KR20220019796A - 신장 손상을 갖는 환자에서 파브리 질병을 치료하는 방법 - Google Patents

신장 손상을 갖는 환자에서 파브리 질병을 치료하는 방법 Download PDF

Info

Publication number
KR20220019796A
KR20220019796A KR1020227000878A KR20227000878A KR20220019796A KR 20220019796 A KR20220019796 A KR 20220019796A KR 1020227000878 A KR1020227000878 A KR 1020227000878A KR 20227000878 A KR20227000878 A KR 20227000878A KR 20220019796 A KR20220019796 A KR 20220019796A
Authority
KR
South Korea
Prior art keywords
migalastat
patient
frequency
days
period
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
KR1020227000878A
Other languages
English (en)
Korean (ko)
Inventor
프랭클린 존슨
Original Assignee
아미쿠스 세라퓨틱스, 인코포레이티드
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 아미쿠스 세라퓨틱스, 인코포레이티드 filed Critical 아미쿠스 세라퓨틱스, 인코포레이티드
Publication of KR20220019796A publication Critical patent/KR20220019796A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
KR1020227000878A 2019-06-11 2020-06-11 신장 손상을 갖는 환자에서 파브리 질병을 치료하는 방법 Pending KR20220019796A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962859904P 2019-06-11 2019-06-11
US62/859,904 2019-06-11
PCT/US2020/037174 WO2020252129A1 (en) 2019-06-11 2020-06-11 Methods of treating fabry disease in patients having renal impairment

Publications (1)

Publication Number Publication Date
KR20220019796A true KR20220019796A (ko) 2022-02-17

Family

ID=71950725

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227000878A Pending KR20220019796A (ko) 2019-06-11 2020-06-11 신장 손상을 갖는 환자에서 파브리 질병을 치료하는 방법

Country Status (16)

Country Link
US (1) US20220313670A1 (https=)
EP (1) EP3982962A1 (https=)
JP (2) JP7677910B2 (https=)
KR (1) KR20220019796A (https=)
CN (1) CN114423427A (https=)
AR (1) AR120055A1 (https=)
AU (2) AU2020291002A1 (https=)
BR (1) BR112021024886A2 (https=)
CA (1) CA3141226A1 (https=)
CL (1) CL2021003280A1 (https=)
EA (1) EA202290024A1 (https=)
IL (1) IL288677A (https=)
MX (1) MX2021015352A (https=)
PH (1) PH12021553102A1 (https=)
TW (1) TW202112372A (https=)
WO (1) WO2020252129A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
US12594268B2 (en) 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
MX2009010557A (es) 2007-03-30 2009-11-19 Amicus Therapeutics Inc Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
TW202408513A (zh) * 2016-07-19 2024-03-01 美商阿米庫斯醫療股份有限公司 治療ert初治的和已經歷ert的患者中之法布瑞氏症
WO2018127920A1 (en) * 2017-01-05 2018-07-12 Protalix Ltd. Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase
CA3224546C (en) * 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
AR111971A1 (es) * 2017-05-30 2019-09-04 Amicus Therapeutics Inc Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal
RS65230B1 (sr) * 2017-05-30 2024-03-29 Amicus Therapeutics Inc Migalastat za lečenje pacijenata koji boluju od fabrijeve bolesti sa oštećenjem bubrega

Also Published As

Publication number Publication date
AU2026200618A1 (en) 2026-04-16
WO2020252129A1 (en) 2020-12-17
IL288677A (en) 2022-02-01
CN114423427A (zh) 2022-04-29
EP3982962A1 (en) 2022-04-20
PH12021553102A1 (en) 2023-10-09
BR112021024886A2 (pt) 2022-01-25
CL2021003280A1 (es) 2022-10-07
US20220313670A1 (en) 2022-10-06
CA3141226A1 (en) 2020-12-17
JP2025131572A (ja) 2025-09-09
AR120055A1 (es) 2022-02-02
AU2020291002A1 (en) 2022-01-06
MX2021015352A (es) 2022-04-06
JP7677910B2 (ja) 2025-05-15
TW202112372A (zh) 2021-04-01
EA202290024A1 (ru) 2022-03-14
JP2022536687A (ja) 2022-08-18

Similar Documents

Publication Publication Date Title
JP7784398B2 (ja) Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法
KR102427610B1 (ko) 신장 손상을 갖는 파브리 환자를 치료하는 방법
KR102790184B1 (ko) 파브리병을 앓는 환자에서 심장 기능을 강화 및/또는 안정화시키는 방법
AU2025217396A1 (en) Treatment of fabry disease in ert-naïve and ert-experienced patients
KR102004114B1 (ko) Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
KR20220019796A (ko) 신장 손상을 갖는 환자에서 파브리 질병을 치료하는 방법
AU2025200074A1 (en) Treatment of patients with classic fabry disease
JP2026016375A (ja) Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法
JP2025128105A (ja) ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用
CN111278438A (zh) 治疗具有肾损害的法布里患者的方法
HK40085360A (en) Methods of treating fabry disease in patients having a mutation in the gla gene
HK40073744A (en) Methods of treating fabry disease in patients having renal impairment
HK40044440A (en) Treatment of patients with classic fabry disease
HK40031708A (en) Methods of treating fabry patients having renal impairment
HK40039916A (en) Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease
HK40001720A (en) Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
HK40007037A (en) Treatment of fabry disease in ert-naïve and ert-experienced patients

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

D22 Grant of ip right intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D22-EXM-PE0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701