HRP20220639T1 - Kimerni i hibridni polipeptidi faktora viii i postupci njihove upotrebe - Google Patents

Kimerni i hibridni polipeptidi faktora viii i postupci njihove upotrebe Download PDF

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HRP20220639T1
HRP20220639T1 HRP20220639TT HRP20220639T HRP20220639T1 HR P20220639 T1 HRP20220639 T1 HR P20220639T1 HR P20220639T T HRP20220639T T HR P20220639TT HR P20220639 T HRP20220639 T HR P20220639T HR P20220639 T1 HRP20220639 T1 HR P20220639T1
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fviii
pharmaceutical preparation
bleeding
chimeric polypeptide
preparation according
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HRP20220639TT
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Jennifer A. Dumont
Susan Low
Alan Bitonti
Glenn Pierce
Alvin Luk
Haiyan Jiang
Byron Mckinney
Matt Ottmer
Jurg Sommer
Karen Nugent
Lian Li
Robert Peters
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Bioverativ Therapeutics Inc.
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Publication of HRP20220639T1 publication Critical patent/HRP20220639T1/hr

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    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • 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
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • 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
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/86Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving blood coagulating time or factors, or their receptors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/31Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hematology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Pathology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Food Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • Peptides Or Proteins (AREA)

Claims (19)

1. Farmaceutski pripravak, koji sadrži (i) kimerni polipeptid, koji sadrži dio Faktora VIII (FVIII) i drugi dio, te (ii) najmanje jednu farmaceutski prihvatljivu pomoćnu tvar, naznačen time što je (1) 25% do 40% FVIII dijela kimernog polipeptida jednolančani FVIII i (2) 60% do 75% FVIII dijela kimernog polipeptida je obrađeni FVIII, pri čemu jednolančani FVIII sadrži FVIII teškog lanca i FVIII lakog lanca na jednom polipeptidnom lancu i obrađeni FVIII sadrži FVIII teškog lanca i FVIII lakog lanca na dva polipeptidna lanca.
2. Farmaceutski pripravak prema zahtjevu 1, naznačen time što (a) 25% do 35% FVIII dijela kimernog polipeptida je jednolančani FVIII, ili (b) 30% do 40% FVIII dijela kimernog polipeptida je jednolančani FVIII.
3. Farmaceutski pripravak prema zahtjevu 1, naznačen time što (a) oko 25% FVIII dijela kimernog polipeptida je jednolančani FVIII i oko 75% FVIII dijela kimernog polipeptida je obrađeni FVIII, (b) oko 30% FVIII dijela kimernog polipeptida je jednolančani FVIII i oko 70% FVIII dijela kimernog polipeptida je obrađeni FVIII, ili (c) oko 35% FVIII dijela kimernog polipeptida je jednolančani FVIII i oko 65% FVIII dijela kimernog polipeptida je obrađeni FVIII, ili (d) 40% FVIII dijela kimernog polipeptida je jednolančani FVIII i 60% FVIII dijela kimernog polipeptida je obrađeni FVIII.
4. Farmaceutski pripravak prema bilo kojem od zahtjeva 1-3, naznačen time što kimerni polipeptid koji sadrži jednolančani FVIII ima aktivnost FVIII usporedivu s kimernim polipeptidom koji sadrži obrađeni FVIII.
5. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 4, naznačen time što kimerni polipeptid je dugo djelujući FVIII polipeptid.
6. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 5, naznačen time što drugi dio obuhvaća Fc regiju.
7. Farmaceutski pripravak prema zahtjevu 6, naznačen time što kimerni polipeptid je FVIIIFc monomerni dimer hibrid.
8. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 7, naznačen time što FVIII dio sadrži FVIII izbrisan iz B-domene ili pune duljine, zreli FVIII, ili pri čemu ostatak 1645 odgovara punoj dužini, zreli FVIII, ostatak 1648 koji odgovara punoj dužini, mature FVIII, ili oba ostatka u jednom lancu FVIII je supstituiran ili mutiran u usporedbi s divljim tipom FVIII.
9. Farmaceutski pripravak prema zahtjevu 8, naznačen time što FVIII dio sadrži FVIII koji je izbrisan iz B-domene.
10. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 9, naznačen time što dio FVIII sadrži aminokiselinsku sekvencu koja je najmanje 95% identična aminokiselinama 20 do 1457 iz SEQ ID NO: 2.
11. Farmaceutski pripravak prema zahtjevu 10, naznačen time što dio FVIII sadrži aminokiselinsku sekvencu identičnu aminokiselinama 20 do 1457 iz SEQ ID NO: 2.
12. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 11, naznačen time što drugi dio sadrži aminokiselinsku sekvencu koja je najmanje 95% identična aminokiselinama 21 do 247 iz SEQ ID NO: 4.
13. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 12, naznačen time što drugi dio sadrži aminokiselinsku sekvencu identičnu aminokiselinama 21 do 247 iz SEQ ID NO: 4.
14. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 13 naznačen time što je prikladan za potkožno, intradermalno, intravaskularno, intravenozno, intramuskularno, spinalno, intrakranijalno, intratekalno, intraokularno, periokularno, intraorbitalno, intrasinovijalno ili intraperitonealno davanje putem injekcije ljudima.
15. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 14, naznačen time što je za uporabu za prevenciju, smanjenje ili liječenje epizode krvarenja kod subjekta, profilaktičko liječenje epizode krvarenja kod subjekta, o liječenje epizode krvarenja na zahtjev kod subjekta, ili za održavanje homeostaze kod subjekta kojem je potreban operativni zahvat.
16. Farmaceutski pripravak za uporabu prema zahtjevu 15, naznačen time što je epizoda krvarenja povezana s bolešću ili stanjem koje se sastoji od hemartroze, krvarenja mišića, oralnog krvarenja, krvarenja, krvarenja u mišiće, oralnog krvarenja, trauma, trauma capitis (ozljede glave), gastrointestinalnog krvarenja, intrakranijalnog krvarenja, intraabdominalnog krvarenja, intratorakalnog krvarenja, prijeloma kostiju, krvarenja u središnjem živčanom sustavu, krvarenja u retrofaringealnom prostoru, krvarenja u retroperitonealnom prostoru, ili krvarenja u iliopsoas ovojnici.
17. Farmaceutski pripravak za uporabu prema zahtjevu 16, naznačen time što je bolest ili stanje hemofilija A.
18. Farmaceutski pripravak za uporabu prema zahtjevu 16, naznačen time što je operacija mala operacija, velika operacija, vađenje zuba, tonzilektomija, ingvinalna herniotomija, sinovektomija, cjelokupna zamjena koljena, kraniotomija, osteosinteza, operacija ozljede, intrakranijalna operacija, intraabdominalna operacija, intratorakalna operacija, hitna operacija, ili operacija zamjene zglobova.
19. Farmaceutski pripravak prema bilo kojem od zahtjeva 1 do 14, ili farmaceutski pripravak za uporabu prema bilo kojem od zahtjeva 15-18, naznačen time što je liofiliziran.
HRP20220639TT 2011-07-08 2012-07-06 Kimerni i hibridni polipeptidi faktora viii i postupci njihove upotrebe HRP20220639T1 (hr)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201161506015P 2011-07-08 2011-07-08
US201161522647P 2011-08-11 2011-08-11
US201161541561P 2011-09-30 2011-09-30
US201161569158P 2011-12-09 2011-12-09
US201261586443P 2012-01-13 2012-01-13
US201261622789P 2012-04-11 2012-04-11
US201261657641P 2012-06-08 2012-06-08
EP18211156.7A EP3513804B1 (en) 2011-07-08 2012-07-06 Factor viii chimeric and hybrid polypeptides, and methods of use thereof

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HRP20190535TT HRP20190535T1 (hr) 2011-07-08 2019-03-19 Kimerni i hibridni polipeptidi faktora viii i postupci njihove upotrebe

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US (3) US10010622B2 (hr)
EP (3) EP4169525A1 (hr)
JP (5) JP2014522838A (hr)
KR (2) KR101829603B1 (hr)
CN (2) CN103796670A (hr)
AR (1) AR087091A1 (hr)
AU (3) AU2012282875B2 (hr)
BR (1) BR112014000466A2 (hr)
CA (1) CA2841066C (hr)
CL (1) CL2014000024A1 (hr)
CO (1) CO6940381A2 (hr)
CY (1) CY1122900T1 (hr)
DK (2) DK3513804T3 (hr)
EA (1) EA029045B1 (hr)
ES (2) ES2722209T3 (hr)
HR (2) HRP20220639T1 (hr)
HU (1) HUE043763T2 (hr)
IL (2) IL230333B (hr)
LT (2) LT3513804T (hr)
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PT (2) PT3513804T (hr)
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SI (2) SI2729161T1 (hr)
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