JP2023513700A - ファブリー病の治療方法 - Google Patents

ファブリー病の治療方法 Download PDF

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Publication number
JP2023513700A
JP2023513700A JP2022548459A JP2022548459A JP2023513700A JP 2023513700 A JP2023513700 A JP 2023513700A JP 2022548459 A JP2022548459 A JP 2022548459A JP 2022548459 A JP2022548459 A JP 2022548459A JP 2023513700 A JP2023513700 A JP 2023513700A
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Prior art keywords
patient
determining
miglastat
therapy
fabry disease
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JP2022548459A
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English (en)
Japanese (ja)
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JP2023513700A5 (https=
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ニーナ スクバン，
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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 JP2023513700A publication Critical patent/JP2023513700A/ja
Publication of JP2023513700A5 publication Critical patent/JP2023513700A5/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
    • A61B—DIAGNOSIS; SURGERY; IDENTIFICATION
    • A61B5/00—Measuring for diagnostic purposes; Identification of persons
    • A61B5/42—Detecting, measuring or recording for evaluating the gastrointestinal, the endocrine or the exocrine systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61B—DIAGNOSIS; SURGERY; IDENTIFICATION
    • A61B5/00—Measuring for diagnostic purposes; Identification of persons
    • A61B5/48—Other medical applications
    • A61B5/4848—Monitoring or testing the effects of treatment, e.g. of medication
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Medical Informatics (AREA)
  • Surgery (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pathology (AREA)
  • Biomedical Technology (AREA)
  • Physiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2022548459A 2020-02-10 2021-02-10 ファブリー病の治療方法 Pending JP2023513700A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062972392P 2020-02-10 2020-02-10
US62/972,392 2020-02-10
PCT/US2021/017446 WO2021163183A1 (en) 2020-02-10 2021-02-10 Methods of treating fabry disease

Publications (2)

Publication Number Publication Date
JP2023513700A true JP2023513700A (ja) 2023-04-03
JP2023513700A5 JP2023513700A5 (https=) 2024-02-21

Family

ID=77272302

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022548459A Pending JP2023513700A (ja) 2020-02-10 2021-02-10 ファブリー病の治療方法

Country Status (11)

Country Link
US (1) US20210251972A1 (https=)
EP (2) EP4732901A2 (https=)
JP (1) JP2023513700A (https=)
KR (1) KR20220150902A (https=)
AU (1) AU2021218578A1 (https=)
CA (1) CA3167476A1 (https=)
DK (1) DK4103178T3 (https=)
FI (1) FI4103178T3 (https=)
LT (1) LT4103178T (https=)
PT (1) PT4103178T (https=)
WO (1) WO2021163183A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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 (5)

* 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.
US9999618B2 (en) * 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
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

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
GALAFOLD(ASSESSMENT REPORT)[ONLINE], JPN6025005384, 2016, ISSN: 0005671819 *
ICHIEI NARITA ET AL.: ""Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT stu", CLINICAL AND EXPERIMENTAL NEPHROLOGY, vol. 24, no. 2, JPN6025005385, 30 December 2019 (2019-12-30), pages 157 - 166, ISSN: 0005671820 *
ガラフォルド カプセル123MG 医薬品インタビューフォーム 第3版[ONLINE]２０１９年掲載, JPN6025005383, 2019, ISSN: 0005671818 *

Also Published As

Publication number Publication date
CA3167476A1 (en) 2021-08-19
LT4103178T (lt) 2026-04-10
EP4732901A2 (en) 2026-04-29
FI4103178T3 (fi) 2026-03-23
EP4103178A1 (en) 2022-12-21
DK4103178T3 (da) 2026-03-30
EP4103178B1 (en) 2025-12-24
AU2021218578A1 (en) 2022-08-25
PT4103178T (pt) 2026-03-30
US20210251972A1 (en) 2021-08-19
EP4103178A4 (en) 2024-03-20
KR20220150902A (ko) 2022-11-11
WO2021163183A1 (en) 2021-08-19

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