MX2019008076A - Regimen terapeutico para el tratamiento de fabry usando alfa-galactosidasa estabilizada. - Google Patents

Regimen terapeutico para el tratamiento de fabry usando alfa-galactosidasa estabilizada.

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Publication number
MX2019008076A
MX2019008076A MX2019008076A MX2019008076A MX2019008076A MX 2019008076 A MX2019008076 A MX 2019008076A MX 2019008076 A MX2019008076 A MX 2019008076A MX 2019008076 A MX2019008076 A MX 2019008076A MX 2019008076 A MX2019008076 A MX 2019008076A
Authority
MX
Mexico
Prior art keywords
galactosidase
fabry
treatment
therapeutic regimen
alpha
Prior art date
Application number
MX2019008076A
Other languages
English (en)
Inventor
Almon Einat
CHERTKOFF Raul
Alon Sari
SHAALTIERL Yoseph
Original Assignee
Protalix Ltd
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
Application filed by Protalix Ltd filed Critical Protalix Ltd
Publication of MX2019008076A publication Critical patent/MX2019008076A/es

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    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61K9/08—Solutions
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

Se desvelan en el presente documento, procedimientos de tratamiento de la enfermedad de Fabry a través de la administración de proteína alfa galactosidasa humana recombinante de procedencia vegetal estabilizada que comprende al menos dos monómeros de alfa galactosidasa que se unen covalentemente entre sí a través de una fracción de enlace, y las dosis unitarias de proteína. Los protocolos desvelados son seguros, tienen intervalos de más de 2 semanas entre las administraciones y muestran una mejora importante en los parámetros de enfermedad del paciente, en términos de reducción de la acumulación de Gb3, dolor y parámetros GI, estabilización renal y cardíaca en el entorno clínico.
MX2019008076A 2017-01-05 2018-01-05 Regimen terapeutico para el tratamiento de fabry usando alfa-galactosidasa estabilizada. MX2019008076A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762442537P 2017-01-05 2017-01-05
PCT/IL2018/050018 WO2018127920A1 (en) 2017-01-05 2018-01-05 Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase

Publications (1)

Publication Number Publication Date
MX2019008076A true MX2019008076A (es) 2019-08-29

Family

ID=62790830

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019008076A MX2019008076A (es) 2017-01-05 2018-01-05 Regimen terapeutico para el tratamiento de fabry usando alfa-galactosidasa estabilizada.

Country Status (14)

Country Link
US (1) US20200155654A1 (es)
EP (1) EP3565583A4 (es)
JP (2) JP2020504131A (es)
KR (2) KR20240042110A (es)
CN (2) CN110381987A (es)
AU (1) AU2018205891A1 (es)
BR (1) BR112019013920A2 (es)
CA (1) CA3048151A1 (es)
CL (1) CL2019001867A1 (es)
IL (1) IL267863A (es)
MX (1) MX2019008076A (es)
NZ (1) NZ755725A (es)
WO (1) WO2018127920A1 (es)
ZA (1) ZA201904850B (es)

Families Citing this family (1)

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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2361631A1 (en) * 2002-04-25 2011-08-31 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency
US7341720B2 (en) * 2005-04-06 2008-03-11 Genzyme Corporation Targeting of glycoprotein therapeutics
US9999618B2 (en) * 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US9194011B2 (en) * 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
ES2606532T3 (es) * 2010-03-02 2017-03-24 Protalix Ltd. Alfa-galactosidasa estabilizada y usos de la misma
AU2011356137A1 (en) * 2011-01-20 2013-08-15 Protalix Ltd. Nucleic acid construct for expression of alpha-galactosidase in plants and plant cells
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease

Also Published As

Publication number Publication date
US20200155654A1 (en) 2020-05-21
KR20190103320A (ko) 2019-09-04
CN110381987A (zh) 2019-10-25
CL2019001867A1 (es) 2019-10-04
JP2020504131A (ja) 2020-02-06
RU2019124280A (ru) 2021-02-06
RU2019124280A3 (es) 2021-09-14
ZA201904850B (en) 2022-04-28
AU2018205891A1 (en) 2019-08-15
NZ755725A (en) 2023-06-30
EP3565583A4 (en) 2020-12-02
JP2023022244A (ja) 2023-02-14
WO2018127920A1 (en) 2018-07-12
CA3048151A1 (en) 2018-07-12
EP3565583A1 (en) 2019-11-13
KR20240042110A (ko) 2024-04-01
CN117959455A (zh) 2024-05-03
IL267863A (en) 2019-09-26
BR112019013920A2 (pt) 2020-02-04

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