JP2025501646A - Ｇｍ２ガングリオシドーシスの処置 - Google Patents

Ｇｍ２ガングリオシドーシスの処置 Download PDF

Info

Publication number
JP2025501646A
JP2025501646A JP2024539849A JP2024539849A JP2025501646A JP 2025501646 A JP2025501646 A JP 2025501646A JP 2024539849 A JP2024539849 A JP 2024539849A JP 2024539849 A JP2024539849 A JP 2024539849A JP 2025501646 A JP2025501646 A JP 2025501646A
Authority
JP
Japan
Prior art keywords
compound
subject
formula
disease
use according
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.)
Pending
Application number
JP2024539849A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025501646A5 (https=
Inventor
ジェニファー ロビン，
フリッツ ブラッター，
ロバート ヘット，
カイル ランズクローナー，
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.)
Azafaros BV
Original Assignee
Azafaros BV
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
Priority claimed from EP22150783.3A external-priority patent/EP4209215A1/en
Application filed by Azafaros BV filed Critical Azafaros BV
Publication of JP2025501646A publication Critical patent/JP2025501646A/ja
Publication of JP2025501646A5 publication Critical patent/JP2025501646A5/ja
Pending legal-status Critical Current

Links

Images

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
    • 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
    • A61P25/28—Drugs 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)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pyridine Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024539849A 2022-01-10 2023-01-10 Ｇｍ２ガングリオシドーシスの処置 Pending JP2025501646A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP22150783.3 2022-01-10
EP22150783.3A EP4209215A1 (en) 2022-01-10 2022-01-10 Treatment of gm2 gangliosidosis
EP22193136 2022-08-31
EP22193136.3 2022-08-31
PCT/EP2023/050459 WO2023131720A1 (en) 2022-01-10 2023-01-10 Treatment of gm2 gangliosidosis

Publications (2)

Publication Number Publication Date
JP2025501646A true JP2025501646A (ja) 2025-01-22
JP2025501646A5 JP2025501646A5 (https=) 2026-01-20

Family

ID=84982296

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024539849A Pending JP2025501646A (ja) 2022-01-10 2023-01-10 Ｇｍ２ガングリオシドーシスの処置

Country Status (9)

Country Link
US (1) US20250120958A1 (https=)
EP (2) EP4714497A2 (https=)
JP (1) JP2025501646A (https=)
AU (1) AU2023205441A1 (https=)
CA (1) CA3242701A1 (https=)
IL (1) IL314212A (https=)
MX (1) MX2024008583A (https=)
TW (1) TW202337456A (https=)
WO (1) WO2023131720A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202539666A (zh) * 2024-02-01 2025-10-16 荷蘭商雅捷法羅斯公司 Gm1神經節苷脂儲積症之治療
EP4595964A1 (en) * 2024-02-01 2025-08-06 Azafaros B.V. Treatment of gm1 gangliosidosis
WO2026013261A1 (en) 2024-07-11 2026-01-15 Azafaros B.V. Nizubaglustat for use in the treatment of lysosomal storage disorders
EP4678174A1 (en) 2024-07-11 2026-01-14 Azafaros B.V. Nizubaglustat for use in the treatment of lysosomal storage disorders

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3461812B1 (en) 2014-03-27 2021-10-13 Academisch Medisch Centrum N-(5-(biphen-4-ylmethyloxy)pentyl)-substituted iminosugars as inhibitors of glucosylceramide synthase
EP3978474A1 (en) * 2020-10-02 2022-04-06 Azafaros B.V. Crystalline forms of a pharmaceutical compound

Also Published As

Publication number Publication date
WO2023131720A1 (en) 2023-07-13
TW202337456A (zh) 2023-10-01
EP4714497A2 (en) 2026-03-25
US20250120958A1 (en) 2025-04-17
EP4463157A1 (en) 2024-11-20
IL314212A (en) 2024-09-01
CA3242701A1 (en) 2023-07-13
MX2024008583A (es) 2024-07-23
EP4463157C0 (en) 2026-02-25
EP4463157B1 (en) 2026-02-25
AU2023205441A1 (en) 2024-07-25

Similar Documents

Publication Publication Date Title
JP2025501646A (ja) Ｇｍ２ガングリオシドーシスの処置
JP7809655B2 (ja) 医薬化合物の結晶形態
SG178000A1 (en) Methods and combination therapies for treating alzheimer's disease
KR20180051561A (ko) 특정 환자 집단에서 신경퇴행성 장애를 치료하는 방법
TW201632511A (zh) 治療精神分裂症之組成物及方法
JP2024542523A (ja) 神経変性障害の治療選択及び治療
EP4209215A1 (en) Treatment of gm2 gangliosidosis
CN118632693A (zh) Gm2神经节苷脂贮积症的治疗
EP4101448B1 (en) Composition comprising rilmenidine compound as active ingredient for treatment of fragile x syndrome or related developmental disability
EP4088721B1 (en) Composition for treating fragile x syndrome or related developmental disorders, comprising lisuride compound as active ingredient
CA2550610A1 (en) Use of gaboxadol for treating insomnia
TW202539666A (zh) Gm1神經節苷脂儲積症之治療
CN108601772A (zh) 用于治疗tdp-43蛋白质病的他克莫司
EP4595964A1 (en) Treatment of gm1 gangliosidosis
KR102374917B1 (ko) 피렌페론 화합물을 유효성분으로 포함하는 취약 x 증후군 및 관련 발달 장애치료용 조성물
TW200800198A (en) Pharmaceutical dosage forms and compositions
KR102089376B1 (ko) 디아민 유도체를 포함하는 뇌졸중, 신경 세포 손상, 파킨슨병 또는 루게릭병의 예방 또는 치료용 조성물
WO2026013261A1 (en) Nizubaglustat for use in the treatment of lysosomal storage disorders
TW202606667A (zh) 使用葡萄糖神經醯胺合成酶抑制劑治療患有肝損傷之受試者的方法
JP2006290772A (ja) 多発性硬化症治療剤
KR20140130695A (ko) 섬유근육통 및 만성 피로 증후군을 치료하기 위한 (1r,4r)-6''-플루오로-(N-메틸- 또는 N,N-디메틸-)-4-페닐-4'',9''-디하이드로-3''H-스피로-[사이클로헥산-1,1''-피라노[3,4,b]인돌]-4-아민
EA045552B1 (ru) Кристаллические формы фармацевтического соединения
KR20200046525A (ko) 피렌페론 화합물을 유효성분으로 포함하는 취약 x 증후군 및 관련 발달 장애치료용 조성물

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260106

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20260106