HRP20191923T1 - Postupci i sastavi za isporuku arilsulfataze a u cns - Google Patents

Postupci i sastavi za isporuku arilsulfataze a u cns Download PDF

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Publication number
HRP20191923T1
HRP20191923T1 HRP20191923TT HRP20191923T HRP20191923T1 HR P20191923 T1 HRP20191923 T1 HR P20191923T1 HR P20191923T T HRP20191923T T HR P20191923TT HR P20191923 T HRP20191923 T HR P20191923T HR P20191923 T1 HRP20191923 T1 HR P20191923T1
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HR
Croatia
Prior art keywords
stable aqueous
optionally
concentration
polysorbate
formula
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HRP20191923TT
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English (en)
Inventor
Nazila Salamat-Miller
Katherine Taylor
Paul Campolieto
Zahra Shahrokh
Jing Pan
Lawrence Charnas
Teresa Leah Wright
Pericles Calias
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Shire Human Genetic Therapies, Inc.
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Application filed by Shire Human Genetic Therapies, Inc. filed Critical Shire Human Genetic Therapies, Inc.
Publication of HRP20191923T1 publication Critical patent/HRP20191923T1/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
    • A61K9/0012Galenical 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
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • 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)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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)
    • 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)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K38/54Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
    • 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
    • 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
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate 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
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/06Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013Iduronate-2-sulfatase (3.1.6.13)
    • 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
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Inorganic Chemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Psychiatry (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hospice & Palliative Care (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Claims (16)

1. Stabilna vodena formula za intratekalnu primjenu koja sadrži protein arilsulfatazu A (ASA) u koncentraciji većoj od 10 mg/ml, sol, polisorbatni surfaktant i puferski agens, pri čemu je sol NaCl.
2. Stabilna vodena formula prema patentnom zahtjevu 1, gdje je ASA protein prisutan u koncentraciji u rasponu od 15-100 mg/ml, opcionalno gdje je ASA protein prisutan u koncentraciji izabranoj između 30 mg/ml, 50 mg/ml ili 100 mg/ml.
3. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, gdje ASA protein sadrži aminokiselinsku sekvencu SEQ ID NO: 1.
4. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, pri čemu: (i) ASA protein se proizvodi iz ljudske stanične linije, ili (ii) ASA protein se proizvodi iz CHO stanica.
5. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, gde je NaCl prisutan u koncentraciji koja je u rasponu od 30-300 mM, opcionalno u koncentraciji u rasponu od 137-154 mM, opcionalno u koncentraciji od približno 154 mM.
6. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, gde je polisorbatni surfaktant izabran iz skupine koja se sastoji od polisorbata 20, polisorbata 40, polisorbata 60, polisorbata 80 i njihove kombinacije, opcionalno gdje polisorbatni surfaktant je polisorbat 20, opcionalno gdje je polisorbatni surfaktant prisutan u koncentraciji u rasponu od 0,001 do 0,5% i opcionalno gdje je polisorbat 20 prisutan u koncentraciji od približno 0,005%.
7. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, gdje je puferski agens izabran iz skupine koja se sastoji od fosfata, acetata, histidina, sukcinata, citrata, Trisa i njihovih kombinacija, a opcionalno gdje je puferski agens fosfat i prisutan je u koncentraciji ne većoj od 50 mM i opcionalno u koncentraciji ne većoj od 20 mM.
8. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, pri čemu formula ima pH od približno 3-8,0, približno 6,0-6,5 ili približno 6,0.
9. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, pri čemu je formula rekonstituirana iz liofilizirane formule s razblaživačem.
10. Stabilna vodena formula prema bilo kojem od prethodnih patentnih zahtjeva, pri čemu formula dalјe sadrži stabilizator.
11. Stabilna vodena formula prema patentnom zahtjevu 10, gdje je stabilizator izabran iz skupine koja se sastoji od saharoze, glukoze, manitola, sorbitola, PEG 4000, histidina, arginina, lizina, fosfolipida i njihove kombinacije.
12. Posuda za skladištenje koja sadrži jedinični dozni oblik stabilne vodene formule prema bilo kojem od prethodnih patentnih zahtjeva.
13. Posuda za skladištenje prema patentnom zahtjevu 12, pri čemu je posuda za skladištenje izabrana između ampule, bočice, patrone, rezervoara, lyo-injektora ili prethodno napunjene šprice, opcionalno gdje je posuda za skladištenje prethodno napunjena šprica i opcionalno je izabrana između borosilikatnih staklenih šprica s premazom od pečenog silikona, borosilikatnih staklenih šprica s raspršenim silikonom ili plastičnih šprica od smola bez silikona.
14. Posuda za skladištenje prema patentnom zahtjevu 12, pri čemu je stabilna vodena formula prisutna u obujmu manjem od oko 50,0 mL, opcionalno gdje je stabilna vodena formula prisutna u obujmu manjem od oko 5,0 mL.
15. Stabilna vodena formula prema bilo kojem od patentnih zahtjeva 1-11, za upotrebu u postupku liječenja bolesti metakromatske leukodistrofije (MLD) koji obuhvaća korak intratekalne primjene formule subjektu.
16. Stabilna vodena formula za upotrebu prema patentnom zahtjevu 15, pri čemu: (i) intratekalna primjena se upotrebljava zajedno sa intravenskom primjenom, opcionalno gdje intravenska primjena nije češća od jednom mjesečno ili nije češća od jednom u svaka dva mjeseca, ili (ii) intratekalna primjena se upotrebljava u odsutstvu intravenske primjene, i/ili (iii) intratekalna primjena se vrši jednom u svaka dva tjedna, jednom mjesečno ili jednom u svaka dva mjeseca, i/ili (iv) intratekalna primjena se upotrebljava u odsutstvu istovremene imunosupresivne terapije.
HRP20191923TT 2010-06-25 2019-10-22 Postupci i sastavi za isporuku arilsulfataze a u cns HRP20191923T1 (hr)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US35885710P 2010-06-25 2010-06-25
US36078610P 2010-07-01 2010-07-01
US38786210P 2010-09-29 2010-09-29
US201161435710P 2011-01-24 2011-01-24
US201161442115P 2011-02-11 2011-02-11
US201161476210P 2011-04-15 2011-04-15
US201161495268P 2011-06-09 2011-06-09
PCT/US2011/041926 WO2011163650A2 (en) 2010-06-25 2011-06-25 Methods and compositions for cns delivery of arylsulfatase a
EP11799037.4A EP2585104B1 (en) 2010-06-25 2011-06-25 Methods and compositions for cns delivery of arylsulfatase a

Publications (1)

Publication Number Publication Date
HRP20191923T1 true HRP20191923T1 (hr) 2020-01-10

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US (1) US9770410B2 (hr)
EP (3) EP3626257B1 (hr)
JP (1) JP6045493B2 (hr)
KR (4) KR102094094B1 (hr)
CN (1) CN103282046B (hr)
AU (4) AU2011270670B2 (hr)
BR (1) BR112012033216B1 (hr)
CA (1) CA2803003C (hr)
CY (2) CY1122282T1 (hr)
DK (1) DK2585104T3 (hr)
ES (1) ES2754776T3 (hr)
HR (1) HRP20191923T1 (hr)
IL (3) IL291556B2 (hr)
LT (1) LT2585104T (hr)
ME (1) ME03647B (hr)
MX (1) MX344795B (hr)
NZ (2) NZ605873A (hr)
PL (2) PL3626257T3 (hr)
RS (1) RS59468B1 (hr)
RU (1) RU2671503C2 (hr)
SI (1) SI2585104T1 (hr)
TW (1) TWI667037B (hr)
UA (1) UA115650C2 (hr)
WO (1) WO2011163650A2 (hr)
ZA (1) ZA201300675B (hr)

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