CN114828960A - 治疗儿童个体的cln2病的方法 - Google Patents

治疗儿童个体的cln2病的方法 Download PDF

Info

Publication number
CN114828960A
CN114828960A CN202080068971.3A CN202080068971A CN114828960A CN 114828960 A CN114828960 A CN 114828960A CN 202080068971 A CN202080068971 A CN 202080068971A CN 114828960 A CN114828960 A CN 114828960A
Authority
CN
China
Prior art keywords
individual
cln2
patients
study
rhtpp1
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
CN202080068971.3A
Other languages
English (en)
Chinese (zh)
Inventor
D·雅各布
J·汉斯肖
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 CN114828960A publication Critical patent/CN114828960A/zh
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)
CN202080068971.3A 2019-08-29 2020-08-31 治疗儿童个体的cln2病的方法 Pending CN114828960A (zh)

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

Publications (1)

Publication Number Publication Date
CN114828960A true CN114828960A (zh) 2022-07-29

Family

ID=72470612

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080068971.3A Pending CN114828960A (zh) 2019-08-29 2020-08-31 治疗儿童个体的cln2病的方法

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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103096918A (zh) * 2010-06-25 2013-05-08 夏尔人类遗传性治疗公司 治疗试剂的cns 递送
CN107847615A (zh) * 2015-05-08 2018-03-27 生物马林药物股份有限公司 用于治疗cln2疾病的tpp1调配物及方法

Family Cites Families (6)

* 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
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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103096918A (zh) * 2010-06-25 2013-05-08 夏尔人类遗传性治疗公司 治疗试剂的cns 递送
CN107847615A (zh) * 2015-05-08 2018-03-27 生物马林药物股份有限公司 用于治疗cln2疾病的tpp1调配物及方法

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ALFRIED KOHLSCHÜTTER等: "Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses", 《CNS DRUGS》, vol. 33, no. 4, 1 April 2019 (2019-04-01), pages 315 - 325, XP055694540, DOI: 10.1007/s40263-019-00620-8 *
BIOMARIN PHARMACEUTICALS: "ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS", pages 3 - 4, Retrieved from the Internet <URL:https://www.ema.europa.eu/en/documents/product-information/brineura-epar-product-information_en> *
BIOMARIN PHARMACUETICALS: "BioMarin Announces Ongoing Study Demonstrates Durable Treatment Benefit from Brineura® (cerliponase alfa) for 3 Years Reduced Rate of Decline Maintained in Children with CLN2 Disease, a Form of Batten Disease", Retrieved from the Internet <URL:http://investors.biomarin.com/2019-02-07-BioMarin-Announces-Ongoing-Study-Demonstrates-Durable-Treatment-Benefit-from-Brineura-R-cerliponase-alfa-for-3-years> *

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
JP2022546042A (ja) 2022-11-02
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
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
Hughes et al. A randomised, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of three dosing schedules of agalsidase alfa enzyme replacement therapy for Fabry disease
Shavelle et al. Life expectancy
JP2025165414A (ja) 小児対象のｃｌｎ２疾患を治療するための方法
HK40077860A (en) Methods for treating cln2 disease in pediatric subjects
RU2848083C1 (ru) Способы лечения заболевания cln2 у детей
HK40117495A (en) Methods for treating cln2 disease in pediatric subjects
HK40077045A (en) Methods for treating cln2 disease in pediatric subjects
HK40077045B (en) Methods for treating cln2 disease in pediatric subjects
Palabiyik Biotinidase deficiency
Vrdoljak Neonatalne konvulzije
Barber CASE STUDY 19 The person with rheumatoid arthritis
Gunawan et al. Tuberous sclerosis complex in a child: diagnosis and management
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
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40077860

Country of ref document: HK