JP2014528901A - ファブリー病の治療用投薬レジメン - Google Patents

ファブリー病の治療用投薬レジメン Download PDF

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Publication number
JP2014528901A
JP2014528901A JP2013557862A JP2013557862A JP2014528901A JP 2014528901 A JP2014528901 A JP 2014528901A JP 2013557862 A JP2013557862 A JP 2013557862A JP 2013557862 A JP2013557862 A JP 2013557862A JP 2014528901 A JP2014528901 A JP 2014528901A
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gal
agarsidase
deoxygalactonojirimycin
administration
administered
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JP2013557862A
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Japanese (ja)
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JP2014528901A5 (https=
Inventor
ダグラス、スチュアート、グリーン
ケネス、ジョセフ、バレンツァノ
Original Assignee
アミカス セラピューティックス インコーポレイテッド
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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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=46831259&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP2014528901(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by アミカス セラピューティックス インコーポレイテッド, アミカス セラピューティックス インコーポレイテッド filed Critical アミカス セラピューティックス インコーポレイテッド
Publication of JP2014528901A publication Critical patent/JP2014528901A/ja
Publication of JP2014528901A5 publication Critical patent/JP2014528901A5/ja
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • 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
    • 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)
    • C12Y302/01062—Glycosylceramidase (3.2.1.62)
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/20—Pills, tablets, discs, rods

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Hydrogenated Pyridines (AREA)
JP2013557862A 2011-03-11 2012-03-08 ファブリー病の治療用投薬レジメン Pending JP2014528901A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201161451798P 2011-03-11 2011-03-11
US61/451,798 2011-03-11
US201161578201P 2011-12-20 2011-12-20
US61/578,201 2011-12-20
US201261596165P 2012-02-07 2012-02-07
US61/596,165 2012-02-07
PCT/US2012/028260 WO2012125402A2 (en) 2011-03-11 2012-03-08 Dosing regimens for the treatment of fabry disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2017038469A Division JP2017132780A (ja) 2011-03-11 2017-03-01 ファブリー病の治療用投薬レジメン

Publications (2)

Publication Number Publication Date
JP2014528901A true JP2014528901A (ja) 2014-10-30
JP2014528901A5 JP2014528901A5 (https=) 2015-04-30

Family

ID=46831259

Family Applications (6)

Application Number Title Priority Date Filing Date
JP2013557862A Pending JP2014528901A (ja) 2011-03-11 2012-03-08 ファブリー病の治療用投薬レジメン
JP2017038469A Pending JP2017132780A (ja) 2011-03-11 2017-03-01 ファブリー病の治療用投薬レジメン
JP2019186952A Pending JP2020033360A (ja) 2011-03-11 2019-10-10 ファブリー病の治療用投薬レジメン
JP2021018456A Withdrawn JP2021098697A (ja) 2011-03-11 2021-02-08 ファブリー病の治療用投薬レジメン
JP2023045001A Pending JP2023093448A (ja) 2011-03-11 2023-03-22 ファブリー病の治療用投薬レジメン
JP2025179237A Pending JP2026035578A (ja) 2011-03-11 2025-10-24 ファブリー病の治療用投薬レジメン

Family Applications After (5)

Application Number Title Priority Date Filing Date
JP2017038469A Pending JP2017132780A (ja) 2011-03-11 2017-03-01 ファブリー病の治療用投薬レジメン
JP2019186952A Pending JP2020033360A (ja) 2011-03-11 2019-10-10 ファブリー病の治療用投薬レジメン
JP2021018456A Withdrawn JP2021098697A (ja) 2011-03-11 2021-02-08 ファブリー病の治療用投薬レジメン
JP2023045001A Pending JP2023093448A (ja) 2011-03-11 2023-03-22 ファブリー病の治療用投薬レジメン
JP2025179237A Pending JP2026035578A (ja) 2011-03-11 2025-10-24 ファブリー病の治療用投薬レジメン

Country Status (16)

Country Link
US (4) US20140219986A1 (https=)
EP (2) EP3698792A1 (https=)
JP (6) JP2014528901A (https=)
KR (1) KR20140011367A (https=)
CN (2) CN107088225A (https=)
AU (6) AU2012229330B2 (https=)
CA (1) CA2829947C (https=)
CL (1) CL2013002601A1 (https=)
EA (1) EA031874B1 (https=)
ES (1) ES2807502T3 (https=)
HK (1) HK1242998A1 (https=)
MX (1) MX2013010446A (https=)
SG (2) SG10201604757RA (https=)
TW (3) TWI624259B (https=)
WO (1) WO2012125402A2 (https=)
ZA (1) ZA201306735B (https=)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018230628A1 (ja) 2017-06-14 2018-12-20 学校法人 明治薬科大学 ファブリー病治療用医薬の組合せ物及びその利用
JP2019510030A (ja) * 2016-03-22 2019-04-11 アミカス セラピューティックス インコーポレイテッド Ｇｌａ遺伝子にｇ９３３１ａ変異を有する患者においてファブリー病を処置する方法
JP2020510909A (ja) * 2017-02-09 2020-04-09 コグノア，インク． デジタル個別化医療のためのプラットフォームとシステム
JP2022536687A (ja) * 2019-06-11 2022-08-18 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有する患者のファブリー病を治療する方法
JP2024084670A (ja) * 2022-12-13 2024-06-25 アミカス セラピューティックス インコーポレイテッド ミガラスタットの薬物動態を改善する方法

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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
UY34317A (es) 2011-09-12 2013-02-28 Genzyme Corp Anticuerpo antireceptor de célula T (alfa)/ß
US9790268B2 (en) 2012-09-12 2017-10-17 Genzyme Corporation Fc containing polypeptides with altered glycosylation and reduced effector function
US9701753B2 (en) 2013-03-11 2017-07-11 Genzyme Corporation Hyperglycosylated binding polypeptides
EP3108002A1 (en) * 2014-02-17 2016-12-28 GlaxoSmithKline Intellectual Property Development Limited Microbiological process
WO2015143091A1 (en) 2014-03-19 2015-09-24 Genzyme Corporation Site-specific glycoengineering of targeting moieties
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2017027709A1 (en) 2015-08-11 2017-02-16 Cognoa, Inc. Methods and apparatus to determine developmental progress with artificial intelligence and user input
US11972336B2 (en) 2015-12-18 2024-04-30 Cognoa, Inc. Machine learning platform and system for data analysis
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
EP3539033B1 (en) 2016-11-14 2024-10-02 Cognoa, Inc. Methods and apparatus for evaluating developmental conditions and providing control over coverage and reliability
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
RS65230B1 (sr) * 2017-05-30 2024-03-29 Amicus Therapeutics Inc Migalastat za lečenje pacijenata koji boluju od fabrijeve bolesti sa oštećenjem bubrega
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
KR102785528B1 (ko) 2019-03-22 2025-03-21 코그노아, 인크. 개인 맞춤식 디지털 치료 방법 및 디바이스
CN120192414A (zh) 2019-04-03 2025-06-24 建新公司 具有降低的断裂的抗αβTCR结合多肽
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
AU2020343020A1 (en) 2019-09-06 2022-03-31 Cognoa, Inc. Methods, systems, and devices for the diagnosis of behavioral disorders, developmental delays, and neurologic impairments
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
US20240120050A1 (en) * 2022-10-07 2024-04-11 Insight Direct Usa, Inc. Machine learning method for predicting a health outcome of a patient using video and audio analytics
TR2024011149A1 (tr) * 2024-08-23 2026-03-23 Sanovel İlaç Sanayi̇ Ve Ti̇caret Anoni̇m Şi̇rketi̇ Mi̇galastat i̇çeren kapsül formülasyonlari
TR2024011147A1 (tr) 2024-08-23 2026-03-23 Sanovel İlaç Sanayi̇ Ve Ti̇caret Anoni̇m Şi̇rketi̇ Mi̇galastat i̇çeren kapsül formülasyonu

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JP2003528799A (ja) * 1998-06-01 2003-09-30 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー リソソームα−ガラクトシダーゼＡの増強方法
JP2006516645A (ja) * 2003-01-31 2006-07-06 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー タンパク質欠損性障害の治療のための併用療法
JP2009537149A (ja) * 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
US20100266571A1 (en) * 2007-04-26 2010-10-21 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US20100291060A1 (en) * 2007-08-29 2010-11-18 Shire Human Genetic Therapies, Inc Subcutaneous administration of alpha-galactosidase a

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CN100457106C (zh) * 2005-06-15 2009-02-04 同济大学 1-脱氧野尻霉素在制备治疗糖尿病肾病药物中的应用
MX2009010557A (es) * 2007-03-30 2009-11-19 Amicus Therapeutics Inc Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.

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JP2003528799A (ja) * 1998-06-01 2003-09-30 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー リソソームα−ガラクトシダーゼＡの増強方法
JP2006516645A (ja) * 2003-01-31 2006-07-06 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー タンパク質欠損性障害の治療のための併用療法
JP2009537149A (ja) * 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
US20100266571A1 (en) * 2007-04-26 2010-10-21 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US20100291060A1 (en) * 2007-08-29 2010-11-18 Shire Human Genetic Therapies, Inc Subcutaneous administration of alpha-galactosidase a

Cited By (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019510030A (ja) * 2016-03-22 2019-04-11 アミカス セラピューティックス インコーポレイテッド Ｇｌａ遺伝子にｇ９３３１ａ変異を有する患者においてファブリー病を処置する方法
JP7098529B2 (ja) 2016-03-22 2022-07-11 アミカス セラピューティックス インコーポレイテッド Ｇｌａ遺伝子にｇ９３３１ａ変異を有する患者においてファブリー病を処置する方法
JP2020510909A (ja) * 2017-02-09 2020-04-09 コグノア，インク． デジタル個別化医療のためのプラットフォームとシステム
JP7324709B2 (ja) 2017-02-09 2023-08-10 コグノア，インク． デジタル個別化医療のためのプラットフォームとシステム
WO2018230628A1 (ja) 2017-06-14 2018-12-20 学校法人 明治薬科大学 ファブリー病治療用医薬の組合せ物及びその利用
JP2022536687A (ja) * 2019-06-11 2022-08-18 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有する患者のファブリー病を治療する方法
JP7677910B2 (ja) 2019-06-11 2025-05-15 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有する患者のファブリー病を治療する方法
JP2024084670A (ja) * 2022-12-13 2024-06-25 アミカス セラピューティックス インコーポレイテッド ミガラスタットの薬物動態を改善する方法
JP7757345B2 (ja) 2022-12-13 2025-10-21 アミカス セラピューティックス インコーポレイテッド ミガラスタットの薬物動態を改善する方法

Also Published As

Publication number Publication date
JP2021098697A (ja) 2021-07-01
WO2012125402A3 (en) 2014-05-01
US20180153999A1 (en) 2018-06-07
TW201302197A (zh) 2013-01-16
AU2020201715A1 (en) 2020-04-09
JP2017132780A (ja) 2017-08-03
HK1242998A1 (zh) 2018-07-06
CN103974619B (zh) 2017-02-15
CA2829947C (en) 2019-01-15
US20240115708A1 (en) 2024-04-11
ZA201306735B (en) 2014-07-30
AU2024205251A1 (en) 2024-08-22
SG193379A1 (en) 2013-10-30
ES2807502T3 (es) 2021-02-23
KR20140011367A (ko) 2014-01-28
JP2026035578A (ja) 2026-03-04
EP2683382B1 (en) 2020-04-22
JP2023093448A (ja) 2023-07-04
SG10201604757RA (en) 2016-08-30
WO2012125402A2 (en) 2012-09-20
TW201634048A (zh) 2016-10-01
AU2012229330B2 (en) 2016-04-21
EP2683382A4 (en) 2014-10-22
AU2016204445B2 (en) 2018-03-22
TW201740945A (zh) 2017-12-01
EA201301018A1 (ru) 2014-04-30
JP2020033360A (ja) 2020-03-05
CA2829947A1 (en) 2012-09-20
AU2018201637A1 (en) 2018-04-05
CN103974619A (zh) 2014-08-06
CL2013002601A1 (es) 2014-03-28
NZ615726A (en) 2015-01-30
US20140219986A1 (en) 2014-08-07
MX2013010446A (es) 2014-03-27
EA031874B1 (ru) 2019-03-29
AU2016204445A1 (en) 2016-07-21
AU2012229330A1 (en) 2013-10-03
US20200268890A1 (en) 2020-08-27
TWI624259B (zh) 2018-05-21
EP3698792A1 (en) 2020-08-26
EP2683382A2 (en) 2014-01-15
CN107088225A (zh) 2017-08-25
AU2022203011A1 (en) 2022-05-26
TWI624258B (zh) 2018-05-21

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