JP7677910B2 - 腎機能障害を有する患者のファブリー病を治療する方法 - Google Patents

腎機能障害を有する患者のファブリー病を治療する方法 Download PDF

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JP7677910B2
JP7677910B2 JP2021573404A JP2021573404A JP7677910B2 JP 7677910 B2 JP7677910 B2 JP 7677910B2 JP 2021573404 A JP2021573404 A JP 2021573404A JP 2021573404 A JP2021573404 A JP 2021573404A JP 7677910 B2 JP7677910 B2 JP 7677910B2
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migalastat
patient
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JP2022536687A (ja
JPWO2020252129A5 (enExample
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フランクリン ジョンソン，
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アミカス セラピューティックス インコーポレイテッド
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    • 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

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  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
JP2021573404A 2019-06-11 2020-06-11 腎機能障害を有する患者のファブリー病を治療する方法 Active JP7677910B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025076158A JP2025131572A (ja) 2019-06-11 2025-05-01 腎機能障害を有する患者のファブリー病を治療する方法

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

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JP2025076158A Division JP2025131572A (ja) 2019-06-11 2025-05-01 腎機能障害を有する患者のファブリー病を治療する方法

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JP2022536687A JP2022536687A (ja) 2022-08-18
JPWO2020252129A5 JPWO2020252129A5 (enExample) 2023-06-20
JP7677910B2 true JP7677910B2 (ja) 2025-05-15

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JP2021573404A Active JP7677910B2 (ja) 2019-06-11 2020-06-11 腎機能障害を有する患者のファブリー病を治療する方法
JP2025076158A Pending JP2025131572A (ja) 2019-06-11 2025-05-01 腎機能障害を有する患者のファブリー病を治療する方法

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

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FI3457135T3 (fi) 2006-05-16 2025-03-08 Amicus Therapeutics Inc Fabryn taudin hoitovaihtoehtoja
HUE026543T2 (hu) 2008-02-12 2016-06-28 Amicus Therapeutics Inc Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére
BR112019025083B1 (pt) 2017-05-30 2022-08-09 Amicus Therapeutics, Inc Métodos de tratamento de pacientes portadores de fabry com insuficiência renal
CA3090496C (en) 2018-02-06 2024-03-26 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin Methods of treating fabry disease in patients having a mutation in the gla gene
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014528901A (ja) 2011-03-11 2014-10-30 アミカス セラピューティックス インコーポレイテッド ファブリー病の治療用投薬レジメン
WO2018127920A1 (en) 2017-01-05 2018-07-12 Protalix Ltd. Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase
JP2018526368A (ja) 2016-07-19 2018-09-13 アミカス セラピューティックス インコーポレイテッド Ｅｒｔナイーブ患者及びｅｒｔ経験患者におけるファブリー病の処置
WO2018222655A1 (en) 2017-05-30 2018-12-06 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment

Family Cites Families (6)

* 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
WO2008121826A2 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
HUE026543T2 (hu) 2008-02-12 2016-06-28 Amicus Therapeutics Inc Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére
TWI795408B (zh) * 2017-05-30 2023-03-11 美商阿米庫斯醫療股份有限公司 治療有腎損傷的法布里患者的方法
AR111971A1 (es) * 2017-05-30 2019-09-04 Amicus Therapeutics Inc Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014528901A (ja) 2011-03-11 2014-10-30 アミカス セラピューティックス インコーポレイテッド ファブリー病の治療用投薬レジメン
JP2018526368A (ja) 2016-07-19 2018-09-13 アミカス セラピューティックス インコーポレイテッド Ｅｒｔナイーブ患者及びｅｒｔ経験患者におけるファブリー病の処置
WO2018127920A1 (en) 2017-01-05 2018-07-12 Protalix Ltd. Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase
WO2018222655A1 (en) 2017-05-30 2018-12-06 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment

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Publication number Publication date
KR20220019796A (ko) 2022-02-17
AR120055A1 (es) 2022-02-02
US20220313670A1 (en) 2022-10-06
BR112021024886A2 (pt) 2022-01-25
JP2022536687A (ja) 2022-08-18
EP3982962A1 (en) 2022-04-20
JP2025131572A (ja) 2025-09-09
IL288677A (en) 2022-02-01
PH12021553102A1 (en) 2023-10-09
CN114423427A (zh) 2022-04-29
CA3141226A1 (en) 2020-12-17
CL2021003280A1 (es) 2022-10-07
TW202112372A (zh) 2021-04-01
EA202290024A1 (ru) 2022-03-14
AU2020291002A1 (en) 2022-01-06
WO2020252129A1 (en) 2020-12-17
MX2021015352A (es) 2022-04-06

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