HRP20210894T1 - Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti - Google Patents
Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti Download PDFInfo
- Publication number
- HRP20210894T1 HRP20210894T1 HRP20210894TT HRP20210894T HRP20210894T1 HR P20210894 T1 HRP20210894 T1 HR P20210894T1 HR P20210894T T HRP20210894T T HR P20210894TT HR P20210894 T HRP20210894 T HR P20210894T HR P20210894 T1 HRP20210894 T1 HR P20210894T1
- Authority
- HR
- Croatia
- Prior art keywords
- preparation
- rhgaa
- molecules
- miglustat
- potential
- Prior art date
Links
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 title claims 4
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 title claims 3
- 206010053185 Glycogen storage disease type II Diseases 0.000 title claims 3
- 239000002253 acid Substances 0.000 title claims 3
- 108010028144 alpha-Glucosidases Proteins 0.000 title claims 3
- 201000004502 glycogen storage disease II Diseases 0.000 title claims 3
- 230000003190 augmentative effect Effects 0.000 title 1
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims 19
- 102000045921 human GAA Human genes 0.000 claims 19
- UQRORFVVSGFNRO-UTINFBMNSA-N miglustat Chemical compound CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO UQRORFVVSGFNRO-UTINFBMNSA-N 0.000 claims 9
- 229960001512 miglustat Drugs 0.000 claims 9
- 230000004988 N-glycosylation Effects 0.000 claims 8
- 239000002552 dosage form Substances 0.000 claims 8
- 238000001990 intravenous administration Methods 0.000 claims 4
- 238000001802 infusion Methods 0.000 claims 3
- 241000699802 Cricetulus griseus Species 0.000 claims 2
- ZAIHWUXYHDAQEV-YXTXVXLGSA-N P(=O)(O)(O)O.O=C[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO.O=C[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO Chemical compound P(=O)(O)(O)O.O=C[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO.O=C[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO ZAIHWUXYHDAQEV-YXTXVXLGSA-N 0.000 claims 2
- 102000016679 alpha-Glucosidases Human genes 0.000 claims 2
- 210000001672 ovary Anatomy 0.000 claims 2
- VFRROHXSMXFLSN-KVTDHHQDSA-N aldehydo-D-mannose 6-phosphate Chemical compound OP(=O)(O)OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)C=O VFRROHXSMXFLSN-KVTDHHQDSA-N 0.000 claims 1
- 150000001413 amino acids Chemical class 0.000 claims 1
- 238000002648 combination therapy Methods 0.000 claims 1
- 238000006206 glycosylation reaction Methods 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 230000004481 post-translational protein modification Effects 0.000 claims 1
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
- A61K31/05—Phenols
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- 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
-
- 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)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Wood Science & Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
- Medicinal Preparation (AREA)
Claims (11)
1. Pripravak koji sadrži molekule ljudske rekombinantne kisele α-glukozidaze (rhGAA) za upotrebu u liječenju Pompeove bolesti u kombinaciji s miglustatom kod pacijenta kojem je to potrebno;
naznačen time što se pripravak daje intravenski u dozi od 5 mg/kg do 20 mg/kg i miglustat se daje oralno u dozi od 260 mg ili 130 mg;
pri čemu se molekule rhGAA proizvode u stanicama jajnika kineskog hrčka (CHO), molekule rhGAA sadrže sedam potencijalnih mjesta N-glikozilacije, 40%-60% N-glikana na molekulama rhGAA su kompleksni tip N-glikana, te najmanje 50% molekula rhGAA nosi jedinicu bis-manoza-6-fosfat (bis-M6P) na prvom potencijalnom mjestu N-glikozilacije.
2. Pripravak za uporabu prema patentnom zahtjevu 1, naznačen time što su molekule rhGAA eksprimirane sa
(i) sekvencom koja je najmanje 95% identična sa SEQ ID NO: 4; ili
(ii) sekvencom SEQ ID NO: 4, pri čemu molekule rhGAA prolaze post-translacijsku modifikaciju kako bi se uklonilo prvih 56 aminokiselina.
3. Pripravak za uporabu prema patentnom zahtjevu 1 ili zahtjevu 2, naznačen time što najmanje 55% molekula rhGAA nosi jedinicu bis-M6P na prvom potencijalnom mjestu N-glikozilacije.
4. Pripravak za uporabu prema bilo kojem od patentnih zahtjeva 1-3, naznačen time što je najmanje 70% molekula rhGAA fosforilirano na prvom potencijalnom mjestu N-glikozilacije.
5. Pripravak za uporabu prema bilo kojem od patentnih zahtjeva 1-4, naznačen time što najmanje 40% molekula rhGAA nosi jedinicu mono-manoza-6-fosfat (mono-M6P) na drugom potencijalnom mjestu N-glikozilacije.
6. Pripravak za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što najmanje 40% molekula rhGAA nosi jedinicu bis-M6P na četvrtom potencijalnom mjestu N-glikozilacije.
7. Pripravak za uporabu prema bilo kojem od patentnih zahtjeva 1-6, naznačen time što najmanje 25% molekula rhGAA nosi jedinicu mono-M6P na četvrtom potencijalnom mjestu N-glikozilacije.
8. Pripravak za uporabu prema bilo kojem od patentnih zahtjeva 1-7, naznačen time što se pripravak daje u dozi od 20 mg/kg intravenskom infuzijom tijekom približno četiri sata svaka 2 tjedna, pri čemu se miglustat daje jedan sat prije intravenske infuzije pripravka, i pri čemu pacijent posti tijekom najmanje dva sata prije i najmanje dva sata nakon oralne primjene miglustata.
9. Pripravak za uporabu prema bilo kojem od patentnih zahtjeva 1-8, naznačen time što se pripravak daje intravenski u dozi od 20 mg/kg i miglustat se daje oralno u dozi od 260 mg.
10. Komplet za kombiniranu terapiju Pompeove bolesti kod pacijenta kojem je to potrebno, te komplet uključuje farmaceutski prihvatljivi oblik doziranja koji sadrži miglustat konfiguriran za oralnu primjenu u dozi od 260 mg ili 130 mg, farmaceutski prihvatljiv oblik doziranja koji sadrži molekule ljudske rekombinantne kisele α-glukozidaze (rhGAA) konfigurirane za intravensku primjenu u dozi od 5 mg/kg do 20 mg/kg, i upute za primjenu farmaceutski prihvatljivog oblika doziranja koji sadrži miglustat i farmaceutski prihvatljivog oblika doziranja koji sadrži molekule rhGAA pacijentu kojem je to potrebno;
pri čemu se molekule rhGAA proizvode u stanicama jajnika kineskog hrčka (CHO), molekule rhGAA sadrže sedam potencijalnih mjesta N-glikozilacije, i najmanje 50% molekula rhGAA nosi jedinicu bis-manoza-6-fosfat (bis-M6P) na prvom potencijalnom mjestu N-glikozilacije.
11. Komplet u skladu s patentnim zahtjevom 10, naznačen time što upute sadrže upute za primjenu farmaceutski prihvatljivog oblika doziranja koji sadrži molekule rhGAA u dozi od 20 mg/kg intravenskom infuzijom tijekom približno četiri sata svaka 2 tjedna, pri čemu upute sadrže upute za primjenu farmaceutski prihvatljivog oblika doziranja koji sadrži molekule rhGAA jedan sat nakon oralne primjene farmaceutski prihvatljivog oblika doziranja koji sadrži miglustat, i pri čemu upute nadalje sadrže upute da pacijent posti tijekom najmanje dva sata prije i najmanje dva sata nakon oralne primjene farmaceutski prihvatljivog oblika doziranja koji sadrži miglustat.
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562272890P | 2015-12-30 | 2015-12-30 | |
| US201662300479P | 2016-02-26 | 2016-02-26 | |
| US201662315412P | 2016-03-30 | 2016-03-30 | |
| US201662402454P | 2016-09-30 | 2016-09-30 | |
| US201662428867P | 2016-12-01 | 2016-12-01 | |
| US201662431791P | 2016-12-08 | 2016-12-08 | |
| EP16826898.5A EP3397273B1 (en) | 2015-12-30 | 2016-12-29 | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| PCT/US2016/069243 WO2017117407A1 (en) | 2015-12-30 | 2016-12-29 | Augmented acid alpha-glucosidase for the treatment of pompe disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20210894T1 true HRP20210894T1 (hr) | 2021-07-23 |
Family
ID=60186557
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20231465TT HRP20231465T1 (hr) | 2015-12-30 | 2016-12-29 | Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti |
| HRP20210894TT HRP20210894T1 (hr) | 2015-12-30 | 2021-06-07 | Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20231465TT HRP20231465T1 (hr) | 2015-12-30 | 2016-12-29 | Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti |
Country Status (15)
| Country | Link |
|---|---|
| EP (3) | EP3957320B1 (hr) |
| CN (1) | CN108472340A (hr) |
| CY (1) | CY1124637T1 (hr) |
| DK (2) | DK3957320T3 (hr) |
| ES (2) | ES2883844T3 (hr) |
| FI (1) | FI3957320T3 (hr) |
| HK (1) | HK1253014A1 (hr) |
| HR (2) | HRP20231465T1 (hr) |
| HU (2) | HUE054733T2 (hr) |
| LT (2) | LT3957320T (hr) |
| PL (2) | PL3397273T3 (hr) |
| PT (2) | PT3397273T (hr) |
| RS (2) | RS62212B1 (hr) |
| SI (2) | SI3957320T1 (hr) |
| TW (2) | TW202245831A (hr) |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7723296B2 (en) * | 2001-01-18 | 2010-05-25 | Genzyme Corporation | Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof |
| PT2444102E (pt) | 2003-01-31 | 2015-09-17 | Sinai School Medicine | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
| JP4914224B2 (ja) * | 2004-02-10 | 2012-04-11 | バイオマリン ファーマシューティカル インコーポレイテッド | 酸性αグルコシダーゼおよびそのフラグメント |
| MX2007014470A (es) | 2005-05-17 | 2008-02-06 | Amicus Therapeutics Inc | Metodo para el tratamiento de enfermedad de pompe usando derivados de 1-desoxinojirimicina. |
| EP2946785B1 (en) | 2008-02-12 | 2018-10-24 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| CN102574887A (zh) * | 2009-05-26 | 2012-07-11 | 阿米库斯治疗学公司 | 利用药物分子伴侣改善生物制剂的生产和纯化 |
| HUE053565T2 (hu) | 2012-05-03 | 2021-07-28 | Amicus Therapeutics Inc | Adagolási rendek Pompe betegség kezelésére |
| EP2874648A4 (en) | 2012-07-17 | 2015-12-30 | Amicus Therapeutics Inc | CO-FORMULATION OF ALPHA-GALACTOSIDASE A AND 1-DEOXYGALACTONOJIRIMYCIN |
| RS65066B1 (sr) * | 2014-09-30 | 2024-02-29 | Amicus Therapeutics Inc | Visoko potentna kisela alfa-glukozidaza sa pojačanim ugljenim hidratima |
-
2016
- 2016-12-29 CN CN201680077801.5A patent/CN108472340A/zh active Pending
- 2016-12-29 PL PL16826898T patent/PL3397273T3/pl unknown
- 2016-12-29 HU HUE16826898A patent/HUE054733T2/hu unknown
- 2016-12-29 PT PT168268985T patent/PT3397273T/pt unknown
- 2016-12-29 DK DK21174249.9T patent/DK3957320T3/da active
- 2016-12-29 LT LTEP21174249.9T patent/LT3957320T/lt unknown
- 2016-12-29 EP EP21174249.9A patent/EP3957320B1/en active Active
- 2016-12-29 ES ES16826898T patent/ES2883844T3/es active Active
- 2016-12-29 HU HUE21174249A patent/HUE064049T2/hu unknown
- 2016-12-29 RS RS20210984A patent/RS62212B1/sr unknown
- 2016-12-29 SI SI201631766T patent/SI3957320T1/sl unknown
- 2016-12-29 HR HRP20231465TT patent/HRP20231465T1/hr unknown
- 2016-12-29 LT LTEP16826898.5T patent/LT3397273T/lt unknown
- 2016-12-29 RS RS20231076A patent/RS64843B1/sr unknown
- 2016-12-29 DK DK16826898.5T patent/DK3397273T3/da active
- 2016-12-29 EP EP23188938.7A patent/EP4285902A3/en active Pending
- 2016-12-29 EP EP16826898.5A patent/EP3397273B1/en active Active
- 2016-12-29 PL PL21174249.9T patent/PL3957320T3/pl unknown
- 2016-12-29 ES ES21174249T patent/ES2965190T3/es active Active
- 2016-12-29 PT PT211742499T patent/PT3957320T/pt unknown
- 2016-12-29 FI FIEP21174249.9T patent/FI3957320T3/fi active
- 2016-12-29 SI SI201631258T patent/SI3397273T1/sl unknown
- 2016-12-30 TW TW111105262A patent/TW202245831A/zh unknown
- 2016-12-30 TW TW105144116A patent/TWI753874B/zh active
-
2018
- 2018-09-27 HK HK18112390.1A patent/HK1253014A1/zh unknown
-
2021
- 2021-06-07 HR HRP20210894TT patent/HRP20210894T1/hr unknown
- 2021-08-06 CY CY20211100705T patent/CY1124637T1/el unknown
Also Published As
| Publication number | Publication date |
|---|---|
| RS64843B1 (sr) | 2023-12-29 |
| HUE064049T2 (hu) | 2024-02-28 |
| HRP20231465T1 (hr) | 2024-03-01 |
| ES2965190T3 (es) | 2024-04-11 |
| DK3397273T3 (da) | 2021-08-09 |
| TWI753874B (zh) | 2022-02-01 |
| LT3957320T (lt) | 2023-12-11 |
| FI3957320T3 (fi) | 2023-11-23 |
| EP3397273A1 (en) | 2018-11-07 |
| EP4285902A2 (en) | 2023-12-06 |
| CN108472340A (zh) | 2018-08-31 |
| DK3957320T3 (da) | 2023-11-27 |
| LT3397273T (lt) | 2021-06-25 |
| TW202245831A (zh) | 2022-12-01 |
| EP3957320A1 (en) | 2022-02-23 |
| PL3397273T3 (pl) | 2021-12-06 |
| PL3957320T3 (pl) | 2024-02-19 |
| PT3397273T (pt) | 2021-08-09 |
| EP3957320B1 (en) | 2023-08-23 |
| EP4285902A3 (en) | 2024-02-21 |
| TW201726163A (zh) | 2017-08-01 |
| SI3397273T1 (sl) | 2021-11-30 |
| PT3957320T (pt) | 2023-11-23 |
| RS62212B1 (sr) | 2021-09-30 |
| CY1124637T1 (el) | 2022-07-22 |
| HUE054733T2 (hu) | 2021-09-28 |
| EP3397273B1 (en) | 2021-05-19 |
| SI3957320T1 (sl) | 2024-01-31 |
| ES2883844T3 (es) | 2021-12-09 |
| HK1253014A1 (zh) | 2019-06-06 |
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| HRP20210894T1 (hr) | Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti |