MX349992B - Composiciones de alta concentracion de alfa-glucosidasa para el tratamiento de la enfermedad de pompe. - Google Patents
Composiciones de alta concentracion de alfa-glucosidasa para el tratamiento de la enfermedad de pompe.Info
- Publication number
- MX349992B MX349992B MX2014010669A MX2014010669A MX349992B MX 349992 B MX349992 B MX 349992B MX 2014010669 A MX2014010669 A MX 2014010669A MX 2014010669 A MX2014010669 A MX 2014010669A MX 349992 B MX349992 B MX 349992B
- Authority
- MX
- Mexico
- Prior art keywords
- glucosidase
- pompe disease
- compositions
- treatment
- high concentration
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 4
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 title abstract 3
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 title abstract 2
- 206010053185 Glycogen storage disease type II Diseases 0.000 title abstract 2
- 201000004502 glycogen storage disease II Diseases 0.000 title abstract 2
- 108010028144 alpha-Glucosidases Proteins 0.000 title 1
- 239000002253 acid Substances 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 3
- 102000004190 Enzymes Human genes 0.000 abstract 1
- 108090000790 Enzymes Proteins 0.000 abstract 1
- 108010006519 Molecular Chaperones Proteins 0.000 abstract 1
- 238000009472 formulation Methods 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
Classifications
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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
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
-
- 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
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
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- 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)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
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- 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
- C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
-
- 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)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Neurosurgery (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente invención se refiere a composiciones que comprenden concentraciones altas de a-glucosidasa ácida en combinación con una chaperona activa específica para el sitio para la a-glucosidasa ácida y métodos para tratar la enfermedad de Pompe en un individuo que lo necesita, que incluye un método para administrar al individuo dichas composiciones. La presente solicitud proporciona además métodos para aumentar la estabilidad in vitro e in vivo de una formulación enzimática de a-glucosidasa ácida.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261607920P | 2012-03-07 | 2012-03-07 | |
US201361750718P | 2013-01-09 | 2013-01-09 | |
PCT/US2013/029660 WO2013134530A1 (en) | 2012-03-07 | 2013-03-07 | High concentration alpha-glucosidase compositions for the treatment of pompe disease |
Publications (2)
Publication Number | Publication Date |
---|---|
MX2014010669A MX2014010669A (es) | 2014-10-17 |
MX349992B true MX349992B (es) | 2017-08-22 |
Family
ID=49117351
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2014010669A MX349992B (es) | 2012-03-07 | 2013-03-07 | Composiciones de alta concentracion de alfa-glucosidasa para el tratamiento de la enfermedad de pompe. |
Country Status (7)
Country | Link |
---|---|
US (6) | US9404100B2 (es) |
EP (1) | EP2823043A4 (es) |
JP (4) | JP6236406B2 (es) |
KR (5) | KR20200032244A (es) |
CA (1) | CA2866683A1 (es) |
MX (1) | MX349992B (es) |
WO (1) | WO2013134530A1 (es) |
Families Citing this family (28)
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KR20200032244A (ko) * | 2012-03-07 | 2020-03-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
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US10945984B2 (en) | 2012-08-22 | 2021-03-16 | Arbor Pharmaceuticals, Llc | Methods of administering monomethyl fumarate and prodrugs thereof having reduced side effects |
CA2897432A1 (en) * | 2013-01-09 | 2014-07-17 | Amicus Therapeutics, Inc. | Stable parenteral dnj compositions |
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US10179118B2 (en) | 2013-03-24 | 2019-01-15 | Arbor Pharmaceuticals, Llc | Pharmaceutical compositions of dimethyl fumarate |
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CA3007152A1 (en) * | 2015-12-07 | 2017-06-15 | Erasmus University Medical Center Rotterdam | Enzymatic replacement therapy and antisense therapy for pompe disease |
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KR20220145918A (ko) | 2016-03-30 | 2022-10-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
US10512676B2 (en) | 2016-03-30 | 2019-12-24 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
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US20210393747A1 (en) * | 2017-05-15 | 2021-12-23 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
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US20220331408A1 (en) * | 2019-07-09 | 2022-10-20 | Genethon | Treatment of glycogen storage disease (gsd) |
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2013
- 2013-03-07 KR KR1020207007487A patent/KR20200032244A/ko not_active Application Discontinuation
- 2013-03-07 KR KR1020237039814A patent/KR20230164207A/ko active Application Filing
- 2013-03-07 KR KR1020237014919A patent/KR20230066482A/ko not_active Application Discontinuation
- 2013-03-07 KR KR1020217015348A patent/KR20210062731A/ko not_active Application Discontinuation
- 2013-03-07 CA CA2866683A patent/CA2866683A1/en active Pending
- 2013-03-07 US US14/379,131 patent/US9404100B2/en active Active
- 2013-03-07 JP JP2014561115A patent/JP6236406B2/ja active Active
- 2013-03-07 KR KR1020147027104A patent/KR20140135222A/ko not_active Application Discontinuation
- 2013-03-07 EP EP13757576.7A patent/EP2823043A4/en not_active Ceased
- 2013-03-07 MX MX2014010669A patent/MX349992B/es active IP Right Grant
- 2013-03-07 WO PCT/US2013/029660 patent/WO2013134530A1/en active Application Filing
- 2013-12-13 US US14/106,670 patent/US9303249B2/en active Active
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2016
- 2016-06-24 US US15/192,137 patent/US10046033B2/en active Active
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2017
- 2017-08-21 JP JP2017158641A patent/JP6875955B2/ja active Active
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2018
- 2018-06-22 US US16/015,556 patent/US10512677B2/en active Active
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2019
- 2019-09-24 US US16/580,541 patent/US11278599B2/en active Active
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2020
- 2020-04-24 JP JP2020077151A patent/JP6952831B2/ja active Active
-
2021
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US20180360928A1 (en) | 2018-12-20 |
JP2022008596A (ja) | 2022-01-13 |
KR20210062731A (ko) | 2021-05-31 |
KR20140135222A (ko) | 2014-11-25 |
US11278599B2 (en) | 2022-03-22 |
JP6952831B2 (ja) | 2021-10-27 |
US20170056483A1 (en) | 2017-03-02 |
US9404100B2 (en) | 2016-08-02 |
KR20200032244A (ko) | 2020-03-25 |
US20220347272A1 (en) | 2022-11-03 |
JP6236406B2 (ja) | 2017-11-22 |
MX2014010669A (es) | 2014-10-17 |
JP6875955B2 (ja) | 2021-05-26 |
KR20230164207A (ko) | 2023-12-01 |
US20200188494A1 (en) | 2020-06-18 |
JP2020143071A (ja) | 2020-09-10 |
WO2013134530A1 (en) | 2013-09-12 |
JP2015509542A (ja) | 2015-03-30 |
US10512677B2 (en) | 2019-12-24 |
JP2018027945A (ja) | 2018-02-22 |
US20150044194A1 (en) | 2015-02-12 |
EP2823043A4 (en) | 2016-09-07 |
US10046033B2 (en) | 2018-08-14 |
US9303249B2 (en) | 2016-04-05 |
CA2866683A1 (en) | 2013-09-12 |
EP2823043A1 (en) | 2015-01-14 |
KR20230066482A (ko) | 2023-05-15 |
US20140193390A1 (en) | 2014-07-10 |
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