CA3225511A1 - Methods of treating fabry disease in pediatric patients - Google Patents

Methods of treating fabry disease in pediatric patients Download PDF

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Publication number
CA3225511A1
CA3225511A1 CA3225511A CA3225511A CA3225511A1 CA 3225511 A1 CA3225511 A1 CA 3225511A1 CA 3225511 A CA3225511 A CA 3225511A CA 3225511 A CA3225511 A CA 3225511A CA 3225511 A1 CA3225511 A1 CA 3225511A1
Authority
CA
Canada
Prior art keywords
migalastat
patient
years
range
day
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
CA3225511A
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
Amicus Therapeutics Inc
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 Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of CA3225511A1 publication Critical patent/CA3225511A1/en
Pending legal-status Critical Current

Links

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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3225511A 2021-07-12 2022-07-12 Methods of treating fabry disease in pediatric patients Pending CA3225511A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163220816P 2021-07-12 2021-07-12
US63/220,816 2021-07-12
PCT/US2022/073626 WO2023288210A1 (en) 2021-07-12 2022-07-12 Methods of treating fabry disease in pediatric patients

Publications (1)

Publication Number Publication Date
CA3225511A1 true CA3225511A1 (en) 2023-01-19

Family

ID=83149437

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3225511A Pending CA3225511A1 (en) 2021-07-12 2022-07-12 Methods of treating fabry disease in pediatric patients

Country Status (7)

Country Link
US (1) US20240293387A1 (https=)
EP (1) EP4370120A1 (https=)
JP (1) JP2024525760A (https=)
KR (1) KR20240034203A (https=)
AU (1) AU2022310691A1 (https=)
CA (1) CA3225511A1 (https=)
WO (1) WO2023288210A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3851105B1 (en) 2019-08-07 2026-03-04 Amicus Therapeutics, Inc. Migalastat for use in treating fabry disease in patients having a mutation in the gla gene

Family Cites Families (3)

* 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
SI3470077T1 (sl) 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem

Also Published As

Publication number Publication date
AU2022310691A1 (en) 2024-01-25
EP4370120A1 (en) 2024-05-22
JP2024525760A (ja) 2024-07-12
WO2023288210A1 (en) 2023-01-19
US20240293387A1 (en) 2024-09-05
KR20240034203A (ko) 2024-03-13

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