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ME02062B - Primena lizozomalne kisele lipaze za lecenje nedostatka lizozomalne kisele lipaze kod pacijenata - Google Patents

Primena lizozomalne kisele lipaze za lecenje nedostatka lizozomalne kisele lipaze kod pacijenata

Info

Publication number
ME02062B
ME02062B MEP-2015-41A MEP4115A ME02062B ME 02062 B ME02062 B ME 02062B ME P4115 A MEP4115 A ME P4115A ME 02062 B ME02062 B ME 02062B
Authority
ME
Montenegro
Prior art keywords
recombinant human
lal
use according
human lal
sufficient
Prior art date
Application number
MEP-2015-41A
Other languages
English (en)
Inventor
Anthony Quinn
Original Assignee
Synageva Biopharma Corp
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=45806910&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ME02062(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from PCT/US2011/033699 external-priority patent/WO2011133960A2/en
Application filed by Synageva Biopharma Corp filed Critical Synageva Biopharma Corp
Publication of ME02062B publication Critical patent/ME02062B/me

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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
    • A61K38/43Enzymes; 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/13Amines
    • A61K31/135Amines having aromatic rings, e.g. ketamine, nortriptyline
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/13Amines
    • A61K31/135Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/138Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365Lactones
    • A61K31/366Lactones having six-membered rings, e.g. delta-lactones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/397Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having four-membered rings, e.g. azetidine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • 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)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18Carboxylic ester hydrolases (3.1.1)
    • 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)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18Carboxylic ester hydrolases (3.1.1)
    • C12N9/20Triglyceride splitting, e.g. by means of lipase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/01Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01013Sterol esterase (3.1.1.13)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Claims (33)

1.Rekombinantna humana lizozomalna kisela lipaze (Lysosomal acid lipase) (LAL) za primenu u lečenju ljudskog pacijenta koji pati od nedostatka LAL primenom na ljudskog pacijenta, pri čemu je primena dovoljna da normalizuje serumske nivoe transaminaze iz jetre navedenog ljudskog pacijenta i da smanji oštećenje jetre kod navedenog ljudskog pacijenta, i pri čemu rekombinanatna humana LAL je za primenu na ljudskog pacijenta između jednog puta svakih 7 dana i jednog puta svakih 30 dana.
2.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena transaminaza jetre je odabrana iz grupe koja se sastoji od seruma aspartata transaminaze (AST) i alanin transaminaze (ALT).
3.Rekombinantna humana LAL za primenu prema zahtevu 2, pri čemu navedena transaminaza jetre je AST.
4.Rekombinantna humana LAL za primenu prema zahtevu 2, pri čemu navedena transaminaza jetre je ALT.
5. Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da se minimizira hepatomegalija.
6. Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da poveća serumske nivoe hemoglobina.
7. Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da smanji veličinu jetre.
8.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da smanji serumske nivoe feritina.
9.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena rekombinanatna humana LAL se primenjuje jedan put svakih 7 dana.
10.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena rekombinantna humana LAL se primenjuje jedan put svakih 14 dana.
11.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedeni ljudski pacijent pati od Wolmanove bolesti.
12.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedeni ljudski pacijent pati od obolenja skladištenja holesteril estra.
13.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu rekombinantna humana LAL obuhvata bar jednu manozu ili manoza-6-fosfat.
14.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena količina rekombinantne humane LAL dovoljna da normalizuje serumske nivoe transaminaze jetre je oko 1 mg po kilogramu telesne težine navedenog ljudskog pacijenta.
15.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da se postigne LAL polu-vek (t1/2) koji je manji od 20 minuta.
16.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da se postigne LAL polu-vek (t1/2) od oko 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16 ili 17 minuta.
17.Rekombinanatna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da se postigne Cmax od 200 ng/mL do 1500 ng/mL.
18.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da se postigne Cmax od 200 ng/mL do 800 ng/mL.
19.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena rekombinantna humana LAL se primenjuje intravenozno.
20.Rekombinanatna humana LAL za primenu prema zahtevu 19, pri čemu navedena primena je infuzijom.
21.Rekombinantna humana LAL za primenu prema zahtevu 20, pri čemu infuzija je za jedan do četiri sata.
22. Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedena primena je dovoljna da smanji limfadenopatiju.
23.Rekombinantna humana LAL za primenu prema zahtevu 1, pri čemu navedeni ljudski pacijent je manji od 1 godine starosti i navedena primena je dovoljna da se poveća brzina rasta navedenog ljudskog pacijenta.
24.Rekombinantna humana LAL za primenu prema zahtevu 1, dalje obuhvata primenu drugog leka.
25.Rekombinantna humana LAL za primenu prema zahtevu 24, pri čemu drugi lek je lek koji smanjuje holesterol.
26.Rekombinantna humana LAL za primenu prema zahtevu 25, pri čemu navedeni lek za smanjenje holesterola je statin.
27.Rekombinanatna humana LAL za primenu prema zahtevu 25, pri čemu navedeni lek koji smanjuje holesterol je ezetimib.
28. Rekombinantna humana LAL za primenu prema zahtevu 24, pri čemu drugi lek je imunosupresant.
29.Rekombinantna humana LAL za primenu prema zahtevu 24, pri čemu navedeni drugi lek je antihistamin.
30.Rekombinantna humana LAL za primenu prema zahtevu 29, pri čemu navedeni antihistamin je difenhidramin.
31.Rekombinanatna humana LAL za primenu prema zahtevu 29 ili 30, pri čemu antihistamin se primenjuje u dozi od 1 mg do 5 mg po kilogramu telesne težine navedenog ljudskog pacijenta.
32.Rekombinantna humana LAL za primenu prema zahtevu 29 ili 30, pri čemu antihistamin se primenjuje između 20 i 90 minuta pre primene rekombinantne humane LAL.
33.Primena rekombinantne humane lizozomalne kisele lipaze (Lysosomal acid lipase) (LAL) u proizvodnji medikamenta za lečenje ljudskog pacijenta koji pati od nedostatka LAL normalizovanjem serumskih nivoa transaminaze jetre navedenog ljudskog pacijenta i smanjenjem oštećenja jetre kod navedenog ljudskog pacijenta između jednog puta svakih 7 dana i jednog puta svakih 30 dana kao što je definisano u bilo kom od zahteva 1 do 32.
MEP-2015-41A 2010-09-09 2011-09-09 Primena lizozomalne kisele lipaze za lecenje nedostatka lizozomalne kisele lipaze kod pacijenata ME02062B (me)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US40301110P 2010-09-09 2010-09-09
US45601410P 2010-10-29 2010-10-29
US201161432372P 2011-01-13 2011-01-13
PCT/US2011/033699 WO2011133960A2 (en) 2010-04-23 2011-04-23 Lysosomal storage disease enzyme
PCT/US2011/051096 WO2012050695A1 (en) 2010-09-09 2011-09-09 Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
EP11758644.6A EP2613798B2 (en) 2010-09-09 2011-09-09 Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients

Publications (1)

Publication Number Publication Date
ME02062B true ME02062B (me) 2015-05-20

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US (6) US8663631B2 (me)
EP (3) EP2613798B2 (me)
JP (3) JP5693728B2 (me)
KR (1) KR20150038636A (me)
CN (2) CN105457018A (me)
AR (1) AR082953A1 (me)
AU (1) AU2011314293B2 (me)
BR (2) BR112013005673B1 (me)
CA (2) CA2810999C (me)
CL (1) CL2013000664A1 (me)
CO (1) CO6710913A2 (me)
CY (1) CY1116544T1 (me)
DK (2) DK2977057T3 (me)
ES (2) ES2535605T5 (me)
HK (2) HK1220907A1 (me)
HR (1) HRP20150438T1 (me)
HU (1) HUE048688T2 (me)
IL (2) IL225095A (me)
ME (1) ME02062B (me)
MX (2) MX2013002704A (me)
NZ (3) NZ700824A (me)
PL (2) PL2613798T5 (me)
PT (2) PT2977057T (me)
RS (1) RS53947B1 (me)
RU (1) RU2550961C2 (me)
SG (2) SG188456A1 (me)
SI (2) SI2977057T1 (me)
SM (1) SMT201500108B (me)
TW (1) TWI482625B (me)
WO (1) WO2012050695A1 (me)

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