JP2024525760A - 小児患者のファブリー病を治療する方法 - Google Patents

小児患者のファブリー病を治療する方法 Download PDF

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JP2024525760A
JP2024525760A JP2024501910A JP2024501910A JP2024525760A JP 2024525760 A JP2024525760 A JP 2024525760A JP 2024501910 A JP2024501910 A JP 2024501910A JP 2024501910 A JP2024501910 A JP 2024501910A JP 2024525760 A JP2024525760 A JP 2024525760A
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Prior art keywords
migalastat
patient
years
day
fabry disease
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JP2024501910A
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Japanese (ja)
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JP2024525760A5 (https=
JPWO2023288210A5 (https=
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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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Publication of JP2024525760A5 publication Critical patent/JP2024525760A5/ja
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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
    • 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

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  • 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)
JP2024501910A 2021-07-12 2022-07-12 小児患者のファブリー病を治療する方法 Pending JP2024525760A (ja)

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 (3)

Publication Number Publication Date
JP2024525760A true JP2024525760A (ja) 2024-07-12
JP2024525760A5 JP2024525760A5 (https=) 2025-04-28
JPWO2023288210A5 JPWO2023288210A5 (https=) 2025-04-28

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JP2024501910A Pending JP2024525760A (ja) 2021-07-12 2022-07-12 小児患者のファブリー病を治療する方法

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

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
"" Galafold 123mg hard capsules ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS"", INTERNETARCHIVE WAYBACKMACHINE, JPN6025032472, 5 July 2019 (2019-07-05), ISSN: 0005661956 *
"Migalastat 150mg every pther day achieves bioequivalent exposures in adolescent and adult patients w", MOLECULAR GENETICS AND METABOLISM, vol. 132, no. 2, JPN6025032474, 1 February 2021 (2021-02-01), pages 590 - 210, ISSN: 0005661958 *
"Migalastat for the treatment of Fabry disease", EXPERT OPINION ON ORPHAN DRUGS, vol. 6, no. 5, JPN6025032475, 2018, pages 301 - 309, ISSN: 0005661959 *
"Record History: NCT04049760, Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subje", CRINICALTRIALS.GOV ARCHIVE [ONLINE], JPN6025032476, 16 February 2021 (2021-02-16), ISSN: 0005661960 *
AIST産業総合研究所 化学物質リスク管理研究センター 暴露係数ハンドブック 人体関連 体重, JPN6025032473, 30 March 2007 (2007-03-30), ISSN: 0005661957 *

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Publication number Publication date
AU2022310691A1 (en) 2024-01-25
EP4370120A1 (en) 2024-05-22
WO2023288210A1 (en) 2023-01-19
CA3225511A1 (en) 2023-01-19
US20240293387A1 (en) 2024-09-05
KR20240034203A (ko) 2024-03-13

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