CA2860085A1 - Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol - Google Patents

Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol Download PDF

Info

Publication number
CA2860085A1
CA2860085A1 CA2860085A CA2860085A CA2860085A1 CA 2860085 A1 CA2860085 A1 CA 2860085A1 CA 2860085 A CA2860085 A CA 2860085A CA 2860085 A CA2860085 A CA 2860085A CA 2860085 A1 CA2860085 A1 CA 2860085A1
Authority
CA
Canada
Prior art keywords
constituent
disease
lysosomal
ambroxol
pharmaceutically acceptable
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
CA2860085A
Other languages
English (en)
French (fr)
Inventor
Jan Lukas
Anahit PEWS-DAVTYAN
Matthias Beller
Arndt Rolfs
Muhammad Sharif
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Centogene IP GmbH
Original Assignee
Centogene IP GmbH
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 Centogene IP GmbH filed Critical Centogene IP GmbH
Publication of CA2860085A1 publication Critical patent/CA2860085A1/en
Abandoned legal-status Critical Current

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
    • A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • 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
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • 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
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/137—Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine or methadone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Emergency Medicine (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Hydrogenated Pyridines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CA2860085A 2011-12-22 2012-12-21 Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol Abandoned CA2860085A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP11010078.1 2011-12-22
EP11010078 2011-12-22
PCT/EP2012/005363 WO2013091897A1 (en) 2011-12-22 2012-12-21 Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol

Publications (1)

Publication Number Publication Date
CA2860085A1 true CA2860085A1 (en) 2013-06-27

Family

ID=47559375

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2860085A Abandoned CA2860085A1 (en) 2011-12-22 2012-12-21 Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol

Country Status (10)

Country Link
US (2) US20150258081A1 (https=)
EP (1) EP2793872B1 (https=)
JP (2) JP6320931B2 (https=)
KR (1) KR102076585B1 (https=)
AU (2) AU2012359165A1 (https=)
BR (1) BR112014014808A2 (https=)
CA (1) CA2860085A1 (https=)
ES (1) ES2864774T3 (https=)
IL (1) IL233173B (https=)
WO (1) WO2013091897A1 (https=)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
CN114540327B (zh) 2014-09-30 2026-03-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP6981961B2 (ja) * 2015-07-30 2021-12-17 ホライズン オルファン エルエルシー フコシダーゼ阻害剤
KR20250007693A (ko) 2015-12-30 2025-01-14 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
KR20190087673A (ko) 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US20190254992A1 (en) * 2016-06-29 2019-08-22 Hadasit Medical Research Services & Development Limited Combinations of beta-glycolipides and 4-[(2-amino-3,5-dibromophenyl)methylamino]cyclohexan-1-ol, compositions and uses thereof in the treatment of disorders associated with protein misfolding and protein aggregations
WO2018089797A1 (en) * 2016-11-14 2018-05-17 Mingwu Wang Formulations for the treatment of ocular surface diseases and related methods
CN110573492A (zh) * 2017-04-25 2019-12-13 阿米库斯治疗学公司 用于预防和/或治疗中枢神经系统退行性失调和/或溶酶体贮积失调的新颖组合物
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
US12594268B2 (en) 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
US11612619B2 (en) 2018-11-01 2023-03-28 National Institute Of Health (Nih), U.S. Dept. Of Health And Human Services (Dhhs), U.S. Government Compostions and methods for enabling cholesterol catabolism in human cells
EP3884383B1 (en) * 2018-12-21 2025-04-16 Shenzhen Yinwang Intelligent Technologies Co., Ltd. Data deterministic deliverable communication technology based on qos as a service
WO2020168294A1 (en) * 2019-02-15 2020-08-20 Lysosomal And Rare Disorders Research And Treatment Center, Llc Methods and compositions for treating lysosomal storage disorders: ambroxol as treatment agent for mucopolysaccharidoses iii (sanfilippo syndrome)
EP3996696A1 (en) * 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
US12544402B2 (en) 2020-02-28 2026-02-10 Repair Biotechnologies, Inc. Targeted expression of microbial cholesterol catalysis genes reduces excess lipid
CN112773779B (zh) * 2021-02-04 2022-08-16 上海交通大学医学院附属新华医院 氨溴索、千金藤和汉防已在治疗酸性鞘磷脂酶缺乏症中的应用
CN114644572B (zh) * 2022-03-22 2023-03-28 华南农业大学 一种溴己新半抗原、人工抗原、抗体及其制备方法和应用
KR20260002803A (ko) * 2023-05-02 2026-01-06 지위, 엘엘씨 치료적 사용을 위한 암브록솔의 변형된 형태

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19933148A1 (de) 1999-07-20 2001-01-25 Boehringer Ingelheim Int Ambroxolhaltige Lutschtablette
EP2857036B1 (en) * 2003-01-31 2016-12-21 Mount Sinai School of Medicine of New York University Combination therapy for treating protein deficiency disorders
PL2533050T6 (pl) * 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
JP5337405B2 (ja) * 2007-09-17 2013-11-06 ザ・ホスピタル・フォー・シック・チルドレン ゴーシェ病の治療方法
CA2718182A1 (en) * 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Treatment of pompe disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
EP2353403B1 (de) * 2010-02-01 2012-07-11 Symrise AG Verwendung von 1-(2,4-Dihydroxy-phenyl)-3-(3-hydroxy-4-methoxy-phenyl)-propan-1-on

Also Published As

Publication number Publication date
US20210228560A1 (en) 2021-07-29
AU2012359165A1 (en) 2014-07-10
EP2793872B1 (en) 2021-03-10
AU2017279780A1 (en) 2018-01-25
ES2864774T3 (es) 2021-10-14
IL233173A0 (en) 2014-07-31
KR20140107481A (ko) 2014-09-04
AU2017279780B2 (en) 2019-10-03
EP2793872A1 (en) 2014-10-29
KR102076585B1 (ko) 2020-02-12
WO2013091897A8 (en) 2014-10-09
BR112014014808A2 (pt) 2018-09-25
JP2015502964A (ja) 2015-01-29
IL233173B (en) 2020-10-29
JP2018076353A (ja) 2018-05-17
JP6320931B2 (ja) 2018-05-09
WO2013091897A1 (en) 2013-06-27
US20150258081A1 (en) 2015-09-17

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Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20171031

FZDE Discontinued

Effective date: 20220815