HRP20230576T1 - Rekombinantna ljudska kisela alfa-glukozidaza - Google Patents
Rekombinantna ljudska kisela alfa-glukozidaza Download PDFInfo
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- 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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- rhgaa
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Gastroenterology & Hepatology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
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- Biochemistry (AREA)
- Biomedical Technology (AREA)
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- Genetics & Genomics (AREA)
- General Engineering & Computer Science (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.
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762506569P | 2017-05-15 | 2017-05-15 | |
| US201762506561P | 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 | |
| PCT/US2018/032815 WO2018213340A1 (en) | 2017-05-15 | 2018-05-15 | Recombinant human acid alpha-glucosidase |
| EP18802722.1A EP3624831B1 (en) | 2017-05-15 | 2018-05-15 | Recombinant human acid alpha-glucosidase |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20230576T1 true HRP20230576T1 (hr) | 2023-09-01 |
Family
ID=64274660
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20230576TT HRP20230576T1 (hr) | 2017-05-15 | 2018-05-15 | Rekombinantna ljudska kisela alfa-glukozidaza |
Country Status (24)
| Country | Link |
|---|---|
| US (2) | US12246062B2 (hr) |
| EP (2) | EP3624831B1 (hr) |
| JP (3) | JP7193476B2 (hr) |
| CN (3) | CN119113091A (hr) |
| AU (1) | AU2018270925B2 (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) |
| SM (1) | SMT202300187T1 (hr) |
| TW (1) | TW202448499A (hr) |
| WO (1) | WO2018213340A1 (hr) |
| ZA (1) | ZA201907552B (hr) |
Families Citing this family (10)
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| KR102760654B1 (ko) | 2017-06-07 | 2025-02-04 | 리제너론 파마슈티칼스 인코포레이티드 | 효소 내재화를 위한 조성물 및 방법 |
| MA51796A (fr) | 2018-02-07 | 2020-12-16 | Regeneron Pharma | Procédés et compositions pour l'administration de protéines thérapeutiques |
| SG11202011232VA (en) | 2018-05-17 | 2020-12-30 | Regeneron Pharma | Anti-cd63 antibodies, conjugates, and uses thereof |
| 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 |
| CN114280182A (zh) * | 2021-12-23 | 2022-04-05 | 福建省中医药科学院 | 一种太子参均一多糖的hplc-fld检测方法 |
| WO2023150387A1 (en) * | 2022-02-07 | 2023-08-10 | M6P Therapeutics, Inc. | Compositions comprising acid alpha glucosidase and methods of use thereof |
| IL316776A (en) * | 2022-05-05 | 2025-01-01 | Amicus Therapeutics Inc | Methods of treating Pompe disease |
| 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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| KR102076585B1 (ko) | 2011-12-22 | 2020-02-12 | 센토제너 아이피 게엠베하 | 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물 |
| MX349992B (es) | 2012-03-07 | 2017-08-22 | Amicus Therapeutics Inc | Composiciones de alta concentracion de alfa-glucosidasa para el tratamiento de la enfermedad de pompe. |
| AU2013234042B2 (en) | 2012-03-15 | 2017-11-02 | Oxyrane Uk Limited | Methods and materials for treatment of Pompe's disease |
| EP4397364A3 (en) | 2012-05-03 | 2024-09-04 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of pompe disease |
| US20150147309A1 (en) | 2012-06-06 | 2015-05-28 | Fondazione Telethon | Allosteric chaperones and uses thereof |
| CN104918619B (zh) * | 2013-01-09 | 2019-04-02 | 阿米库斯治疗学公司 | 稳定的肠胃外dnj组合物 |
| ES2732308T3 (es) | 2013-12-23 | 2019-11-21 | Bcn Peptides Sa | Análogos de bicalutamida o (s)-bicalutamida como compuestos activadores de la exocitosis para su uso en el tratamiento de un desorden de acúmulo lisosomal o glucogenosis |
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| WO2017049161A1 (en) * | 2015-09-18 | 2017-03-23 | Duke University | ACID ALPHA-GLUCOSIDASE AND A β-2 AGONIST FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDER |
| WO2017117407A1 (en) * | 2015-12-30 | 2017-07-06 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| BR112018069965A2 (pt) | 2016-03-30 | 2019-01-29 | Amicus Therapeutics Inc | formulações compreendendo alfa-glucosidase ácida recombinante |
| BR112018070189A2 (pt) | 2016-03-30 | 2019-02-19 | Amicus Therapeutics, Inc. | método para seleção de proteínas recombinantes ricas em m6p |
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