公开(公告)号：WO2019129586A1

公开(公告)日：2019-07-04

申请号：PCT/EP2018/085965

申请日：2018-12-19

申请人： CHIESI FARMACEUTICI S.P.A.

发明人： VILLETTI, Gino ,  BERTOLINI, Serena ,  SORIO, Claudio

IPC分类号： C07D213/89 ,  A61K31/44 ,  C07D213/61 ,  A61P11/00 ,  A61P37/00

CPC分类号： A61P11/00 ,  A61K31/44 ,  A61K31/443 ,  A61K31/47 ,  A61P37/00 ,  C07D213/61 ,  C07D213/89 ,  A61K2300/00

摘要： The present invention relates to the use of agents, which are 1-phenyl-2-pyridinyl alkyl alcohol derivatives, for the prevention and/or treatment of cystic fibrosis in a subject, wherein the subject is characterized by at least one mutation in the gene encoding the CFTR protein, wherein the at least one mutation is causative for incorrect folding and/or processing of the CFTR protein. By the use of the compound according to the present invention, cystic fibrosis in the subject may be prevented or treated. The agent to be used according to the present invention has the capacity to restore the presence of the mutant CFTR protein at the cell surface, and thus act as CFTR correctors. The agent to be used according to the present invention may be administered to a subject in need thereof alone or in combination therapy with other agents, and is suitably administered by inhalation.