公开(公告)号：EP4378524A3

公开(公告)日：2024-08-14

申请号：EP24168024.8

申请日：2017-07-19

申请人： Amicus Therapeutics, Inc.

发明人： Castelli, Jeff

IPC分类号： A61K31/445 ,  A61P9/00 ,  A61P3/00 ,  A61P13/12

CPC分类号： A61K31/445 ,  A61K31/45 ,  A61K31/7008 ,  A61P13/12 ,  A61P3/00 ,  A61P39/02 ,  A61P43/00 ,  A61P9/00

摘要： Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-naïve Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.

公开(公告)号：EP4374918A3

公开(公告)日：2024-08-14

申请号：EP24164126.5

申请日：2018-05-30

申请人： Amicus Therapeutics, Inc.

发明人： Castelli, Jeff

IPC分类号： A61K31/445 ,  A61P13/12

CPC分类号： A61K31/445 ,  A61P13/12 ,  C12Y302/01022

摘要： Provided are methods for treatment of Fabry disease in a patient having renal impairment and/or elevated proteinuria. Certain methods comprise administering to the patient about 100 to about 150 mg free base equivalent of migalastat or salt thereof at a frequency of once every other day. Certain methods also provide for the stabilization of renal function, reducing left ventricular mass index, reducing plasma globotriaosylsphingosine and/or increasing α-galactosidase A activity in the patient.

公开(公告)号：EP4389213A2

公开(公告)日：2024-06-26

申请号：EP24168026.3

申请日：2017-07-19

申请人： Amicus Therapeutics, Inc.

发明人： Castelli, Jeff

IPC分类号： A61P13/12

CPC分类号： A61K31/445 ,  A61K31/45 ,  A61K31/7008 ,  A61P13/12 ,  A61P3/00 ,  A61P39/02 ,  A61P43/00 ,  A61P9/00

摘要： Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-naïve Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.

公开(公告)号：EP4327869A3

公开(公告)日：2024-05-22

申请号：EP23219933.1

申请日：2018-05-30

申请人： Amicus Therapeutics, Inc.

发明人： Castelli, Jeff

IPC分类号： A61K31/445 ,  A61P13/12

CPC分类号： A61K31/445 ,  A61P13/12 ,  C12Y302/01022

摘要： Provided are methods for treatment of Fabry disease in a patient having renal impairment and/or elevated proteinuria. Certain methods comprise administering to the patient about 100 to about 150 mg free base equivalent of migalastat or salt thereof at a frequency of once every other day. Certain methods also provide for the stabilization of renal function, reducing left ventricular mass index, reducing plasma globotriaosylsphingosine and/or increasing α-galactosidase A activity in the patient.

公开(公告)号：EP3630114B1

公开(公告)日：2023-11-01

申请号：EP18733415.6

申请日：2018-05-30

发明人： CASTELLI, Jeff

IPC分类号： A61K31/445 ,  A61P13/12

公开(公告)号：EP3698792A1

公开(公告)日：2020-08-26

申请号：EP20160164.8

申请日：2012-03-08

申请人： Amicus Therapeutics, Inc.

发明人： GREENE, Douglas Stuart ,  VALENZANO, Kenneth Joseph

IPC分类号： A61K31/445 ,  A61K38/47 ,  A61P3/00 ,  A61K9/48

摘要： A pharmaceutical composition comprising migalastat hydrochloride, magnesium stearate and pregelatinized starch.

公开(公告)号：EP3675853A1

公开(公告)日：2020-07-08

申请号：EP18766482.6

申请日：2018-08-28

申请人： Amicus Therapeutics, Inc.

发明人： CASTELLI, Jeff ,  BARTH, Jay ,  SKUBAN, Nina

IPC分类号： A61K31/445 ,  A61P9/04 ,  A61P43/00

公开(公告)号：EP1885366B1

公开(公告)日：2018-05-30

申请号：EP06772163.9

申请日：2006-06-05

申请人： Amicus Therapeutics, Inc. ,  Université de Montréal

发明人： Fan, Jian-qiang ,  Vanlenzano, Ken ,  Lee, Gary ,  Bouvier, Michel ,  René, Patricia

IPC分类号： A61K31/497 ,  A61K31/505 ,  A61K9/00 ,  A61K31/495

CPC分类号： A61K31/495 ,  A61K9/0053 ,  A61K31/497 ,  A61K31/505 ,  C07K14/723

摘要： The invention relates to methods of enhancing normal melanocortin-4 receptor (MC4R) activity, and to enhancing activity of an MC4R having a mutation which affects protein folding and/or processing of the MC4R. The invention provides a method of treating an individual having a condition in which increased activity of an MC4R at the cell surface would be beneficial, for example in obesity, by administering an effective amount of a pharmacological chaperone for the MC4R. The invention provides MC4R pharmacological chaperones which enhance the activity of MC4R. The invention further provides a method of screening to identify pharmacological chaperones which enhance folding of an MC4R in the endoplasmic reticulum (ER), in order to enhance the activity of the MC4R at the cell surface.

公开(公告)号：EP2787345B8

公开(公告)日：2016-04-13

申请号：EP14168748.3

申请日：2007-05-16

申请人： Amicus Therapeutics, Inc.

发明人： Lockhart, David ,  Castelli, Jeff

IPC分类号： G01N33/53 ,  G01N33/567 ,  G01N33/573 ,  A61K31/435

CPC分类号： A61K31/445 ,  A61K9/0053 ,  A61K9/48 ,  A61K31/45 ,  A61K45/06 ,  C07D211/46 ,  G01N33/5094 ,  G01N2333/924 ,  G01N2333/94 ,  G01N2800/04 ,  G01N2800/52 ,  A61K2300/00

公开(公告)号：EP2976092A2

公开(公告)日：2016-01-27

申请号：EP14764160.9

申请日：2014-03-14

申请人： Amicus Therapeutics, Inc.

发明人： DO, Hung

IPC分类号： A61K35/54 ,  A61K38/47 ,  C12N9/24

CPC分类号： A61K47/64 ,  A61K38/30 ,  A61K38/47 ,  A61K47/60 ,  C07K14/65 ,  C07K2319/00 ,  C12N9/16 ,  C12N9/20 ,  C12N9/2402 ,  C12N9/2408 ,  C12N9/2434 ,  C12N9/2465 ,  C12N9/2488 ,  C12N9/96 ,  C12Y302/0102

摘要： Disclosed herein are methods of chemical conjugation comprising contacting a lysosomal enzyme with a first crosslinking agent to introduce aldehyde groups; contacting a lysosomal targeting peptide with a second crosslinking agent to introduce a hydrazide group at the N-terminal residue; contacting the lysosomal enzyme with aldehyde groups of step a. with the lysosomal targeting peptide with a hydrazide group at the N-terminal residue of step b; and forming a lysosomal enzyme-lysosomal targeting peptide conjugate.