MX2023007551A - Arimoclomol para el tratamiento de la enfermedad de niemann pick, tipo c, en pacientes con mutaciones de sentido erroneo de tipo reticulo endoplasmatico (er). - Google Patents
Arimoclomol para el tratamiento de la enfermedad de niemann pick, tipo c, en pacientes con mutaciones de sentido erroneo de tipo reticulo endoplasmatico (er).Info
- Publication number
- MX2023007551A MX2023007551A MX2023007551A MX2023007551A MX2023007551A MX 2023007551 A MX2023007551 A MX 2023007551A MX 2023007551 A MX2023007551 A MX 2023007551A MX 2023007551 A MX2023007551 A MX 2023007551A MX 2023007551 A MX2023007551 A MX 2023007551A
- Authority
- MX
- Mexico
- Prior art keywords
- type
- arimoclomol
- pick disease
- patients
- treatment
- Prior art date
Links
- 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 abstract 2
- 208000014060 Niemann-Pick disease Diseases 0.000 title abstract 2
- 229950011582 arimoclomol Drugs 0.000 title abstract 2
- 230000035772 mutation Effects 0.000 title abstract 2
- 210000002472 endoplasmic reticulum Anatomy 0.000 abstract 2
- 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 abstract 1
- 239000002253 acid Substances 0.000 abstract 1
- 239000008186 active pharmaceutical agent Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 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
- A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
- A61K31/444—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring heteroatom, e.g. amrinone
-
- 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
- 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
- A61K31/4412—Non condensed pyridines; Hydrogenated derivatives thereof having oxo groups directly attached to the heterocyclic ring
-
- 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
- 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
- A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
-
- 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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Analytical Chemistry (AREA)
- Physiology (AREA)
- Pathology (AREA)
- Biophysics (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- Nutrition Science (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063130438P | 2020-12-24 | 2020-12-24 | |
| EP21155579 | 2021-02-05 | ||
| US202163185111P | 2021-05-06 | 2021-05-06 | |
| PCT/EP2021/087483 WO2022136640A1 (en) | 2020-12-24 | 2021-12-23 | Arimoclomol for the treatment of niemann pick disease, type c, in patients with er type missense mutations |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023007551A true MX2023007551A (es) | 2023-09-04 |
Family
ID=79830856
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023007551A MX2023007551A (es) | 2020-12-24 | 2021-12-23 | Arimoclomol para el tratamiento de la enfermedad de niemann pick, tipo c, en pacientes con mutaciones de sentido erroneo de tipo reticulo endoplasmatico (er). |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20240115558A1 (https=) |
| EP (1) | EP4267139A1 (https=) |
| JP (1) | JP2024502304A (https=) |
| KR (1) | KR20230128037A (https=) |
| AU (1) | AU2021405780A1 (https=) |
| CA (1) | CA3205873A1 (https=) |
| IL (1) | IL303968A (https=) |
| MX (1) | MX2023007551A (https=) |
| WO (1) | WO2022136640A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2023366474A1 (en) * | 2022-10-27 | 2025-05-15 | Zevra Denmark A/S | Arimoclomol compositions for use in treating niemann pick disease type c (npc) |
| WO2026010894A1 (en) * | 2024-06-30 | 2026-01-08 | Zevra Therapeutics, Inc. | Arimoclomol in combination with miglustat for use in the treatment of lysosomal storage disorders |
| US20260000661A1 (en) * | 2024-07-01 | 2026-01-01 | Zevra Therapeutics, Inc. | Use of arimoclomol in activating clear gene expression as treatment for lysosomal storage disorders |
| WO2026010893A1 (en) * | 2024-07-01 | 2026-01-08 | Zevra Therapeutics, Inc. | Arimoclomol in combination with miglustat for use in the treatment of lysosomal storage disorders such as niemann-pick disease, type-c |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HU222994B1 (hu) | 1995-11-02 | 2004-01-28 | BIOREX Kutató és Fejlesztő Rt. | Hidroxilaminszármazékok és azok alkalmazása sejtek molekuláris chaperon-termelésének fokozására alkalmas gyógyszerkészítmények előállítására |
| HUP9900475D0 (en) | 1999-02-26 | 1999-04-28 | Biorex Kutato Fejlesztoe Kft | O-(3-piperidino-2-hydroxy-1-propyl)-hiyroximic acid-halogenid derivative, it's use for treating insulin resistance, and pharmaceutical compositions containing them as active component |
| HUP0001583A2 (hu) | 2000-04-18 | 2002-11-28 | BIOREX Kutató és Fejlesztő Rt. | Egy piridin-1-oxid-származék és eljárás annak átalakítására gyógyászati hatású vegyületekké |
| RU2013125923A (ru) * | 2010-11-30 | 2015-01-10 | Орфазиме Апс | СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70 |
-
2021
- 2021-12-23 WO PCT/EP2021/087483 patent/WO2022136640A1/en not_active Ceased
- 2021-12-23 CA CA3205873A patent/CA3205873A1/en active Pending
- 2021-12-23 MX MX2023007551A patent/MX2023007551A/es unknown
- 2021-12-23 EP EP21847473.2A patent/EP4267139A1/en active Pending
- 2021-12-23 US US18/267,591 patent/US20240115558A1/en active Pending
- 2021-12-23 JP JP2023539080A patent/JP2024502304A/ja active Pending
- 2021-12-23 KR KR1020237024972A patent/KR20230128037A/ko active Pending
- 2021-12-23 AU AU2021405780A patent/AU2021405780A1/en active Pending
- 2021-12-23 IL IL303968A patent/IL303968A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230128037A (ko) | 2023-09-01 |
| IL303968A (en) | 2023-08-01 |
| WO2022136640A1 (en) | 2022-06-30 |
| US20240115558A1 (en) | 2024-04-11 |
| JP2024502304A (ja) | 2024-01-18 |
| CA3205873A1 (en) | 2022-06-30 |
| EP4267139A1 (en) | 2023-11-01 |
| AU2021405780A1 (en) | 2023-07-06 |
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