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

신장 손상을 갖는 환자에서 파브리 질병을 치료하는 방법 Download PDF

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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
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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
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English (en)
Korean (ko)
Inventor
프랭클린 존슨
Original Assignee
아미쿠스 세라퓨틱스, 인코포레이티드
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Application filed by 아미쿠스 세라퓨틱스, 인코포레이티드 filed Critical 아미쿠스 세라퓨틱스, 인코포레이티드
Publication of KR20220019796A publication Critical patent/KR20220019796A/ko
Pending legal-status Critical Current

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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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (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)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (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)
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 (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
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
DK3470077T3 (da) 2008-02-12 2020-11-30 Amicus Therapeutics Inc Fremgangsmåde til forudsigelse af respons på behandling af sygdomme med farmakologiske chaperoner
CA3224546A1 (en) 2017-05-30 2018-12-06 Jeff Castelli Use of migalastat in the treatment of fabry patients having renal impairment
JP7555818B2 (ja) 2018-02-06 2024-09-25 アミカス セラピューティックス インコーポレイテッド 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用
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
AU2008232614A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of Fabry disease using pharmacological chaperones
US9056101B2 (en) * 2007-04-26 2015-06-16 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
DK3470077T3 (da) 2008-02-12 2020-11-30 Amicus Therapeutics Inc Fremgangsmåde til forudsigelse af respons på behandling af sygdomme med farmakologiske chaperoner
SG193379A1 (en) * 2011-03-11 2013-10-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
IL313907A (en) * 2016-07-19 2024-08-01 Amicus Therapeutics Inc Treatment of Fabry disease in patients experienced with ERT-NAIVE and ERT
MX2019008076A (es) * 2017-01-05 2019-08-29 Protalix Ltd Regimen terapeutico para el tratamiento de fabry usando alfa-galactosidasa estabilizada.
CA3224546A1 (en) * 2017-05-30 2018-12-06 Jeff Castelli Use of migalastat in the treatment of fabry patients having renal impairment
AR111971A1 (es) * 2017-05-30 2019-09-04 Amicus Therapeutics Inc Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal
HUE069837T2 (hu) * 2017-05-30 2025-04-28 Amicus Therapeutics Inc Bizonyos mutációkat hordozó Fabry-kóros betegek kezelése

Also Published As

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

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