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

    • 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)
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 (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
KR20220019796A (ko) 2022-02-17
US20220313670A1 (en) 2022-10-06
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

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CA3141226A1 (en) Methods of treating fabry disease in patients having renal impairment
US20260076951A1 (en) Methods of treating fabry disease in patients having renal impairment
EP4667050A2 (en) Migalastat formulations having improved pharmacokinetics

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