Details
| Stereochemistry | ACHIRAL |
| Molecular Formula | C13H17O2.Na |
| Molecular Weight | 228.2626 |
| Optical Activity | NONE |
| Defined Stereocenters | 0 / 0 |
| E/Z Centers | 0 |
| Charge | 0 |
SHOW SMILES / InChI
SMILES
[Na+].CCCCCC1=CC(CC([O-])=O)=CC=C1
InChI
InChIKey=CNSJWSLXKSJQFE-UHFFFAOYSA-M
InChI=1S/C13H18O2.Na/c1-2-3-4-6-11-7-5-8-12(9-11)10-13(14)15;/h5,7-9H,2-4,6,10H2,1H3,(H,14,15);/q;+1/p-1
| Molecular Formula | Na |
| Molecular Weight | 22.98976928 |
| Charge | 1 |
| Count |
|
| Stereochemistry | ACHIRAL |
| Additional Stereochemistry | No |
| Defined Stereocenters | 0 / 0 |
| E/Z Centers | 0 |
| Optical Activity | NONE |
| Molecular Formula | C13H17O2 |
| Molecular Weight | 205.2729 |
| Charge | -1 |
| Count |
|
| Stereochemistry | ACHIRAL |
| Additional Stereochemistry | No |
| Defined Stereocenters | 0 / 0 |
| E/Z Centers | 0 |
| Optical Activity | NONE |
PBI 4050, a synthetic analog of a medium-chain fatty acid that displays agonist and antagonist ligand affinity toward GPR40 and GPR84, that was developed for managing inflammatory and fibrosis-related diseases. In addition, PBI-4050 may exert antifibrotic activity in the liver through a novel mechanism of action involving modulation of intracellular ATP levels and the LKB1/AMPK/mTOR pathway in stellate cells. This drug participated in clinical trials for the treatment of acute lung injury, cystic fibrosis, diabetic nephropathies; idiopathic pulmonary fibrosis; metabolic syndrome; scleroderma; type 2 diabetes mellitus. Besides, this drug has granted a Rare Pediatric Disease Designation for the treatment of Alström syndrome (AS). PBI-4050 was also previously granted Orphan Drug Designation by the FDA and the EMA for the treatments of AS and idiopathic pulmonary fibrosis (IPF) as well as PIM (Promising Innovative Medicine) designation by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of IPF and AS. The FDA grants Rare Pediatric Disease Designations for serious or life-threatening diseases wherein the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups, often called neonates, infants, children, and adolescents. Now Prometic Life Sciences plans to file investigational new drug application for pivotal phase III trial for Alstrom's syndrome in the second half of 2019.
Originator
Approval Year
PubMed
| Title | Date | PubMed |
|---|---|---|
| PBI-4050 reduces pulmonary hypertension, lung fibrosis, and right ventricular dysfunction in heart failure. | 2020-01-01 |
|
| Phase 2 clinical trial of PBI-4050 in patients with idiopathic pulmonary fibrosis. | 2019-03 |
|
| Treatment with PBI-4050 in patients with Alström syndrome: study protocol for a phase 2, single-Centre, single-arm, open-label trial. | 2018-11-26 |
|
| Fatty acid receptor modulator PBI-4050 inhibits kidney fibrosis and improves glycemic control. | 2018-05-17 |
|
| A Newly Discovered Antifibrotic Pathway Regulated by Two Fatty Acid Receptors: GPR40 and GPR84. | 2018-05 |
Sample Use Guides
In Vivo Use Guide
Sources: https://clinicaltrials.gov/ct2/show/NCT03184584
Alström Syndrome: four 200 mg capsules (800 mg total) administered orally, once daily
Route of Administration:
Oral
| Substance Class |
Chemical
Created
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admin
on
Edited
Tue Apr 01 19:55:51 GMT 2025
by
admin
on
Tue Apr 01 19:55:51 GMT 2025
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| Record UNII |
R05571KE07
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Validated (UNII)
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Preferred Name | English | ||
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FDA ORPHAN DRUG |
557016
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FDA ORPHAN DRUG |
464214
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1254472-97-3
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EU/3/15/1550(POSITIVE)
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PRIMARY | treatment of idiopathic pulmonary fibrosis | ||
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100000178210
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46944243
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R05571KE07
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admin on Tue Apr 01 19:55:51 GMT 2025 , Edited by admin on Tue Apr 01 19:55:51 GMT 2025
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