JP2022546042A - 小児対象のｃｌｎ２疾患を治療するための方法 - Google Patents

小児対象のｃｌｎ２疾患を治療するための方法 Download PDF

Info

Publication number
JP2022546042A
JP2022546042A JP2022513165A JP2022513165A JP2022546042A JP 2022546042 A JP2022546042 A JP 2022546042A JP 2022513165 A JP2022513165 A JP 2022513165A JP 2022513165 A JP2022513165 A JP 2022513165A JP 2022546042 A JP2022546042 A JP 2022546042A
Authority
JP
Japan
Prior art keywords
subject
rhtpp1
cln2
patients
disease
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
JP2022513165A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022546042A5 (https=
Inventor
デイビッド ジャコビー，
ジョシュア ヘンショウ，
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.)
Biomarin Pharmaceutical Inc
Original Assignee
Biomarin Pharmaceutical Inc
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 Biomarin Pharmaceutical Inc filed Critical Biomarin Pharmaceutical Inc
Publication of JP2022546042A publication Critical patent/JP2022546042A/ja
Publication of JP2022546042A5 publication Critical patent/JP2022546042A5/ja
Priority to JP2025134120A priority Critical patent/JP2025165414A/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
    • 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)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • 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
    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • 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
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • A61K9/0051—Ocular inserts or implants
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61K9/06—Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • 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
    • A61K9/08—Solutions
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Organic Chemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Inorganic Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2022513165A 2019-08-29 2020-08-31 小児対象のｃｌｎ２疾患を治療するための方法 Pending JP2022546042A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025134120A JP2025165414A (ja) 2019-08-29 2025-08-12 小児対象のｃｌｎ２疾患を治療するための方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962893535P 2019-08-29 2019-08-29
US62/893,535 2019-08-29
PCT/US2020/048704 WO2021042020A1 (en) 2019-08-29 2020-08-31 Methods for treating cln2 disease in pediatric subjects

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025134120A Division JP2025165414A (ja) 2019-08-29 2025-08-12 小児対象のｃｌｎ２疾患を治療するための方法

Publications (2)

Publication Number Publication Date
JP2022546042A true JP2022546042A (ja) 2022-11-02
JP2022546042A5 JP2022546042A5 (https=) 2023-09-08

Family

ID=72470612

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022513165A Pending JP2022546042A (ja) 2019-08-29 2020-08-31 小児対象のｃｌｎ２疾患を治療するための方法
JP2025134120A Pending JP2025165414A (ja) 2019-08-29 2025-08-12 小児対象のｃｌｎ２疾患を治療するための方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025134120A Pending JP2025165414A (ja) 2019-08-29 2025-08-12 小児対象のｃｌｎ２疾患を治療するための方法

Country Status (25)

Country Link
US (1) US20220273775A1 (https=)
EP (2) EP4021575B1 (https=)
JP (2) JP2022546042A (https=)
KR (1) KR20220054375A (https=)
CN (1) CN114828960A (https=)
AR (1) AR119871A1 (https=)
AU (1) AU2020336984B2 (https=)
BR (1) BR112022003598A2 (https=)
CA (1) CA3152521A1 (https=)
CL (1) CL2022000496A1 (https=)
DK (1) DK4021575T3 (https=)
ES (1) ES2987156T3 (https=)
FI (1) FI4021575T3 (https=)
HR (2) HRP20240871T1 (https=)
HU (1) HUE067135T2 (https=)
IL (1) IL290763A (https=)
LT (1) LT4021575T (https=)
MX (1) MX2022002451A (https=)
PL (1) PL4021575T3 (https=)
PT (1) PT4021575T (https=)
RS (1) RS65763B1 (https=)
SI (1) SI4021575T1 (https=)
SM (1) SMT202400315T1 (https=)
TW (1) TW202122106A (https=)
WO (1) WO2021042020A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220323019A1 (en) * 2021-04-02 2022-10-13 Neuropace, Inc. Systems and methods for obtaining a clinical response estimate biomarker using machine-learned models trained on implanted neurostimulator data

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018521964A (ja) * 2015-05-08 2018-08-09 バイオマリン ファーマシューティカル インコーポレイテッド Ｔｐｐ１配合物及びｃｌｎ２疾患を治療するための方法

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6302685B1 (en) 1997-09-16 2001-10-16 University Of Medicine And Dentistry Of New Jersey Human lysosomal protein and methods of its use
US20020006400A1 (en) * 2000-05-11 2002-01-17 Peter Lobel Recombinant human CLN2 protein and methods of its production and use
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
LT2588130T (lt) * 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR102738860B1 (ko) * 2015-10-23 2024-12-04 유니버시티 오브 아이오와 리써치 파운데이션 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물
BR112021009370A2 (pt) * 2018-11-14 2021-08-17 Regenxbio Inc. método de tratamento da doença de batten cln2, composição farmacêutica e kit

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018521964A (ja) * 2015-05-08 2018-08-09 バイオマリン ファーマシューティカル インコーポレイテッド Ｔｐｐ１配合物及びｃｌｎ２疾患を治療するための方法

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BRINEURA PRODUCT INFORATION［オンライン］, JPN6024028600, 20 March 2019 (2019-03-20), pages 2024 - 07, ISSN: 0005374311 *
JOURNAL OF NEUROSCIENCE RESEARCH, vol. 92, JPN6024028601, 2014, pages 1591 - 1598, ISSN: 0005374312 *
N ENGL J MED, vol. 378, JPN6024028602, 2018, pages 1898 - 1907, ISSN: 0005374313 *

Also Published As

Publication number Publication date
WO2021042020A1 (en) 2021-03-04
RS65763B1 (sr) 2024-08-30
SI4021575T1 (sl) 2024-10-30
PT4021575T (pt) 2024-07-30
PL4021575T3 (pl) 2024-09-23
EP4438120A3 (en) 2024-12-11
BR112022003598A2 (pt) 2022-05-24
EP4021575B1 (en) 2024-05-22
HRP20220370A1 (hr) 2022-09-16
HRP20240871T1 (hr) 2024-10-11
TW202122106A (zh) 2021-06-16
ES2987156T3 (es) 2024-11-14
EP4438120A2 (en) 2024-10-02
LT4021575T (lt) 2024-08-12
IL290763A (en) 2022-04-01
AU2020336984A1 (en) 2022-03-10
AR119871A1 (es) 2022-01-19
CL2022000496A1 (es) 2022-10-21
MX2022002451A (es) 2022-06-02
CA3152521A1 (en) 2021-03-04
FI4021575T3 (fi) 2024-07-24
HUE067135T2 (hu) 2024-10-28
SMT202400315T1 (it) 2024-09-16
DK4021575T3 (da) 2024-08-05
CN114828960A (zh) 2022-07-29
EP4021575A1 (en) 2022-07-06
KR20220054375A (ko) 2022-05-02
JP2025165414A (ja) 2025-11-04
AU2020336984B2 (en) 2026-04-23
US20220273775A1 (en) 2022-09-01

Similar Documents

Publication Publication Date Title
JP7818561B2 (ja) デクスメデトミジンを用いて小児患者を治療する方法
US11229687B2 (en) TPP-1 formulations and methods for treating CLN2 disease
JP2025165414A (ja) 小児対象のｃｌｎ２疾患を治療するための方法
RU2848083C1 (ru) Способы лечения заболевания cln2 у детей
HK40117495A (en) Methods for treating cln2 disease in pediatric subjects
HK40077045B (en) Methods for treating cln2 disease in pediatric subjects
HK40077045A (en) Methods for treating cln2 disease in pediatric subjects
HK40077860A (en) Methods for treating cln2 disease in pediatric subjects
HK40107015A (en) Tpp1 formulations and methods for treating cln2 disease
Goh et al. Iatrogenic Hypoglycaemia in the Elderly: An Analysis of Diabetic Medication Usage Patterns According to Age
HK1250479B (en) Tpp1 formulations and methods for treating cln2 disease
BR112017023805B1 (pt) Formulações e composições de tpp1, bem como uso no tratamento de doença lcn2

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230831

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230831

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240723

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20240731

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20241018

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20241220

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250122

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20250410

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250812