CA3141226A1 - Methods of treating fabry disease in patients having renal impairment - Google Patents

Methods of treating fabry disease in patients having renal impairment Download PDF

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Publication number
CA3141226A1
CA3141226A1 CA3141226A CA3141226A CA3141226A1 CA 3141226 A1 CA3141226 A1 CA 3141226A1 CA 3141226 A CA3141226 A CA 3141226A CA 3141226 A CA3141226 A CA 3141226A CA 3141226 A1 CA3141226 A1 CA 3141226A1
Authority
CA
Canada
Prior art keywords
migalastat
patient
time period
frequency
renal impairment
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
CA3141226A
Other languages
English (en)
French (fr)
Inventor
Franklin Johnson
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Amicus Therapeutics Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA3141226A1 publication Critical patent/CA3141226A1/en
Pending legal-status Critical Current

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-galactosidase (3.2.1.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
CA3141226A 2019-06-11 2020-06-11 Methods of treating fabry disease in patients having renal impairment Pending CA3141226A1 (en)

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
CA3141226A1 true CA3141226A1 (en) 2020-12-17

Family

ID=71950725

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3141226A Pending CA3141226A1 (en) 2019-06-11 2020-06-11 Methods of treating fabry disease in patients having renal impairment

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
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

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
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
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
CA3031249A1 (en) * 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
AU2018205891B2 (en) * 2017-01-05 2024-11-07 Protalix Ltd. Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase
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
BR112019025083B1 (pt) * 2017-05-30 2022-08-09 Amicus Therapeutics, Inc Métodos de tratamento de pacientes portadores de fabry com insuficiência renal
AR111971A1 (es) * 2017-05-30 2019-09-04 Amicus Therapeutics Inc Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal

Also Published As

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
CL2021003280A1 (es) 2022-10-07
TW202112372A (zh) 2021-04-01
EA202290024A1 (ru) 2022-03-14
JP7677910B2 (ja) 2025-05-15
AU2020291002A1 (en) 2022-01-06
WO2020252129A1 (en) 2020-12-17
MX2021015352A (es) 2022-04-06

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CA3141226A1 (en) Methods of treating fabry disease in patients having renal impairment
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Legal Events

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EEER Examination request

Effective date: 20220929

EEER Examination request

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EEER Examination request

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EEER Examination request

Effective date: 20220929