HRP20230576T1 - Rekombinantna ljudska kisela alfa-glukozidaza - Google Patents

Rekombinantna ljudska kisela alfa-glukozidaza Download PDF

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Publication number
HRP20230576T1
HRP20230576T1 HRP20230576TT HRP20230576T HRP20230576T1 HR P20230576 T1 HRP20230576 T1 HR P20230576T1 HR P20230576T T HRP20230576T T HR P20230576TT HR P20230576 T HRP20230576 T HR P20230576T HR P20230576 T1 HRP20230576 T1 HR P20230576T1
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Croatia
Prior art keywords
rhgaa
moles
subject
potential
use according
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HRP20230576TT
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English (en)
Inventor
Hung Do
Russell GOTSCHALL
Richie Khanna
Yi Lun
Hing CHAR
Sergey TESLER
Wendy SUNDERLAND
Enrique DILONÉ
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Amicus Therapeutics, Inc.
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Application filed by Amicus Therapeutics, Inc. filed Critical Amicus Therapeutics, Inc.
Publication of HRP20230576T1 publication Critical patent/HRP20230576T1/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
    • A61K38/43Enzymes; 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular 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
    • 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
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Claims (9)

1. Pripravak koji sadrži populaciju molekula rekombinantne ljudske kisele α-glukozidaze (rhGAA) za upotrebu u liječenju Pompeove bolesti kod subjekta kojem je to potrebno, pri čemu su molekule rhGAA proizvedene iz stanica jajnika kineskog hrčka (CHO); pri čemu molekule rhGAA sadrže sedam potencijalnih N-glikozilacijskih mjesta; pri čemu molekule rhGAA u prosjeku sadrže 3-4 ostatka manoza-6-fosfata (M6P); pri čemu molekule rhGAA u prosjeku sadrže najmanje oko 0,5 mola bis-manoza-6-fosfata (bis-M6P) po molu rhGAA na prvom potencijalnom mjestu N-glikozilacije kako je određeno uporabom tekućinske kromatografije-tandem masene spektrometrije (LC-MS/MS); pri čemu se populacija rhGAA molekula primjenjuje istovremeno ili uzastopno s farmakološkim pratiocem, i pri čemu je farmakološki pratilac miglustat ili njegova farmaceutski prihvatljiva sol; pri čemu se populacija rhGAA molekula primjenjuje intravenozno u dozi od oko 20 mg/kg, a miglustat ili njegova farmaceutski prihvatljiva sol se primjenjuje oralno u dozi od oko 260 mg; i pri čemu (i) subjekt je pacijent s prebacivanjem na ERT, i, u usporedbi s osnovnom vrijednošću, subjektova plućna funkcija, mjerena testom uspravnog forsiranog vitalnog kapaciteta (FVC), stabilna je ili poboljšana šest mjeseci nakon liječenja; i/ili (ii) subjekt je ambulantni pacijent s prebacivanjem na ERT i, u usporedbi s osnovnom vrijednošću, subjektova razina kreatin kinaze šest mjeseci nakon tretmana smanjena je za najmanje 15%; i/ili (iii) subjekt je ne ambulantni pacijent s prebacivanjem na ERT i, u usporedbi s osnovnom vrijednošću, subjektova razina kreatin kinaze šest mjeseci nakon tretmana smanjena je za najmanje 20%; i/ili (iv) subjekt je pacijent s prebacivanjem na ERT, i, u usporedbi s početnom vrijednošću, razine heksoza tetrasaharida u urinu subjekta šest mjeseci nakon liječenja smanjene su za najmanje 35%.
2. Pripravak za upotrebu prema zahtjevu 1, naznačen time što se miglustat ili njegova farmaceutski prihvatljiva sol primjenjuje prije primjene rhGAA.
3. Pripravak za upotrebu prema zahtjevu 1 ili zahtjevu 2, naznačen time što molekule rhGAA sadrže sekvencu aminokiselina najmanje 95% identičnu sa SEQ ID NO:1 ili SEQ ID NO:5.
4. Pripravak za upotrebu prema bilo kojem od zahtjeva 1 do 3, naznačen time što molekule rhGAA sadrže u prosjeku od oko 0,5 mola do oko 7,0 mola mono-M6P ili bis-M6P po molu rhGAA, kako je određeno pomoću LC-MS/MS.
5. Pripravak za upotrebu prema bilo kojem od zahtjeva 1 do 4, naznačen time što molekule rhGAA sadrže u prosjeku najmanje 2,5 mol M6P po molu rhGAA i najmanje 4 mol sijalinske kiseline po molu rhGAA, kako je određeno pomoću LC-MS/MS.
6. Pripravak za upotrebu prema bilo kojem od zahtjeva 1 do 5, naznačen time što, po molu rhGAA, molekule rhGAA sadrže u prosjeku: (a) oko 0,4 do oko 0,6 mola mono-M6P na drugom potencijalnom mjestu N-glikozilacije; (b) oko 0,4 do oko 0,6 mola bis-M6P na četvrtom potencijalnom mjestu N-glikozilacije; i (c) oko 0,3 do oko 0,4 mola mono-M6P na četvrtom potencijalnom mjestu N-glikozilacije; pri čemu su (a)-(c) određeni pomoću LC-MS/MS.
7. Pripravak za upotrebu prema bilo kojem od zahtjeva 1to 6, naznačen time što, po molu rhGAA, molekule rhGAA nadalje sadrže oko 4 mola do oko 7,3 mola sijalinske kiseline; i pri čemu, po molu rhGAA, molekule rhGAA sadrže u prosjeku: (a) oko 0,9 do oko 1,2 mola sijalinske kiseline na trećem potencijalnom mjestu N-glikozilacije; (b) oko 0,8 do oko 0,9 mola sijalinske kiseline na petom potencijalnom mjestu N-glikozilacije; i (c) oko 1,5 do oko 4,2 mola sijalinske kiseline na šestom potencijalnom mjestu N-glikozilacije; pri čemu su (a)-(c) određeni pomoću LC-MS/MS.
8. Pripravak za upotrebu prema bilo kojem od zahtjeva 1 do 7, naznačen time što je populacija rhGAA molekula formulirana u farmaceutskom pripravku, i pri čemu farmaceutski pripravak nadalje sadrži najmanje jedan pufer odabran iz skupine koja se sastoji od citrata, fosfata, i njihove kombinacije, te najmanje jednu pomoćnu tvar odabranu iz skupine koju čine manitol, polisorbat 80 i njihova kombinacija; pri čemu farmaceutski pripravak ima pH od oko 5,0 do oko 7,0.
9. Pripravak za upotrebu prema bilo kojem od zahtjeva 1 do 8, naznačen time što, je u farmaceutskom pripravku populacija molekula rhGAA prisutna u koncentraciji od oko 5-50 mg/mL, najmanje jedan pufer je natrijev citratni pufer prisutan u koncentraciji od oko 10-100 mM, najmanje jedna pomoćnu tvar je manitol prisutan u koncentraciji od oko 10-50 mg/mL i polisorbat 80 je prisutan u koncentraciji od oko 0.1-1 mg/mL, te farmaceutski pripravak nadalje sadrži vodu i izborno sadrži sredstvo za zakiseljavanje i/ili sredstvo za alkalizaciju; pri čemu farmaceutski pripravak ima pH od oko 6,0.
HRP20230576TT 2017-05-15 2018-05-15 Rekombinantna ljudska kisela alfa-glukozidaza HRP20230576T1 (hr)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201762506561P 2017-05-15 2017-05-15
US201762506569P 2017-05-15 2017-05-15
US201762506574P 2017-05-15 2017-05-15
US201762529300P 2017-07-06 2017-07-06
US201762564083P 2017-09-27 2017-09-27
US201762567334P 2017-10-03 2017-10-03
US201862618021P 2018-01-16 2018-01-16
US201862624638P 2018-01-31 2018-01-31
US201862660758P 2018-04-20 2018-04-20
EP18802722.1A EP3624831B1 (en) 2017-05-15 2018-05-15 Recombinant human acid alpha-glucosidase
PCT/US2018/032815 WO2018213340A1 (en) 2017-05-15 2018-05-15 Recombinant human acid alpha-glucosidase

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US (1) US20210393747A1 (hr)
EP (2) EP4223310A3 (hr)
JP (2) JP7193476B2 (hr)
KR (1) KR20200004414A (hr)
CN (1) CN111356474A (hr)
AU (1) AU2018270925A1 (hr)
BR (1) BR112019024125A2 (hr)
CA (1) CA3063615A1 (hr)
CL (1) CL2019003251A1 (hr)
DK (1) DK3624831T5 (hr)
ES (1) ES2950808T3 (hr)
FI (1) FI3624831T3 (hr)
HR (1) HRP20230576T1 (hr)
HU (1) HUE062504T2 (hr)
IL (2) IL270530B2 (hr)
LT (1) LT3624831T (hr)
MX (1) MX2019013624A (hr)
PL (1) PL3624831T3 (hr)
PT (1) PT3624831T (hr)
RS (1) RS64301B1 (hr)
SI (1) SI3624831T1 (hr)
TW (1) TW201900208A (hr)
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EP3871687A1 (en) * 2020-02-27 2021-09-01 eleva GmbH Enzyme replacement therapy for treating pompe disease
IL305103A (en) * 2021-02-11 2023-10-01 Amicus Therapeutics Inc Recombinant human acid alpha glucosidase and uses thereof
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WO2023215865A1 (en) * 2022-05-05 2023-11-09 Amicus Therapeutics, Inc. Methods for treating pompe disease
WO2024119070A1 (en) * 2022-12-02 2024-06-06 Amicus Therapeutics, Inc. Methods for treating late onset pompe disease in pediatric patients
WO2024119091A1 (en) * 2022-12-02 2024-06-06 Amicus Therapeutics, Inc. Fexamethods for treating infantile-onset pompe disease in pediatric patients

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PT3470077T (pt) 2008-02-12 2020-11-30 Amicus Therapeutics Inc Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas
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IL270530B2 (en) 2024-07-01
KR20200004414A (ko) 2020-01-13
AU2018270925A1 (en) 2019-11-28
EP3624831A4 (en) 2021-02-17
EP3624831A1 (en) 2020-03-25
WO2018213340A1 (en) 2018-11-22
US20210393747A1 (en) 2021-12-23
EP4223310A3 (en) 2023-10-11
PT3624831T (pt) 2023-07-04
DK3624831T5 (da) 2024-09-02
CL2019003251A1 (es) 2020-05-22
HUE062504T2 (hu) 2023-11-28
TW201900208A (zh) 2019-01-01
BR112019024125A2 (pt) 2020-06-02
CN111356474A (zh) 2020-06-30
IL310719A (en) 2024-04-01
IL270530A (en) 2020-01-30
ZA201907552B (en) 2024-09-25
IL270530B1 (en) 2024-03-01
EP3624831B1 (en) 2023-03-29
PL3624831T3 (pl) 2023-08-14
MX2019013624A (es) 2020-01-13
ES2950808T3 (es) 2023-10-13
CA3063615A1 (en) 2018-11-22
JP2023051927A (ja) 2023-04-11
SI3624831T1 (sl) 2023-08-31
EP4223310A2 (en) 2023-08-09
DK3624831T3 (da) 2023-06-26
FI3624831T3 (fi) 2023-06-12
JP7193476B2 (ja) 2022-12-20
LT3624831T (lt) 2023-07-10
JP2020519638A (ja) 2020-07-02
RS64301B1 (sr) 2023-07-31

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