HK1242998A1 - 治疗法布里病的给药方案 - Google Patents

治疗法布里病的给药方案 Download PDF

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Publication number
HK1242998A1
HK1242998A1 HK18102615.1A HK18102615A HK1242998A1 HK 1242998 A1 HK1242998 A1 HK 1242998A1 HK 18102615 A HK18102615 A HK 18102615A HK 1242998 A1 HK1242998 A1 HK 1242998A1
Authority
HK
Hong Kong
Prior art keywords
gal
enzyme
acase
migalastat
activity
Prior art date
Application number
HK18102615.1A
Other languages
English (en)
Chinese (zh)
Inventor
D.S.格林
K.J.瓦伦扎诺
K．J．瓦倫扎諾
Original Assignee
阿米库斯治疗学公司
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.)
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Publication date
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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=HK1242998(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 阿米库斯治疗学公司 filed Critical 阿米库斯治疗学公司
Publication of HK1242998A1 publication Critical patent/HK1242998A1/zh

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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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Obesity (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Cardiology (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)
HK18102615.1A 2011-03-11 2018-02-23 治疗法布里病的给药方案 HK1242998A1 (zh)

Applications Claiming Priority (6)

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

Publications (1)

Publication Number Publication Date
HK1242998A1 true HK1242998A1 (zh) 2018-07-06

Family

ID=46831259

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18102615.1A HK1242998A1 (zh) 2011-03-11 2018-02-23 治疗法布里病的给药方案

Country Status (16)

Country Link
US (4) US20140219986A1 (enExample)
EP (2) EP3698792A1 (enExample)
JP (5) JP2014528901A (enExample)
KR (1) KR20140011367A (enExample)
CN (2) CN103974619B (enExample)
AU (6) AU2012229330B2 (enExample)
CA (1) CA2829947C (enExample)
CL (1) CL2013002601A1 (enExample)
EA (1) EA031874B1 (enExample)
ES (1) ES2807502T3 (enExample)
HK (1) HK1242998A1 (enExample)
MX (1) MX2013010446A (enExample)
SG (2) SG10201604757RA (enExample)
TW (3) TWI624258B (enExample)
WO (1) WO2012125402A2 (enExample)
ZA (1) ZA201306735B (enExample)

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CA2652553C (en) 2006-05-16 2020-08-18 Amicus Therapeutics, Inc. Use of 1-deoxygalactonojirimycin or a salt thereof in the treatment of fabry disease
LT3470077T (lt) 2008-02-12 2021-02-25 Amicus Therapeutics, Inc. Atsako į ligų gydymą farmakologiniu šaperonu prognozavimo būdas
UY34317A (es) 2011-09-12 2013-02-28 Genzyme Corp Anticuerpo antireceptor de célula T (alfa)/ß
IL304738B2 (en) 2013-03-11 2025-08-01 Genzyme Corp Hyperglycosylated binding polypeptides
EP3108002A1 (en) * 2014-02-17 2016-12-28 GlaxoSmithKline Intellectual Property Development Limited Microbiological process
ES2940903T3 (es) 2014-03-19 2023-05-12 Genzyme Corp Glucomanipulación específica del sitio de restos orientadores
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
JP7001593B2 (ja) 2015-08-11 2022-01-19 コグノア， インコーポレイテッド 人工知能およびユーザ入力を用いて発達進度を判定するための方法および装置
US11972336B2 (en) 2015-12-18 2024-04-30 Cognoa, Inc. Machine learning platform and system for data analysis
KR20190087673A (ko) * 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
EP4378524A3 (en) * 2016-07-19 2024-08-14 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
CN118609834A (zh) 2016-11-14 2024-09-06 科格诺亚公司 用于评估发育状况并提供覆盖度和可靠性控制的方法和装置
AU2018219846A1 (en) * 2017-02-09 2019-09-12 Cognoa, Inc. Platform and system for digital personalized medicine
WO2018222655A1 (en) 2017-05-30 2018-12-06 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
TW202322814A (zh) * 2017-05-30 2023-06-16 美商阿米庫斯醫療股份有限公司 治療有腎損傷的法布里患者的方法
JP7066157B2 (ja) 2017-06-14 2022-05-13 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
WO2019157047A1 (en) * 2018-02-06 2019-08-15 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
KR20210046729A (ko) * 2018-08-20 2021-04-28 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
BR112021018770A2 (pt) 2019-03-22 2022-02-15 Cognoa Inc Métodos e dispositivos de terapia digital personalizada
CN113939534B (zh) 2019-04-03 2025-02-28 建新公司 具有降低的断裂的抗αβTCR结合多肽
CA3141226A1 (en) * 2019-06-11 2020-12-17 Franklin Johnson Methods of treating fabry disease in patients having renal impairment
US11833164B2 (en) 2019-08-07 2023-12-05 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
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
US20240197706A1 (en) * 2022-12-13 2024-06-20 Amicus Therapeutics, Inc. Methods of improving the pharmacokinetics of migalastat

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4551177A (en) * 1984-04-23 1985-11-05 National Starch And Chemical Corporation Compressible starches as binders for tablets or capsules
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
ES2619353T3 (es) * 2003-01-31 2017-06-26 Mount Sinai School Of Medicine Of New York University Terapia de combinación para tratar trastornos de deficiencia de proteínas
US7879354B2 (en) * 2004-01-13 2011-02-01 Mcneil-Ppc, Inc. Rapidly disintegrating gelatinous coated tablets
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
CA2652553C (en) * 2006-05-16 2020-08-18 Amicus Therapeutics, Inc. Use of 1-deoxygalactonojirimycin or a salt thereof in the treatment of fabry disease
EP2142197A4 (en) * 2007-03-30 2010-11-10 Amicus Therapeutics Inc PROCESS FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONS
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.
EP2185187A1 (en) * 2007-08-29 2010-05-19 Shire Human Genetic Therapies, Inc. Subcutaneous administration of alpha-galatosidase a

Also Published As

Publication number Publication date
EP2683382A2 (en) 2014-01-15
SG10201604757RA (en) 2016-08-30
EP2683382B1 (en) 2020-04-22
TW201634048A (zh) 2016-10-01
CA2829947A1 (en) 2012-09-20
WO2012125402A2 (en) 2012-09-20
AU2020201715A1 (en) 2020-04-09
JP2021098697A (ja) 2021-07-01
AU2016204445B2 (en) 2018-03-22
EP3698792A1 (en) 2020-08-26
JP2020033360A (ja) 2020-03-05
AU2022203011A1 (en) 2022-05-26
AU2024205251A1 (en) 2024-08-22
MX2013010446A (es) 2014-03-27
TWI624258B (zh) 2018-05-21
NZ615726A (en) 2015-01-30
US20140219986A1 (en) 2014-08-07
CN103974619B (zh) 2017-02-15
ZA201306735B (en) 2014-07-30
SG193379A1 (en) 2013-10-30
US20200268890A1 (en) 2020-08-27
CN107088225A (zh) 2017-08-25
AU2012229330B2 (en) 2016-04-21
KR20140011367A (ko) 2014-01-28
EA201301018A1 (ru) 2014-04-30
TW201740945A (zh) 2017-12-01
JP2017132780A (ja) 2017-08-03
JP2023093448A (ja) 2023-07-04
EA031874B1 (ru) 2019-03-29
CN103974619A (zh) 2014-08-06
JP2014528901A (ja) 2014-10-30
US20180153999A1 (en) 2018-06-07
AU2016204445A1 (en) 2016-07-21
EP2683382A4 (en) 2014-10-22
TW201302197A (zh) 2013-01-16
ES2807502T3 (es) 2021-02-23
CA2829947C (en) 2019-01-15
TWI624259B (zh) 2018-05-21
AU2012229330A1 (en) 2013-10-03
WO2012125402A3 (en) 2014-05-01
CL2013002601A1 (es) 2014-03-28
US20240115708A1 (en) 2024-04-11
AU2018201637A1 (en) 2018-04-05

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