JP6662789B2 - 脳アミロイドーシスを処置及び/又は予防する投与計画 - Google Patents

脳アミロイドーシスを処置及び/又は予防する投与計画 Download PDF

Info

Publication number
JP6662789B2
JP6662789B2 JP2016562965A JP2016562965A JP6662789B2 JP 6662789 B2 JP6662789 B2 JP 6662789B2 JP 2016562965 A JP2016562965 A JP 2016562965A JP 2016562965 A JP2016562965 A JP 2016562965A JP 6662789 B2 JP6662789 B2 JP 6662789B2
Authority
JP
Japan
Prior art keywords
period
addnj
enzyme
given
hours
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.)
Expired - Fee Related
Application number
JP2016562965A
Other languages
English (en)
Japanese (ja)
Other versions
JP2017511372A (ja
JP2017511372A5 (enExample
Inventor
アラン ワストマン,ブランドン
アラン ワストマン,ブランドン
Original Assignee
アミカス セラピューティックス インコーポレイテッド
アミカス セラピューティックス インコーポレイテッド
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 アミカス セラピューティックス インコーポレイテッド, アミカス セラピューティックス インコーポレイテッド filed Critical アミカス セラピューティックス インコーポレイテッド
Publication of JP2017511372A publication Critical patent/JP2017511372A/ja
Publication of JP2017511372A5 publication Critical patent/JP2017511372A5/ja
Application granted granted Critical
Publication of JP6662789B2 publication Critical patent/JP6662789B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2016562965A 2014-04-14 2015-04-14 脳アミロイドーシスを処置及び/又は予防する投与計画 Expired - Fee Related JP6662789B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461979408P 2014-04-14 2014-04-14
US61/979,408 2014-04-14
US14/684,967 2015-04-13
US14/684,967 US9675627B2 (en) 2014-04-14 2015-04-13 Dosing regimens for treating and/or preventing cerebral amyloidoses
PCT/US2015/025733 WO2015160796A1 (en) 2014-04-14 2015-04-14 Dosing regimens for treating and/or preventing cerebral amyloidoses

Publications (3)

Publication Number Publication Date
JP2017511372A JP2017511372A (ja) 2017-04-20
JP2017511372A5 JP2017511372A5 (enExample) 2019-05-09
JP6662789B2 true JP6662789B2 (ja) 2020-03-11

Family

ID=53015935

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2016562965A Expired - Fee Related JP6662789B2 (ja) 2014-04-14 2015-04-14 脳アミロイドーシスを処置及び/又は予防する投与計画

Country Status (10)

Country Link
US (2) US9675627B2 (enExample)
EP (1) EP3131547A1 (enExample)
JP (1) JP6662789B2 (enExample)
KR (1) KR20170003576A (enExample)
CN (1) CN107148270A (enExample)
AU (1) AU2015247859B2 (enExample)
CA (1) CA2945707A1 (enExample)
HK (1) HK1243643A1 (enExample)
SG (1) SG11201610349SA (enExample)
WO (1) WO2015160796A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6061677B2 (ja) * 2009-10-19 2017-01-18 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いてガングリオシドの活性を増大させて、アルツハイマー病を治療するための方法
US9994589B2 (en) 2015-12-14 2018-06-12 Amicus Therapeutics, Inc. Compounds and methods for the treatment of alzheimer's disease and/or cerebral amyloid angiopathy
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

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4407957A (en) 1981-03-13 1983-10-04 Damon Corporation Reversible microencapsulation of a core material
SG47470A1 (en) 1991-04-25 1998-04-17 Univ Brown Res Found Implantable biocompatible immunoisolatory vehicle for delivery of a selected therapeutic products
US5766846A (en) 1992-07-10 1998-06-16 Athena Neurosciences Methods of screening for compounds which inhibit soluble β-amyloid peptide production
US5383851A (en) 1992-07-24 1995-01-24 Bioject Inc. Needleless hypodermic injection device
US5569189A (en) 1992-09-28 1996-10-29 Equidyne Systems, Inc. hypodermic jet injector
ATE220327T1 (de) 1992-09-29 2002-07-15 Inhale Therapeutic Syst Pulmonale abgabe von aktiven fragmenten des parathormons
FR2718357B1 (fr) 1994-04-06 1997-10-03 Defarges Alain Moreau Perfectionnements apportés à un dispositif d'injection par jet sans aiguille.
US5599302A (en) 1995-01-09 1997-02-04 Medi-Ject Corporation Medical injection system and method, gas spring thereof and launching device using gas spring
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
US5654007A (en) 1995-06-07 1997-08-05 Inhale Therapeutic Systems Methods and system for processing dispersible fine powders
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US20050031651A1 (en) 2002-12-24 2005-02-10 Francine Gervais Therapeutic formulations for the treatment of beta-amyloid related diseases
WO2004103368A1 (en) 2003-05-22 2004-12-02 The Hospital For Sick Children Treatment of tay sachs or sandhoff diseases by enhancing hexosaminidase activity
MX2007015602A (es) 2005-06-08 2008-02-21 Amicus Therapeutics Inc Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas.
JP2009537149A (ja) * 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
ATE555788T1 (de) 2006-06-23 2012-05-15 Amicus Therapeutics Inc Verfahren zur behandlung neurologischer erkrankungen mittels verstärkung der beta - glucocerebrosidaseaktivität
WO2008134628A2 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2011011181A1 (en) 2009-07-01 2011-01-27 Amicus Therapeutics, Inc. Pharmacological chaperones for the treatment of alzheimer's disease
JP6061677B2 (ja) 2009-10-19 2017-01-18 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いてガングリオシドの活性を増大させて、アルツハイマー病を治療するための方法
US20140219986A1 (en) * 2011-03-11 2014-08-07 Amicus Therapeutics ,Inc. Dosing regimens for the treatment of fabry disease
ES2864774T3 (es) * 2011-12-22 2021-10-14 Centogene Ip Gmbh Combinación de un compuesto que tiene la capacidad de reorganizar una enzima lisosomal y Ambroxol y/o un derivado de Ambroxol
EP2844279B1 (en) * 2012-05-03 2020-12-09 Amicus Therapeutics, Inc. Dosing regimens for the treatment of pompe disease

Also Published As

Publication number Publication date
AU2015247859A1 (en) 2016-12-01
CN107148270A (zh) 2017-09-08
US9675627B2 (en) 2017-06-13
AU2015247859B2 (en) 2020-03-26
JP2017511372A (ja) 2017-04-20
KR20170003576A (ko) 2017-01-09
WO2015160796A1 (en) 2015-10-22
US20160008383A1 (en) 2016-01-14
CA2945707A1 (en) 2015-10-22
US20170273996A1 (en) 2017-09-28
HK1243643A1 (zh) 2018-07-20
EP3131547A1 (en) 2017-02-22
SG11201610349SA (en) 2017-01-27

Similar Documents

Publication Publication Date Title
JP6061922B2 (ja) プロテイノパチーの処置方法
JP5303458B2 (ja) β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法
EP2490712B1 (en) Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases
EP2142197A2 (en) Method for the treatment of fabry disease using pharmacological chaperones
JP2017511372A (ja) 脳アミロイドーシスを処置及び/又は予防する投与計画
Shi et al. PGRN as an emerging regulator of lipid metabolism in neurodegenerative diseases
US20190248790A1 (en) Deuterated compounds for treating fabry, gaucher, parkinson's and related diseases and conditions, and compositions and methods thereof
US20240189307A1 (en) Methods of stabilizing the neuronal proteome against collapse and protecting vascular cells
KR20240034203A (ko) 소아청소년 환자에서 파브리병을 치료하는 방법
TW201620517A (zh) 用於治療及/或預防腦澱粉樣變性(Cerebral Amyloidoses)之給藥方案
HK1175400B (en) Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases

Legal Events

Date Code Title Description
RD02 Notification of acceptance of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7422

Effective date: 20170601

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20170607

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20180412

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20181205

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20181218

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20190318

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20190401

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20190611

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190910

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20200121

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20200213

R150 Certificate of patent or registration of utility model

Ref document number: 6662789

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

LAPS Cancellation because of no payment of annual fees