BR112019013920A2 - método para tratar doença de fabry, e, forma unitária de dosagem. - Google Patents

método para tratar doença de fabry, e, forma unitária de dosagem. Download PDF

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Publication number
BR112019013920A2
BR112019013920A2 BR112019013920A BR112019013920A BR112019013920A2 BR 112019013920 A2 BR112019013920 A2 BR 112019013920A2 BR 112019013920 A BR112019013920 A BR 112019013920A BR 112019013920 A BR112019013920 A BR 112019013920A BR 112019013920 A2 BR112019013920 A2 BR 112019013920A2
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BR
Brazil
Prior art keywords
galactosidase
human
recombinant human
administration
weeks
Prior art date
Application number
BR112019013920A
Other languages
English (en)
Portuguese (pt)
Inventor
Almon Einat
CHERTKOFF Raul
Alon Sari
Shaaltiel 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 BR112019013920A2 publication Critical patent/BR112019013920A2/pt

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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
    • A61K47/59Medicinal 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
    • A61K47/60Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465Hydrolases (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)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Vascular Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
BR112019013920A 2017-01-05 2018-01-05 método para tratar doença de fabry, e, forma unitária de dosagem. BR112019013920A2 (pt)

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
BR112019013920A2 true BR112019013920A2 (pt) 2020-02-04

Family

ID=62790830

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019013920A BR112019013920A2 (pt) 2017-01-05 2018-01-05 método para tratar doença de fabry, e, forma unitária de dosagem.

Country Status (14)

Country Link
US (2) US12194079B2 (enExample)
EP (1) EP3565583A4 (enExample)
JP (2) JP2020504131A (enExample)
KR (2) KR20190103320A (enExample)
CN (2) CN110381987A (enExample)
AU (1) AU2018205891B2 (enExample)
BR (1) BR112019013920A2 (enExample)
CA (1) CA3048151A1 (enExample)
CL (1) CL2019001867A1 (enExample)
IL (1) IL267863A (enExample)
MX (1) MX2019008076A (enExample)
NZ (1) NZ755725A (enExample)
WO (1) WO2018127920A1 (enExample)
ZA (1) ZA201904850B (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12020820B1 (en) * 2017-03-03 2024-06-25 Cerner Innovation, Inc. Predicting sphingolipidoses (fabry's disease) and decision support
AR120055A1 (es) * 2019-06-11 2022-02-02 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal
CN116419759A (zh) * 2020-11-13 2023-07-11 韩美药品株式会社 治疗酶融合蛋白在预防和治疗由法布里病引起或伴有法布里病的神经病中的用途
CN116419760A (zh) * 2020-11-13 2023-07-11 韩美药品株式会社 治疗酶融合蛋白在预防和治疗由法布里病引起或伴有法布里病的肾病中的用途

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5766897A (en) 1990-06-21 1998-06-16 Incyte Pharmaceuticals, Inc. Cysteine-pegylated proteins
CA2052728A1 (en) 1991-10-03 1993-04-04 Guy Ampleman Glycidyl azide polymer
JP5570677B2 (ja) * 2002-04-25 2014-08-13 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド α−ガラクトシダーゼA欠損症の治療
JP5137814B2 (ja) 2005-04-06 2013-02-06 ジェンザイム・コーポレーション 治療ターゲティングのための酸不安定リンカーを介するpegおよびポリシアルリソソーム酵素のコンジュゲート
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US8742079B2 (en) 2007-08-20 2014-06-03 Protalix Ltd. Saccharide-containing protein conjugates and uses thereof
US9194011B2 (en) * 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
US20120230974A1 (en) 2009-11-17 2012-09-13 Protalix Ltd Alkaline alpha galactosidase for the treatment of fabry disease
HUE030959T2 (en) 2010-03-02 2017-06-28 Protalix Ltd Stabilized alpha-galactosidase and its uses
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
NZ755725A (en) 2023-06-30
US20250134968A1 (en) 2025-05-01
CA3048151A1 (en) 2018-07-12
US12194079B2 (en) 2025-01-14
WO2018127920A1 (en) 2018-07-12
RU2019124280A (ru) 2021-02-06
CL2019001867A1 (es) 2019-10-04
KR20190103320A (ko) 2019-09-04
AU2018205891B2 (en) 2024-11-07
CN110381987A (zh) 2019-10-25
CN117959455A (zh) 2024-05-03
JP2020504131A (ja) 2020-02-06
JP2023022244A (ja) 2023-02-14
MX2019008076A (es) 2019-08-29
EP3565583A1 (en) 2019-11-13
EP3565583A4 (en) 2020-12-02
RU2019124280A3 (enExample) 2021-09-14
KR20240042110A (ko) 2024-04-01
IL267863A (en) 2019-09-26
US20200155654A1 (en) 2020-05-21
AU2018205891A1 (en) 2019-08-15
ZA201904850B (en) 2022-04-28

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B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]