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Tezacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector that is a key component of the triple-combination therapy TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor) for the treatment of cystic fibrosis in patients with at least one F508del mutation . Chemically, Tezacaftor functions as a CFTR corrector, improving the processing and trafficking of defective CFTR proteins to the cell surface.

Linerixibat is an oral, minimally absorbed ileal bile acid transporter (IBAT) inhibitor developed for the treatment of cholestatic pruritus in patients with primary biliary cholangitis (PBC) . Chemically, Linerixibat inhibits the ileal bile acid transporter, reducing bile acid reabsorption and thereby decreasing systemic bile acid levels, which are associated with the pruritus experienced by PBC patients .

Aficamten (CAS 2364554-48-1), also known as CK-3773274, is a potent, next-generation cardiac myosin inhibitor designed for the treatment of hypertrophic cardiomyopathy (HCM) . Structurally, Aficamten is a pyrazole carboxamide derivative featuring an indane core with an ethyl-oxadiazole substituent . The molecule has the molecular formula C₁₈H₁₉N₅O₂ and contains a chiral center that contributes to its selective activity as a cardiac myosin inhibitor .

Elafibranor (CAS 824932-88-9), also known as GFT505, is a potent dual peroxisome proliferator-activated receptor (PPAR) α/δ agonist with significant therapeutic potential for metabolic disorders . Structurally, Elafibranor is a sulfonyl-containing carboxylic acid derivative with the molecular formula C₂₂H₂₄O₄S . The molecule features a conjugated system linking aromatic and sulfonyl-substituted rings, contributing to its high-affinity binding to PPARα and PPARδ.

3,4-Difluoro-2-methoxyphenylacetic acid (CAS 1558274-26-2) is a specialized phenylacetic acid derivative featuring a distinctive substitution pattern on the aromatic ring. Structurally, 3,4-Difluoro-2-methoxyphenylacetic acid consists of a phenylacetic acid core with a methoxy group at the 2-position and fluorine atoms at the 3- and 4-positions of the benzene ring.

Omidenepag Isopropyl (CAS 1187451-19-9) is a selective, non-prostaglandin EP2 receptor agonist developed for the treatment of glaucoma and ocular hypertension. Chemically known as isopropyl (5-{[4-(1,3-benzothiazol-2-yl)piperazin-1-yl]carbonyl}-1,2-benzoxazol-3-yl)acetate, Omidenepag Isopropyl features a complex benzoxazole core linked to a piperazine-benzothiazole moiety via an amide bond.