With Psychiatric Disorders
NCT03552211	Not specified	UNKNOWN	Evaluation of the Incidence of Relapses in Patients With Biotin-treated Progressive Multiple Sclerosis
NCT03746821	Not specified	COMPLETED	Biotin Sample Collection Study
NCT05832190	Not specified	TERMINATED	Correcting GUT MicrobioTa by Combined Supplementation of FibERs and BIotiN to Improve Microbiome and Optimize Bariatric Surgery Outcomes
NCT06010745	Not specified	UNKNOWN	Effectiveness of a Novel Dietary Ingredient on Hair Growth and Skin’s Appearance
NCT07348120	Not specified	COMPLETED	Efficacy and Safety of Millet Seed Extract in Telogen Effluvium Treatment

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.

Related molecules

Related molecules

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