CL2013002601A1 - Uso de 1-desoxigalactonojirimicina y una terapia de reemplazo de enzimas con alfa-gal a para tratar la enfermedad de fabry. - Google Patents

Uso de 1-desoxigalactonojirimicina y una terapia de reemplazo de enzimas con alfa-gal a para tratar la enfermedad de fabry.

Info

Publication number
CL2013002601A1
CL2013002601A1 CL2013002601A CL2013002601A CL2013002601A1 CL 2013002601 A1 CL2013002601 A1 CL 2013002601A1 CL 2013002601 A CL2013002601 A CL 2013002601A CL 2013002601 A CL2013002601 A CL 2013002601A CL 2013002601 A1 CL2013002601 A1 CL 2013002601A1
Authority
CL
Chile
Prior art keywords
deoxigalactonojirimycin
gal
alpha
replacement therapy
fabry disease
Prior art date
Application number
CL2013002601A
Other languages
English (en)
Inventor
Douglas Stuart Greene
Kenneth Joseph Valenzano
Original Assignee
Amicus Therapeutics Inc
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.)
Filing date
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=CL2013002601(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of CL2013002601A1 publication Critical patent/CL2013002601A1/es

Links

Classifications

    • 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
    • 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
    • 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (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)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Biochemistry (AREA)
  • Hematology (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)
CL2013002601A 2011-03-11 2013-09-10 Uso de 1-desoxigalactonojirimicina y una terapia de reemplazo de enzimas con alfa-gal a para tratar la enfermedad de fabry. CL2013002601A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161451798P 2011-03-11 2011-03-11
US201161578201P 2011-12-20 2011-12-20
US201261596165P 2012-02-07 2012-02-07

Publications (1)

Publication Number Publication Date
CL2013002601A1 true CL2013002601A1 (es) 2014-03-28

Family

ID=46831259

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2013002601A CL2013002601A1 (es) 2011-03-11 2013-09-10 Uso de 1-desoxigalactonojirimicina y una terapia de reemplazo de enzimas con alfa-gal a para tratar la enfermedad de fabry.

Country Status (16)

Country Link
US (4) US20140219986A1 (es)
EP (2) EP2683382B1 (es)
JP (5) JP2014528901A (es)
KR (1) KR20140011367A (es)
CN (2) CN107088225A (es)
AU (6) AU2012229330B2 (es)
CA (1) CA2829947C (es)
CL (1) CL2013002601A1 (es)
EA (1) EA031874B1 (es)
ES (1) ES2807502T3 (es)
HK (1) HK1242998A1 (es)
MX (1) MX2013010446A (es)
SG (2) SG193379A1 (es)
TW (3) TWI624259B (es)
WO (1) WO2012125402A2 (es)
ZA (1) ZA201306735B (es)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2709445T3 (es) 2006-05-16 2019-04-16 Amicus Therapeutics Inc Opciones de tratamiento para la enfermedad de Fabry
PT3470077T (pt) 2008-02-12 2020-11-30 Amicus Therapeutics Inc Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas
UY34317A (es) 2011-09-12 2013-02-28 Genzyme Corp Anticuerpo antireceptor de célula T (alfa)/ß
SG11201506088RA (en) 2013-03-11 2015-09-29 Genzyme Corp Hyperglycosylated binding polypeptides
US10077458B2 (en) * 2014-02-17 2018-09-18 Glaxosmithkline Intellectual Property Development Limited Microbiological process
HUE061339T2 (hu) 2014-03-19 2023-06-28 Genzyme Corp Targetáló csoportok helyspecifikus glikotechnikai átalakítása
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
WO2017165164A1 (en) * 2016-03-22 2017-09-28 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
TW201806597A (zh) * 2016-07-19 2018-03-01 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
EP3580700A4 (en) * 2017-02-09 2020-11-18 Cognoa, Inc. PLATFORM AND SYSTEM FOR DIGITAL PERSONALIZED MEDICINE
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
EP4245366A3 (en) * 2017-05-30 2023-11-29 Amicus Therapeutics, Inc. Migalastat for treating fabry patients having renal impairment
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
EP3749307A1 (en) * 2018-02-06 2020-12-16 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4282472A3 (en) * 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
KR102643554B1 (ko) 2019-03-22 2024-03-04 코그노아, 인크. 개인 맞춤식 디지털 치료 방법 및 디바이스
TW202112372A (zh) * 2019-06-11 2021-04-01 美商阿米庫斯醫療股份有限公司 治療具有腎損害患者的Fabry氏病之方法
CN114423428A (zh) 2019-08-07 2022-04-29 阿米库斯治疗学公司 治疗在gla基因中具有突变的患者的法布里病的方法
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
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
CA2814774C (en) 2003-01-31 2016-03-22 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
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
ES2709445T3 (es) * 2006-05-16 2019-04-16 Amicus Therapeutics Inc Opciones de tratamiento para la enfermedad de Fabry
EP2142197A4 (en) * 2007-03-30 2010-11-10 Amicus Therapeutics Inc PROCESS FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONS
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2009032171A1 (en) * 2007-08-29 2009-03-12 Shire Human Genetic Therapies, Inc. Subcutaneous administration of alpha-galatosidase a

Also Published As

Publication number Publication date
EP2683382A4 (en) 2014-10-22
JP2017132780A (ja) 2017-08-03
EP2683382B1 (en) 2020-04-22
AU2024205251A1 (en) 2024-08-22
CN103974619A (zh) 2014-08-06
WO2012125402A3 (en) 2014-05-01
AU2016204445B2 (en) 2018-03-22
HK1242998A1 (zh) 2018-07-06
EP2683382A2 (en) 2014-01-15
ZA201306735B (en) 2014-07-30
JP2023093448A (ja) 2023-07-04
CA2829947A1 (en) 2012-09-20
AU2016204445A1 (en) 2016-07-21
AU2012229330A1 (en) 2013-10-03
AU2018201637A1 (en) 2018-04-05
SG10201604757RA (en) 2016-08-30
TW201302197A (zh) 2013-01-16
JP2014528901A (ja) 2014-10-30
TWI624258B (zh) 2018-05-21
CN107088225A (zh) 2017-08-25
EA201301018A1 (ru) 2014-04-30
SG193379A1 (en) 2013-10-30
CA2829947C (en) 2019-01-15
US20180153999A1 (en) 2018-06-07
AU2020201715A1 (en) 2020-04-09
TWI624259B (zh) 2018-05-21
CN103974619B (zh) 2017-02-15
EP3698792A1 (en) 2020-08-26
EA031874B1 (ru) 2019-03-29
KR20140011367A (ko) 2014-01-28
TW201634048A (zh) 2016-10-01
US20240115708A1 (en) 2024-04-11
NZ615726A (en) 2015-01-30
US20140219986A1 (en) 2014-08-07
US20200268890A1 (en) 2020-08-27
ES2807502T3 (es) 2021-02-23
WO2012125402A2 (en) 2012-09-20
MX2013010446A (es) 2014-03-27
AU2022203011A1 (en) 2022-05-26
AU2012229330B2 (en) 2016-04-21
JP2020033360A (ja) 2020-03-05
JP2021098697A (ja) 2021-07-01
TW201740945A (zh) 2017-12-01

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