Inaxaplin Explained

Legal Status:	Investigational
Synonyms:	VX-147
Cas Number:	2446816-88-0
Pubchem:	147289591
Unii:	S2SJ2RVZ6Y
Chemspiderid:	115009344
Chembl:	5083170
Iupac Name:	3-[5,7-Difluoro-2-(4-fluorophenyl)-1''H''-indol-3-yl]-N-[(3''S'',4''R'')-4-hydroxy-2-oxopyrrolidin-3-yl]propanamide
Stdinchi:	1S/C21H18F3N3O3/c22-11-3-1-10(2-4-11)18-13(14-7-12(23)8-15(24)19(14)27-18)5-6-17(29)26-20-16(28)9-25-21(20)30/h1-4,7-8,16,20,27-28H,5-6,9H2,(H,25,30)(H,26,29)/t16-,20+/m1/s1
Stdinchikey:	CTXLPYZCBOVVQK-UZLBHIALSA-N
Smiles:	C1[C@H]([C@@H](C(=O)N1)NC(=O)CCC2=C(NC3=C2C=C(C=C3F)F)C4=CC=C(C=C4)F)O
C:	21
H:	18
F:	3
N:	3
O:	3

Inaxaplin (VX-147) is a small-molecule apolipoprotein L1 inhibitor developed by Vertex Pharmaceuticals for APOL1-mediated kidney disease. In preliminary studies the drug has shown promise in treating people with kidney disease and multiple gain of function mutations on the APOL1 gene.^{[1] [2]}

Notes and References

1. Gbadegesin . Rasheed . Lane . Brandon . Inaxaplin for the treatment of APOL1-associated kidney disease . Nature Reviews Nephrology . August 2023 . 19 . 8 . 479–480 . 10.1038/s41581-023-00721-0. 37106136 . 10461697 .
2. Egbuna . Ogo . Zimmerman . Brandon . Manos . George . Fortier . Anne . Chirieac . Madalina C. . Dakin . Leslie A. . Friedman . David J. . Bramham . Kate . Campbell . Kirk . Knebelmann . Bertrand . Barisoni . Laura . Falk . Ronald J. . Gipson . Debbie S. . Lipkowitz . Michael S. . Ojo . Akinlolu . Bunnage . Mark E. . Pollak . Martin R. . Altshuler . David . Chertow . Glenn M. . Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants . New England Journal of Medicine . 16 March 2023 . 388 . 11 . 969–979 . 10.1056/NEJMoa2202396. 36920755 . 257534251 .