HRP20210047T1 - Arimoclomol za liječenje poremećaja povezanih s glukocerebrozidazom - Google Patents
Arimoclomol za liječenje poremećaja povezanih s glukocerebrozidazom Download PDFInfo
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- HRP20210047T1 HRP20210047T1 HRP20210047TT HRP20210047T HRP20210047T1 HR P20210047 T1 HRP20210047 T1 HR P20210047T1 HR P20210047T T HRP20210047T T HR P20210047TT HR P20210047 T HRP20210047 T HR P20210047T HR P20210047 T1 HRP20210047 T1 HR P20210047T1
- Authority
- HR
- Croatia
- Prior art keywords
- gba
- pharmaceutical ingredient
- active pharmaceutical
- piperidinyl
- propoxy
- Prior art date
Links
- 102000004547 Glucosylceramidase Human genes 0.000 title claims 18
- 108010017544 Glucosylceramidase Proteins 0.000 title claims 18
- SGEIEGAXKLMUIZ-ZPTIMJQQSA-N (3e)-n-[(2r)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl chloride Chemical compound C([C@H](O)CN1CCCCC1)O\N=C(\Cl)C1=CC=C[N+]([O-])=C1 SGEIEGAXKLMUIZ-ZPTIMJQQSA-N 0.000 title 1
- 229950011582 arimoclomol Drugs 0.000 title 1
- 239000008186 active pharmaceutical agent Substances 0.000 claims 17
- 208000018737 Parkinson disease Diseases 0.000 claims 11
- 208000027089 Parkinsonian disease Diseases 0.000 claims 8
- 206010034010 Parkinsonism Diseases 0.000 claims 8
- SGEIEGAXKLMUIZ-PEZBUJJGSA-N (3z)-n-(2-hydroxy-3-piperidin-1-ylpropoxy)-1-oxidopyridin-1-ium-3-carboximidoyl chloride Chemical compound C1CCCCN1CC(O)CO\N=C(/Cl)C1=CC=C[N+]([O-])=C1 SGEIEGAXKLMUIZ-PEZBUJJGSA-N 0.000 claims 7
- 101150028412 GBA gene Proteins 0.000 claims 6
- 208000015872 Gaucher disease Diseases 0.000 claims 3
- 206010064571 Gene mutation Diseases 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- 230000035772 mutation Effects 0.000 claims 3
- VZCYOOQTPOCHFL-UHFFFAOYSA-N trans-butenedioic acid Natural products OC(=O)C=CC(O)=O VZCYOOQTPOCHFL-UHFFFAOYSA-N 0.000 claims 3
- 239000002253 acid Substances 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- 150000001875 compounds Chemical class 0.000 claims 1
- 230000000069 prophylactic effect Effects 0.000 claims 1
- 230000004952 protein activity Effects 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 230000001629 suppression Effects 0.000 claims 1
- 238000013518 transcription Methods 0.000 claims 1
- 230000035897 transcription Effects 0.000 claims 1
Classifications
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- 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
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
- A61K31/4545—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
Claims (15)
1. Aktivni farmaceutski sastojak odabran od N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorida, njegovih stereoizomera i kiselih adicijskih soli, za uporabu u postupku liječenja Parkinsonove bolesti (PD) povezane s glukocerebrozidazom (GBA) ili GBA-povezanog parkinsonizma.
2. Aktivni farmaceutski sastojak za uporabu prema zahtjevu 1 u postupku liječenja GBA-povezane Parkinsonove bolesti.
3. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu navedena GBA-povezana Parkinsonova bolest ili parkinsonizam je povezan/a sa smanjenim razinama GBA enzima i/ili smanjenom aktivnosti GBA enzima.
4. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu navedena GBA-povezana Parkinsonova bolest ili parkinsonizam je povezan/a s jednom ili više individualnih mutacija GBA gena.
5. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu navedena GBA-povezana Parkinsonova bolest ili parkinsonizam je povezan/a s jednom ili više heterozigotnih mutacija GBA gena.
6. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu pojedinac s GBA-povezanom Parkinsonovom bolesti ili parkinsonizmom ostaje klinički nezahvaćen Gaucherovom bolešću.
7. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu navedena jedna ili više individualnih mutacija GBA gena su a) blage (povezane s GD tipa I) ili b) ozbiljne (povezane s GD tipa II i III).
8. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu je navedena mutacija GBA gena odabrana iz skupine koja se sastoji od L444P, D409H, D409V, E235A, E340A, E326K, N370S, N370S/1-BP ins 84G, V394L, A456P, V460V, C342G, G325R, P415R, Y133*, F213I, N188S i IVS2+1G>A/N188S.
9. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu je navedena GBA-povezana Parkinsonova bolest povezana s jednom ili više homozigotnih ili složenih homozigotnih mutacija GBA gena.
10. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva pri čemu navedeni pojedinac s GBA-povezanom Parkinsonovom bolesti ili parkinsonizmom je L444P, A456P, V460V heterozigot.
11. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva pri čemu navedeni pojedinac s GBA-povezanom Parkinsonovom bolesti ili parkinsonizmom je:
a) nositelj GBA mutacije, poput asimptomatskog nositelja, poput nositelja koji je klinički nezahvaćen Gaucherovom bolešću; i/ili
b) klinički nezahvaćeni roditelj ili brat/sestra pacijenta s Gaucherovom bolesti.
12. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od zahtjeva 1 do 3, pri čemu navedena GBA-povezana Parkinsonova bolest ili parkinsonizam je povezan/a sa smanjenom aktivnosti GBA enzima i/ili razinama te navedeni GBA gen je divljeg tipa, i redukcija GBA aktivnosti je zbog suzbijanja aktivnosti proteina ili potiskivanja transkripcije ili translacije gena/proteina, ili je idiopatska.
13. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu navedeni aktivni farmaceutski sastojak je:
a) racemat N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorida;
b) optički aktivni stereoizomer N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorida;
c) enantiomer N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorida;
d) odabran iz skupine koja se sastoji od: (+)-R-N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorida, i (-)-(S)-N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorida;
e) kisela adicijska sol N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorida;
f) odabran iz skupine koja se sastoji od: N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorid citrata, i N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorid maleata; ili
g) odabran iz skupine koja se sastoji od: (+)-R-N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorid citrata, (-)-S-N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorid citrata, (+)-R-N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorid maleata, i (-)-S-N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorid maleata.
14. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu navedeni aktivni farmaceutski sastojak je (+)-R-N-[2-hidroksi-3-(1-piperidinil)-propoksi]-piridin-1-oksid-3-karboksimidoil klorid citrat.
15. Aktivni farmaceutski sastojak za uporabu prema bilo kojem od prethodnih zahtjeva, pri čemu navedeno liječenje je profilaktičko, kurativno ili ublažavajuće.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
DKPA201670281 | 2016-04-29 | ||
PCT/EP2017/060205 WO2017186919A1 (en) | 2016-04-29 | 2017-04-28 | Arimoclomol for treating glucocerebrosidase associated disorders |
EP17720480.7A EP3448382B1 (en) | 2016-04-29 | 2017-04-28 | Arimoclomol for treating glucocerebrosidase associated disorders |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20210047T1 true HRP20210047T1 (hr) | 2021-04-16 |
Family
ID=58645076
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20210047TT HRP20210047T1 (hr) | 2016-04-29 | 2021-01-12 | Arimoclomol za liječenje poremećaja povezanih s glukocerebrozidazom |
Country Status (20)
Country | Link |
---|---|
US (3) | US20190111041A1 (hr) |
EP (2) | EP3448382B1 (hr) |
JP (2) | JP6818045B2 (hr) |
KR (2) | KR102254140B1 (hr) |
CN (1) | CN109069496A (hr) |
AU (2) | AU2017255959B2 (hr) |
BR (1) | BR112018070653A2 (hr) |
CA (1) | CA3021900A1 (hr) |
CY (1) | CY1123717T1 (hr) |
DK (1) | DK3448382T3 (hr) |
ES (1) | ES2831764T3 (hr) |
HR (1) | HRP20210047T1 (hr) |
HU (1) | HUE052158T2 (hr) |
IL (2) | IL262411B (hr) |
LT (1) | LT3448382T (hr) |
PT (1) | PT3448382T (hr) |
RS (1) | RS61291B1 (hr) |
RU (2) | RU2750154C2 (hr) |
SI (1) | SI3448382T1 (hr) |
WO (1) | WO2017186919A1 (hr) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
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CA3183559A1 (en) * | 2020-06-24 | 2021-12-30 | Thomas Kirkegaard Jensen | Arimoclomol for treating gaucher disease |
US20230416224A1 (en) | 2020-11-19 | 2023-12-28 | Zevra Denmark A/S | Processes for preparing arimoclomol citrate and intermediates thereof |
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