ES2754398T3 - Eliglustat (Genz 112638) como inhibidor de glucosilceramida-sintasa para uso en un método de tratamiento de la enfermedad de fabry o de gaucher, comprendiendo el método ajustar la dosis terapéutica individual al metabolismo P-450 del paciente - Google Patents
Eliglustat (Genz 112638) como inhibidor de glucosilceramida-sintasa para uso en un método de tratamiento de la enfermedad de fabry o de gaucher, comprendiendo el método ajustar la dosis terapéutica individual al metabolismo P-450 del paciente Download PDFInfo
- Publication number
- ES2754398T3 ES2754398T3 ES16175117T ES16175117T ES2754398T3 ES 2754398 T3 ES2754398 T3 ES 2754398T3 ES 16175117 T ES16175117 T ES 16175117T ES 16175117 T ES16175117 T ES 16175117T ES 2754398 T3 ES2754398 T3 ES 2754398T3
- Authority
- ES
- Spain
- Prior art keywords
- subject
- formula
- hemitartrate
- compound
- metabolizer
- Prior art date
- Legal status (The legal status 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 status listed.)
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- FJZZPCZKBUKGGU-AUSIDOKSSA-N CCCCCCCC(N[C@H](CN1CCCC1)[C@@H](c(cc1)cc2c1OCCO2)O)=O Chemical compound CCCCCCCC(N[C@H](CN1CCCC1)[C@@H](c(cc1)cc2c1OCCO2)O)=O FJZZPCZKBUKGGU-AUSIDOKSSA-N 0.000 description 1
- QGWOLQNFECNEFO-HYBUGGRVSA-N CCCCCCCC(N[C@H](CN1CCCC1)[C@@H](c(cc1OCCC)ccc1O)O)=O Chemical compound CCCCCCCC(N[C@H](CN1CCCC1)[C@@H](c(cc1OCCC)ccc1O)O)=O QGWOLQNFECNEFO-HYBUGGRVSA-N 0.000 description 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
- A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
- A61K31/4025—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil not condensed and containing further heterocyclic rings, e.g. cromakalim
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
- C07D405/02—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
- C07D405/06—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a carbon chain containing only aliphatic carbon atoms
-
- 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
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/357—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having two or more oxygen atoms in the same ring, e.g. crown ethers, guanadrel
-
- 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
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C59/00—Compounds having carboxyl groups bound to acyclic carbon atoms and containing any of the groups OH, O—metal, —CHO, keto, ether, groups, groups, or groups
- C07C59/235—Saturated compounds containing more than one carboxyl group
- C07C59/245—Saturated compounds containing more than one carboxyl group containing hydroxy or O-metal groups
- C07C59/255—Tartaric acid
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D319/00—Heterocyclic compounds containing six-membered rings having two oxygen atoms as the only ring hetero atoms
- C07D319/10—1,4-Dioxanes; Hydrogenated 1,4-dioxanes
- C07D319/14—1,4-Dioxanes; Hydrogenated 1,4-dioxanes condensed with carbocyclic rings or ring systems
- C07D319/16—1,4-Dioxanes; Hydrogenated 1,4-dioxanes condensed with carbocyclic rings or ring systems condensed with one six-membered ring
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D319/00—Heterocyclic compounds containing six-membered rings having two oxygen atoms as the only ring hetero atoms
- C07D319/10—1,4-Dioxanes; Hydrogenated 1,4-dioxanes
- C07D319/14—1,4-Dioxanes; Hydrogenated 1,4-dioxanes condensed with carbocyclic rings or ring systems
- C07D319/16—1,4-Dioxanes; Hydrogenated 1,4-dioxanes condensed with carbocyclic rings or ring systems condensed with one six-membered ring
- C07D319/18—Ethylenedioxybenzenes, not substituted on the hetero ring
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07B—GENERAL METHODS OF ORGANIC CHEMISTRY; APPARATUS THEREFOR
- C07B2200/00—Indexing scheme relating to specific properties of organic compounds
- C07B2200/13—Crystalline forms, e.g. polymorphs
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Heterocyclic Compounds That Contain Two Or More Ring Oxygen Atoms (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Steroid Compounds (AREA)
- Saccharide Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Molecular Biology (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US26474809P | 2009-11-27 | 2009-11-27 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2754398T3 true ES2754398T3 (es) | 2020-04-17 |
Family
ID=43431870
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES16175117T Active ES2754398T3 (es) | 2009-11-27 | 2010-11-24 | Eliglustat (Genz 112638) como inhibidor de glucosilceramida-sintasa para uso en un método de tratamiento de la enfermedad de fabry o de gaucher, comprendiendo el método ajustar la dosis terapéutica individual al metabolismo P-450 del paciente |
| ES19182732T Active ES2875382T3 (es) | 2009-11-27 | 2010-11-24 | Composición farmacéutica del inhibidor de glucosilceramida sintasa Eliglustat para el tratamiento de la enfermedad de Gaucher, que comprende ajustar la dosis terapéutica individual al metabolismo de P450 del paciente |
| ES14164650.5T Active ES2586947T3 (es) | 2009-11-27 | 2010-11-24 | Genz 112638 para tratar la enfermedad de Gaucher o de Fabry en terapia de combinación |
| ES10785289T Active ES2493940T5 (es) | 2009-11-27 | 2010-11-24 | Una forma amorfa y una forma cristalina de hemitartrato de genz 112638 como inhibidor de glucosilceramida sintasa |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES19182732T Active ES2875382T3 (es) | 2009-11-27 | 2010-11-24 | Composición farmacéutica del inhibidor de glucosilceramida sintasa Eliglustat para el tratamiento de la enfermedad de Gaucher, que comprende ajustar la dosis terapéutica individual al metabolismo de P450 del paciente |
| ES14164650.5T Active ES2586947T3 (es) | 2009-11-27 | 2010-11-24 | Genz 112638 para tratar la enfermedad de Gaucher o de Fabry en terapia de combinación |
| ES10785289T Active ES2493940T5 (es) | 2009-11-27 | 2010-11-24 | Una forma amorfa y una forma cristalina de hemitartrato de genz 112638 como inhibidor de glucosilceramida sintasa |
Country Status (40)
Families Citing this family (28)
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| WO2006007560A2 (en) | 2004-07-01 | 2006-01-19 | University Of Pennsylvania | Targeted protein replacement for the treatment of lysosomal storage disorders |
| NZ625712A (en) | 2009-11-27 | 2016-02-26 | Genzyme Corp | An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase |
| EP2721151B1 (en) * | 2011-06-20 | 2017-12-06 | Mount Sinai School Of Medicine | Anti-tnf-therapy for the mucopolysaccharidoses and other lysosomal disorders |
| SI2753346T1 (sl) | 2011-09-07 | 2020-09-30 | Mount Sinai School Of Medicine | Ceramidaza in diferenciacija celic |
| WO2013078413A1 (en) * | 2011-11-22 | 2013-05-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Modulators of lipid storage |
| HUE051020T2 (hu) | 2012-06-01 | 2021-01-28 | Icahn School Med Mount Sinai | A ceramid szintjei a fertõzések kezelésében és megelõzésében |
| WO2014160390A1 (en) | 2013-03-14 | 2014-10-02 | Icahn School Of Medicine At Mount Sinai | Therapeutic acid ceramidase compositions and methods of making and using them |
| US10227323B2 (en) * | 2013-09-20 | 2019-03-12 | Biomarin Pharmaceutical Inc. | Glucosylceramide synthase inhibitors for the treatment of diseases |
| EP3164128A4 (en) * | 2014-07-03 | 2018-02-28 | Dr. Reddy's Laboratories Ltd. | Amorphous form of eliglustat hemitartarate |
| WO2016166170A1 (en) | 2015-04-14 | 2016-10-20 | Sandoz Ag | Crystalline eliglustat hydrochloride |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| US11116755B2 (en) | 2015-11-18 | 2021-09-14 | Genzyme Corporation | Biomarker of polycystic kidney disease and uses thereof |
| CA3014572C (en) | 2016-02-25 | 2023-10-03 | Asceneuron S.A. | Acid addition salts of piperazine derivatives |
| CN107445938B (zh) * | 2016-05-31 | 2020-04-03 | 北京启慧生物医药有限公司 | 依利格鲁司他半酒石酸盐的结晶形式、制备方法和含有所述结晶形式的药用组合物 |
| CN106349210A (zh) * | 2016-08-24 | 2017-01-25 | 北京阳光诺和药物研究有限公司 | 一种制备酒石酸艾力骨司坦的方法 |
| EP3318277A1 (en) | 2016-11-04 | 2018-05-09 | Institut du Cerveau et de la Moelle Epiniere-ICM | Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases |
| WO2018193090A2 (en) | 2017-04-21 | 2018-10-25 | Amneal Pharmaceuticals Company Gmbh | Process for preparation of eliglustat hemitartrate and intermediates thereof |
| WO2018225085A1 (en) | 2017-06-05 | 2018-12-13 | Cipla Limited | Stable solid dispersions of eliglustat hemitartrate |
| TW201904577A (zh) | 2017-06-16 | 2019-02-01 | 美商貝達醫藥公司 | N-(2-(2-二甲胺基)乙氧基)-4-甲氧基-5-((4-(1-甲基-1h-吲哚-3-基)嘧啶-2-基)胺基)苯基)丙烯醯胺及其鹽之醫藥調配物 |
| EP3664798B1 (en) * | 2017-08-08 | 2025-10-29 | Amneal Pharmaceuticals LLC | Pharmaceutical composition comprising eliglustat |
| US12472144B2 (en) * | 2017-08-08 | 2025-11-18 | Amneal Pharmaceuticals Llc | Pharmaceutical composition comprising eliglustat |
| MX2020006272A (es) | 2017-12-15 | 2020-09-14 | Genzyme Corp | Metodos para tratar la enfermedad de gaucher. |
| WO2019123476A1 (en) * | 2017-12-20 | 2019-06-27 | Sarudbhava Formulations Private Limited | Stable amorphous eliglustat premix and process for the preparation thereof |
| US11760741B2 (en) | 2018-05-02 | 2023-09-19 | Kashiv Biosciences, Llc | Pro-drugs of eliglustat |
| CN112512558A (zh) * | 2018-05-27 | 2021-03-16 | 比奥阿赛斯技术有限公司 | 戈谢病的治疗 |
| CN110878079A (zh) * | 2018-12-31 | 2020-03-13 | 北京启慧生物医药有限公司 | 一种高纯度依利格鲁司他的制备方法 |
| KR20240035456A (ko) * | 2021-07-05 | 2024-03-15 | 페레르 인터내쇼날 에스.에이. | 글리코시다제 저해제를 포함하는 의약 |
| CN116120274A (zh) * | 2021-11-12 | 2023-05-16 | 曙方(上海)医药科技有限公司 | 依利格鲁司他可药用盐及其晶型 |
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