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

Info

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
Authority
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

Links

Classifications

    • 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 (https=)
EP (1) EP3565583A4 (https=)
JP (2) JP2020504131A (https=)
KR (2) KR20190103320A (https=)
CN (2) CN110381987A (https=)
AU (1) AU2018205891B2 (https=)
BR (1) BR112019013920A2 (https=)
CA (1) CA3048151A1 (https=)
CL (1) CL2019001867A1 (https=)
IL (1) IL267863A (https=)
MX (1) MX2019008076A (https=)
NZ (1) NZ755725A (https=)
WO (1) WO2018127920A1 (https=)
ZA (1) ZA201904850B (https=)

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
JP2023549323A (ja) * 2020-11-13 2023-11-24 ハンミ ファーマシューティカル カンパニー リミテッド 治療学的酵素融合タンパク質のファブリー病に起因する又は伴う腎臓疾患の予防及び治療用途

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
EP1503788B1 (en) * 2002-04-25 2011-06-29 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency
EP1877099B1 (en) * 2005-04-06 2012-09-19 Genzyme Corporation Therapeutic conjugates comprising a lysosomal enzyme, polysialic acid and a targeting moiety
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2192924B1 (en) 2007-08-20 2017-10-11 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
WO2011107990A1 (en) 2010-03-02 2011-09-09 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
WO2011061736A1 (en) 2009-11-17 2011-05-26 Protalix Ltd. Alkaline alpha galactosidase for the treatment of fabry disease
CN103443270B (zh) * 2011-01-20 2017-06-06 普罗塔里克斯有限公司 用于在植物和植物细胞中表达α‑半乳糖苷酶的核酸构建体
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
EP3565583A1 (en) 2019-11-13
IL267863A (en) 2019-09-26
ZA201904850B (en) 2022-04-28
RU2019124280A (ru) 2021-02-06
CN110381987A (zh) 2019-10-25
JP2023022244A (ja) 2023-02-14
RU2019124280A3 (https=) 2021-09-14
JP2020504131A (ja) 2020-02-06
CA3048151A1 (en) 2018-07-12
US20250134968A1 (en) 2025-05-01
NZ755725A (en) 2023-06-30
KR20190103320A (ko) 2019-09-04
US12194079B2 (en) 2025-01-14
MX2019008076A (es) 2019-08-29
AU2018205891B2 (en) 2024-11-07
CL2019001867A1 (es) 2019-10-04
EP3565583A4 (en) 2020-12-02
KR20240042110A (ko) 2024-04-01
WO2018127920A1 (en) 2018-07-12
AU2018205891A1 (en) 2019-08-15
CN117959455A (zh) 2024-05-03
US20200155654A1 (en) 2020-05-21

Similar Documents

Publication Publication Date Title
US20250134968A1 (en) Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase
Townsend et al. Chronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogs
ES2931054T3 (es) Métodos para potenciar y/o estabilizar la función cardíaca en pacientes con enfermedad de Fabry
ES2858726T3 (es) Administración de agentes terapéuticos al SNC
RU2751235C2 (ru) Терапевтические слитые белки направленного действия на основе лизосомных ферментов, соответствующие составы и их применения
ES2993833T3 (en) Migalastat for use in methods of treating fabry disease in patients having a mutation in the gla gene
JP2010523578A (ja) 薬理シャペロンを用いるファブリー病の治療方法
TW201302197A (zh) 治療法布裡病之給藥方案
US20150306187A1 (en) Subcutaneous administration of alpha-galactosidase a
ES2973767T3 (es) Tratamiento de enfermedades inflamatorias con inhibidores del complemento
RU2793184C2 (ru) СХЕМА ЛЕЧЕНИЯ ДЛЯ ЛЕЧЕНИЯ БОЛЕЗНИ ФАБРИ С ИСПОЛЬЗОВАНИЕМ СТАБИЛИЗИРОВАННОЙ α-ГАЛАКТОЗИДАЗЫ
JP2022518733A (ja) ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用
CA3015358C (en) Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
RU2783380C2 (ru) Способы и композиции для доставки в цнс арилсульфатазы а
HK40001779A (en) Improved naglu fusion protein formulation
HK40001779B (en) Improved naglu fusion protein formulation
BR112020003974B1 (pt) Métodos para intensificar e/ou estabilizar função cardíaca em pacientes com doença de fabry
Raiman et al. 115. The use of hematopoietic stem cell transplantation in mucopolysaccharidosis type I Hurler: A single center experience

Legal Events

Date Code Title Description
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]