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HRP20240050T1 - Formulacije tpp1 i postupci liječenja bolesti cln2 - Google Patents

Formulacije tpp1 i postupci liječenja bolesti cln2 Download PDF

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Publication number
HRP20240050T1
HRP20240050T1 HRP20240050TT HRP20240050T HRP20240050T1 HR P20240050 T1 HRP20240050 T1 HR P20240050T1 HR P20240050T T HRP20240050T T HR P20240050TT HR P20240050 T HRP20240050 T HR P20240050T HR P20240050 T1 HRP20240050 T1 HR P20240050T1
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HR
Croatia
Prior art keywords
concentration
rhtpp1
preparation
formulation
sodium phosphate
Prior art date
Application number
HRP20240050TT
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English (en)
Inventor
Thomas W. Lester
Saeed Moshashaee
Augustus O. Okhamafe
Charles A. O'neill
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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.)
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Publication date
Application filed by Biomarin Pharmaceutical Inc. filed Critical Biomarin Pharmaceutical Inc.
Publication of HRP20240050T1 publication Critical patent/HRP20240050T1/hr

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • 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
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813Exopeptidases (3.4.11. to 3.4.19)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02Inorganic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/06Nuclear magnetic resonance [NMR] contrast preparations; Magnetic resonance imaging [MRI] contrast preparations
    • A61K49/08Nuclear magnetic resonance [NMR] contrast preparations; Magnetic resonance imaging [MRI] contrast preparations characterised by the carrier
    • A61K49/10Organic compounds
    • A61K49/14Peptides, e.g. proteins
    • 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
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61MDEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/14Infusion devices, e.g. infusing by gravity; Blood infusion; Accessories therefor
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485Exopeptidases (3.4.11-3.4.19)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009Tripeptidyl-peptidase I (3.4.14.9)
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Molecular Biology (AREA)
  • Inorganic Chemistry (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Anesthesiology (AREA)
  • Vascular Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)

Claims (11)

1. Formulacija koja sadržava rekombinantnu humanu tripeptidil peptidazu-1 (rhTPP1) za intracerebroventrikularnu, intratekalnu ili intraokularnu primjenu, naznačena time što rhTPP1 sadrži aminokiselinsku sekvencu SEQ ID NO: 1 ili njezin fragment, pri čemu formulacija sadrži rhTPP1 u koncentraciji od 25 mg/mL do 35 mg/mL, te nadalje sadrži kalijev klorid u koncentraciji od 0,01 mg/mL do 1 mg/mL, magnezijev klorid heksahidrat u koncentraciji od 0,01 mg/mL do 1 mg/mL, i kalcijev klorid dihidrat u koncentraciji od 0,01 mg/mL do 1 mg/mL, i pri čemu formulacija ima pH vrijednost od 6,5.
2. Formulacija prema zahtjevu 1, naznačena time što nadalje sadrži natrijev fosfat dvobazični heptahidrat u koncentraciji od 0,01 mg/mL do 1 mg/mL, natrijev fosfat monobazični monohidrat u koncentraciji od 0,01 mg/mL do 1 mg/mL, i natrijev klorid u koncentraciji od 1 mg/mL do 20 mg/mL.
3. Formulacija prema zahtjevu 1, naznačena time što sadrži natrijev fosfat dvobazični heptahidrat u koncentraciji od 0,11 mg/mL, natrijev fosfat monobazični monohidrat u koncentraciji od 0,08 mg/mL, natrijev klorid u koncentraciji od 8,77 mg/mL, kalijev klorid u koncentraciji od 0,22 mg/mL, magnezijev klorid heksahidrat u koncentraciji od 0,16 mg/mL, i kalcijev klorid dihidrat u koncentraciji od 0,21 mg/mL.
4. Formulacija prema bilo kojem od zahtjeva 1-3 naznačena time što je formulacija bez konzervansa.
5. Pripravak koji sadrži formulaciju prema bilo kojem od zahtjeva 1-4 naznačen time što što je za upotrebu za liječenje neuronalne ceroidne lipofuscinoze (CLN2).
6. Pripravak za upotrebu prema zahtjevu 5, naznačen time što pripravak sadrži dozu od 300 mg rekombinantne humane tripeptidil peptidaze-1 (rhTPP1).
7. Pripravak za upotrebu prema zahtjevu 5, naznačen time što se doza rekombinantne humane tripeptidil peptidaze-1 (rhTPP1) daje subjektu tijekom razdoblja od 4 sata.
8. Pripravak za upotrebu prema zahtjevu 5, naznačen time što se doza rekombinantne humane tripeptidil peptidaze-1 (rhTPP1) daje subjektu brzinom manjom ili jednakom 75 mg/sat.
9. Pripravak koji sadrži rekombinantnu humanu tripeptidil peptidazu-1 (rhTPP1) za primjenu u liječenju neuronalne ceroidne lipofuscinoze (CLN2) kod subjekta koji sadrži dozu rekombinantne humane tripeptidil peptidaze-1 (rhTPP1) djelotvornu da održi fiziološku funkciju ili uspori ili smanji pogoršanje fiziološke funkcije kod subjekta, pri čemu je fiziološka funkcija jezična funkcija, motorička funkcija, vid ili funkcija hranjenja, pri čemu rhTPP1 sadrži aminokiselinsku sekvencu SEQ ID NO: 1 ili njezin fragment, te pri čemu pripravak sadrži rhTPP1 u koncentraciji od 25 mg/mL do 35 mg/mL, te nadalje sadrži kalijev klorid u koncentraciji od 0,01 mg/mL do 1 mg/mL, magnezijev klorid heksahidrat u koncentraciji od 0,01 mg/mL do 1 mg/mL, i kalcijev klorid dihidrat u koncentraciji od 0,01 mg/mL do 1 mg/mL, te pri čemu formulacija ima pH vrijednost od 6,5.
10. Pripravak za upotrebu prema zahtjevu 9, naznačen time što pripravak nadalje sadrži natrijev fosfat dvobazični heptahidrat u koncentraciji od 0,01 mg/mL do 1 mg/mL, natrijev fosfat monobazični monohidrat u koncentraciji od 0,01 mg/mL do 1 mg/mL, i natrijev klorid u koncentraciji od 1 mg/mL do 20 mg/mL.
11. Pripravak za upotrebu prema zahtjevu 9, naznačen time što pripravak sadrži natrijev fosfat dvobazični heptahidrat u koncentraciji od 0,11 mg/mL, natrijev fosfat monobazični monohidrat u koncentraciji od 0,08 mg/mL, natrijev klorid u koncentraciji od 8,77 mg/mL, kalijev klorid u koncentraciji od 0,22 mg/mL, magnezijev klorid heksahidrat u koncentraciji od 0,16 mg/mL, i kalcijev klorid dihidrat u koncentraciji od 0,21 mg/mL.
HRP20240050TT 2015-05-08 2016-05-05 Formulacije tpp1 i postupci liječenja bolesti cln2 HRP20240050T1 (hr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562158789P 2015-05-08 2015-05-08
US201662300171P 2016-02-26 2016-02-26
PCT/US2016/031074 WO2016182862A1 (en) 2015-05-08 2016-05-05 Tpp1 formulations and methods for treating cln2 disease
EP16793229.2A EP3294345B1 (en) 2015-05-08 2016-05-05 Tpp1 formulations and methods for treating cln2 disease

Publications (1)

Publication Number Publication Date
HRP20240050T1 true HRP20240050T1 (hr) 2024-03-29

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HRP20171905AA HRP20171905B1 (hr) 2015-05-08 2016-05-05 Formulacije tpp1 i postupci liječenja bolesti cln2
HRP20240050TT HRP20240050T1 (hr) 2015-05-08 2016-05-05 Formulacije tpp1 i postupci liječenja bolesti cln2

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EP (2) EP3294345B1 (hr)
JP (3) JP6931330B2 (hr)
KR (1) KR102375051B1 (hr)
CN (2) CN107847615B (hr)
AU (1) AU2016262434B2 (hr)
BR (1) BR112017023805A2 (hr)
CA (1) CA2984448A1 (hr)
CL (1) CL2017002825A1 (hr)
DK (1) DK3294345T3 (hr)
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FI (1) FI3294345T3 (hr)
HR (2) HRP20171905B1 (hr)
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IL (3) IL299024A (hr)
LT (1) LT3294345T (hr)
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TW (1) TWI752907B (hr)
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EP4327873A3 (en) 2024-05-01
RU2733464C2 (ru) 2020-10-01
AU2016262434B2 (en) 2022-06-02
CN107847615B (zh) 2022-01-25
IL273913B2 (en) 2023-06-01
AU2016262434A1 (en) 2017-11-16
IL255307B (en) 2021-03-25
JP2024026739A (ja) 2024-02-28
US20200353059A1 (en) 2020-11-12
JP2018521964A (ja) 2018-08-09
MX2017014204A (es) 2018-07-06
US20190216905A1 (en) 2019-07-18
EP3294345A4 (en) 2019-01-16
IL255307A0 (en) 2017-12-31
US20160324942A1 (en) 2016-11-10
ES2968375T3 (es) 2024-05-09
KR20180004158A (ko) 2018-01-10
HRP20171905A2 (hr) 2018-05-18
DK3294345T3 (da) 2024-01-22
US10758598B2 (en) 2020-09-01
TWI752907B (zh) 2022-01-21
WO2016182862A1 (en) 2016-11-17
RU2017142727A (ru) 2019-06-10
HK1250479A1 (en) 2018-12-21
KR102375051B1 (ko) 2022-03-15
RS65101B1 (sr) 2024-02-29
RU2017142727A3 (hr) 2019-12-11
SI3294345T1 (sl) 2024-03-29
US10279015B2 (en) 2019-05-07
JP6931330B2 (ja) 2021-09-01
PT3294345T (pt) 2024-01-18
PL3294345T3 (pl) 2024-07-01
TW201704469A (zh) 2017-02-01
CN107847615A (zh) 2018-03-27
EP3294345A1 (en) 2018-03-21
CN114225018A (zh) 2022-03-25
LT3294345T (lt) 2024-02-12
US11229687B2 (en) 2022-01-25
IL299024A (en) 2023-02-01
HRP20171905B1 (hr) 2023-03-17
JP2021175760A (ja) 2021-11-04
JP7421520B2 (ja) 2024-01-24
HUE065649T2 (hu) 2024-06-28
CA2984448A1 (en) 2016-11-17
EP3294345B1 (en) 2023-10-18
CL2017002825A1 (es) 2018-06-22
FI3294345T3 (fi) 2024-01-15
SMT202400026T1 (it) 2024-03-13
EP4327873A2 (en) 2024-02-28
ZA201707373B (en) 2019-02-27
IL273913A (en) 2020-05-31
BR112017023805A2 (pt) 2018-07-31

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