公开(公告)号：AU2021381509A1

公开(公告)日：2023-06-22

申请号：AU2021381509

申请日：2021-11-17

Applicant: VERTEX PHARMA

Inventor： SHI YI ,  LAI MEI-HSIU ,  MEDEK ALES ,  HU KAN-NIAN ,  SONG ZHENGTIAN ,  TORRICO GUZMAN ELISA ,  SOKOLOWSKY KATHLEEN ,  GIROUX SIMON ,  LIU SIYING ,  OVERHOFF KIRK ,  RODAY SETU ,  SAWANT RUPA ,  SPOSATO MARISA

IPC: A61P1/16 ,  A61K31/416 ,  A61K31/4162 ,  A61P11/00 ,  C07D487/04

Abstract: Solid forms of 4-(5-(4-fluorophenyl)-6-(tetrahydro-2H-pyran-4-yl)-1,5-dihydropyrrolo[2,3-f]indazol-7-yl)benzoic acid (Compound 1) capable of modulating alpha-1 antitrypsin (AAT) activity and methods of treating alpha-1 antitrypsin deficiency (AATD) by administering one or more such forms, and methods of using and preparing the same for treatment of alpha-1 antitrypsin deficiency (AATD).