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
Family has litigation
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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Zoology (AREA)
  • Obesity (AREA)
  • Wood Science & Technology (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (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 (cg-RX-API-DMAC7.html)
EP (2) EP2683382B1 (cg-RX-API-DMAC7.html)
JP (5) JP2014528901A (cg-RX-API-DMAC7.html)
KR (1) KR20140011367A (cg-RX-API-DMAC7.html)
CN (2) CN107088225A (cg-RX-API-DMAC7.html)
AU (6) AU2012229330B2 (cg-RX-API-DMAC7.html)
CA (1) CA2829947C (cg-RX-API-DMAC7.html)
CL (1) CL2013002601A1 (cg-RX-API-DMAC7.html)
EA (1) EA031874B1 (cg-RX-API-DMAC7.html)
ES (1) ES2807502T3 (cg-RX-API-DMAC7.html)
HK (1) HK1242998A1 (cg-RX-API-DMAC7.html)
MX (1) MX2013010446A (cg-RX-API-DMAC7.html)
SG (2) SG10201604757RA (cg-RX-API-DMAC7.html)
TW (3) TW201740945A (cg-RX-API-DMAC7.html)
WO (1) WO2012125402A2 (cg-RX-API-DMAC7.html)
ZA (1) ZA201306735B (cg-RX-API-DMAC7.html)

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DK2533050T6 (da) 2006-05-16 2015-09-21 Amicus Therapeutics Inc Behandlingsmuligheder ved Fabrys sygdom
HUE042882T2 (hu) 2008-02-12 2019-07-29 Amicus Therapeutics Inc Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére
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
WO2014164534A2 (en) 2013-03-11 2014-10-09 Genzyme Corporation Site-specific antibody-drug conjugation through glycoengineering
CN106687596A (zh) * 2014-02-17 2017-05-17 葛兰素史密斯克莱知识产权发展有限公司 微生物学方法
RS64072B1 (sr) 2014-03-19 2023-04-28 Genzyme Corp Mesto-specifični glikoinženjering ciljnih delova
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
EP3432882B1 (en) * 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
KR20240017126A (ko) * 2016-07-19 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 Ert-치료경험이 없는 환자 및 있는 환자에서 파브리병의 치료
WO2018090009A1 (en) 2016-11-14 2018-05-17 Cognoa, Inc. Methods and apparatus for evaluating developmental conditions and providing control over coverage and reliability
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
HUE069837T2 (hu) * 2017-05-30 2025-04-28 Amicus Therapeutics Inc Bizonyos mutációkat hordozó Fabry-kóros betegek kezelése
BR112019025083B1 (pt) 2017-05-30 2022-08-09 Amicus Therapeutics, Inc Métodos de tratamento de pacientes portadores de fabry com insuficiência renal
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
EP3749307B1 (en) * 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4299117A3 (en) * 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
AU2020248351B2 (en) 2019-03-22 2025-09-25 Cognoa, Inc. Personalized digital therapy methods and devices
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
EA202290024A1 (ru) * 2019-06-11 2022-03-14 Амикус Терапьютикс, Инк. Способы лечения болезни фабри у пациентов с почечной недостаточностью
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin 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)

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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
ES2686775T3 (es) * 2003-01-31 2018-10-19 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
DK2533050T6 (da) * 2006-05-16 2015-09-21 Amicus Therapeutics Inc Behandlingsmuligheder ved Fabrys sygdom
US20100113517A1 (en) * 2007-03-30 2010-05-06 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
US9056101B2 (en) * 2007-04-26 2015-06-16 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

Also Published As

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

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