KR20230128037A - Er형 미스센스 돌연변이를 갖는 환자에서, c형 니만픽병의 치료를 위한 아리모클로몰 - Google Patents

Er형 미스센스 돌연변이를 갖는 환자에서, c형 니만픽병의 치료를 위한 아리모클로몰 Download PDF

Info

Publication number
KR20230128037A
KR20230128037A KR1020237024972A KR20237024972A KR20230128037A KR 20230128037 A KR20230128037 A KR 20230128037A KR 1020237024972 A KR1020237024972 A KR 1020237024972A KR 20237024972 A KR20237024972 A KR 20237024972A KR 20230128037 A KR20230128037 A KR 20230128037A
Authority
KR
South Korea
Prior art keywords
pharmaceutical ingredient
active pharmaceutical
day
administered
subject
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.)
Pending
Application number
KR1020237024972A
Other languages
English (en)
Korean (ko)
Inventor
토마스 커크가드 젠슨
달리 크리스틴 아이
니콜라이 하브소이 토르프 피터슨
토마스 블래틀러
라에르케 클레멘트 프레이버그
Original Assignee
제브라 덴마크 에이/에스
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by 제브라 덴마크 에이/에스 filed Critical 제브라 덴마크 에이/에스
Publication of KR20230128037A publication Critical patent/KR20230128037A/ko
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4427Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
    • A61K31/444Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring heteroatom, e.g. amrinone
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4412Non condensed pyridines; Hydrogenated derivatives thereof having oxo groups directly attached to the heterocyclic ring
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/45Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/4523Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545Non 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/106Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic 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)
KR1020237024972A 2020-12-24 2021-12-23 Er형 미스센스 돌연변이를 갖는 환자에서, c형 니만픽병의 치료를 위한 아리모클로몰 Pending KR20230128037A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063130438P 2020-12-24 2020-12-24
US63/130,438 2020-12-24
EP21155579 2021-02-05
EP21155579.2 2021-02-05
US202163185111P 2021-05-06 2021-05-06
US63/185,111 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
KR20230128037A true KR20230128037A (ko) 2023-09-01

Family

ID=79830856

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237024972A Pending KR20230128037A (ko) 2020-12-24 2021-12-23 Er형 미스센스 돌연변이를 갖는 환자에서, c형 니만픽병의 치료를 위한 아리모클로몰

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

Also Published As

Publication number Publication date
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
MX2023007551A (es) 2023-09-04
EP4267139A1 (en) 2023-11-01
AU2021405780A1 (en) 2023-07-06

Similar Documents

Publication Publication Date Title
AU2023206175B2 (en) Treatment of fabry disease in ert-naïve and ert-experienced patients
KR20230128037A (ko) Er형 미스센스 돌연변이를 갖는 환자에서, c형 니만픽병의 치료를 위한 아리모클로몰
IL303969A (en) ugh
US20210401776A1 (en) Method of treating refractory epilepsy syndromes using fenfluramine enantiomers
WO2019017938A1 (en) TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES
US20030087799A1 (en) Modulation
US20160166541A1 (en) Methods For Identifying Analgesic Agents
WO2009045900A2 (en) Methods for treating or preventing diseases associated with low bone mass
US20030096831A1 (en) Ocular growth and nicotinic antagonists
US20220211687A1 (en) Use of arimoclomol in the treatment of niemann pick disease
JP2022518733A (ja) ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用
US20240009180A1 (en) Medicine for alleviating neuropathic pain
AU775516B2 (en) Ocular growth and nicotinic antagonists
CN116916918A (zh) 阿瑞洛莫用于治疗具有er型错义突变的患者的c型尼曼皮克病
KR20220092026A (ko) 니만 픽병의 치료에서의 아리모클로몰의 용도
AU2020294309A1 (en) Use of arimoclomol in the treatment of niemann pick disease
JP7838764B2 (ja) 新型コロナウイルス感染症後遺症の治療薬
Seznec et al. 2003 International Friedreich's Ataxia Research Conference, 14–16 February 2003, Bethesda, MD, USA
HK40094114A (en) Medicine for alleviating neuropathic pain
HK40110848A (en) Treatment of fabry disease in ert-naive and ert-experienced patients
HK40106068A (en) Treatment of fabry disease in ert-naive and ert-experienced patients
HK40112175A (en) Treatment of fabry disease in ert-naive and ert-experienced patients
AU2015271605B2 (en) Treatment of familial exudative vitreoretinopathy through S1PR2 inhibition

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20230720

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20241219

Comment text: Request for Examination of Application