MX395523B - Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert. - Google Patents

Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Info

Publication number
MX395523B
MX395523B MX2018007669A MX2018007669A MX395523B MX 395523 B MX395523 B MX 395523B MX 2018007669 A MX2018007669 A MX 2018007669A MX 2018007669 A MX2018007669 A MX 2018007669A MX 395523 B MX395523 B MX 395523B
Authority
MX
Mexico
Prior art keywords
ert
treatment
untreated
fabry disease
previously treated
Prior art date
Application number
MX2018007669A
Other languages
English (en)
Other versions
MX2018007669A (es
Inventor
Jeff Castelli
Original Assignee
Amicus Therapeutics Inc
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
Priority claimed from US15/213,920 external-priority patent/US9999618B2/en
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of MX2018007669A publication Critical patent/MX2018007669A/es
Publication of MX395523B publication Critical patent/MX395523B/es

Links

Classifications

    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • A61P39/00—General protective or antinoxious agents
    • A61P39/02—Antidotes
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Toxicology (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)

Abstract

Se proveen regímenes de dosificación para el tratamiento de la enfermedad de Fabry en un paciente. Algunos métodos se relacionan con el tratamiento de pacientes con Fabry no tratados previamente o tratados con ERT. Algunos métodos comprenden administrar al paciente aproximadamente 123 mg de equivalentes de base libre de migalastat para mejorar la masa ventricular izquierda y/o para mejorar la globotriaosilceramida en podocitos.
MX2018007669A 2016-07-19 2017-07-19 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert. MX395523B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US15/213,920 US9999618B2 (en) 2007-04-26 2016-07-19 Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
PCT/US2017/042872 WO2018017721A1 (en) 2016-07-19 2017-07-19 Treatment of fabry disease in ert-naïve and ert-experienced patients

Publications (2)

Publication Number Publication Date
MX2018007669A MX2018007669A (es) 2018-09-12
MX395523B true MX395523B (es) 2025-03-25

Family

ID=59416847

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018007669A MX395523B (es) 2016-07-19 2017-07-19 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.
MX2022011334A MX2022011334A (es) 2016-07-19 2018-06-20 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2022011334A MX2022011334A (es) 2016-07-19 2018-06-20 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Country Status (14)

Country Link
US (2) US12070453B2 (es)
EP (5) EP4389213A3 (es)
JP (4) JP6752270B2 (es)
KR (3) KR20230061563A (es)
CN (1) CN109661230A (es)
AR (1) AR109103A1 (es)
AU (3) AU2017298376A1 (es)
CA (1) CA3031249A1 (es)
CL (1) CL2018001677A1 (es)
CO (1) CO2018006358A2 (es)
IL (2) IL313907A (es)
MX (2) MX395523B (es)
TW (2) TWI875676B (es)
WO (1) WO2018017721A1 (es)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2533050B3 (en) 2006-05-16 2015-06-24 Amicus Therapeutics, Inc. Treatment Options For Fabry Disease
SI2252313T1 (sl) 2008-02-12 2015-08-31 Amicus Therapeutics, Inc. Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
CA3031249A1 (en) * 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
US12020820B1 (en) * 2017-03-03 2024-06-25 Cerner Innovation, Inc. Predicting sphingolipidoses (fabry's disease) and decision support
KR102631805B1 (ko) 2017-05-30 2024-01-31 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
EP3630114B1 (en) 2017-05-30 2023-11-01 Amicus Therapeutics, Inc. Migalastat for treating fabry patients having renal impairment
JP7555818B2 (ja) * 2018-02-06 2024-09-25 アミカス セラピューティックス インコーポレイテッド 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用
HUE064761T2 (hu) * 2018-02-06 2024-04-28 Amicus Therapeutics Inc Klasszikus FABRY-betegségben szenvedõ betegek kezelése migalasztáttal
US20210315875A1 (en) * 2018-08-20 2021-10-14 Amicus Therapeutics, Inc. Methods of Treating Fabry Disease in Patients Having a Mutation in the GLA Gene
TW202042812A (zh) * 2019-01-22 2020-12-01 美商阿米庫斯醫療股份有限公司 減少法布瑞氏症患者中之腦血管事件之方法
US20220313670A1 (en) * 2019-06-11 2022-10-06 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease In Patients Having Renal Impairment
HRP20260109T1 (hr) 2019-08-07 2026-03-13 Amicus Therapeutics, Inc. Migalastat za upotrebu u liječenju fabryjeve bolesti kod pacijenata koji imaju mutaciju u gla genu
US20210251972A1 (en) * 2020-02-10 2021-08-19 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
KR102471225B1 (ko) 2021-11-18 2022-11-28 박기용 어플리케이션을 통한 소비자 및 농장 연결 시스템

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0463109A4 (en) 1989-03-24 1992-11-19 Research Corporation Technologies, Inc. Recombinant alpha-galactosidase, a therapy for fabry disease
WO1992000277A1 (en) 1990-06-29 1992-01-09 Nippon Shinyaku Co., Ltd. Piperidine derivative
US6270954B1 (en) 1996-04-10 2001-08-07 The Regents Of The University Of California Correction of genetic defects using chemical chaperones
EP0959877A4 (en) 1996-04-10 2000-08-23 Univ California CORRECTION OF GENETIC ERRORS USING CHEMICAL COMPANIES
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
CN102586205A (zh) 2003-01-31 2012-07-18 纽约大学西奈山医学院 酶和酶的活性部位特异性陪伴分子的组合
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
BRPI0416216A (pt) 2003-11-12 2006-12-26 Amicus Therapeutics Inc composto de glicoimidazol e polihidroxiciclohexenila e método para sintetizar composto de glicoimidazol e polihidroxiciclohexenila, métodos para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir uma -glicosidade, e para tratar doença de gaucher, derivado de glicoimidazol e derivados de polihidroxiciclohexilamina
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
WO2006133446A2 (en) 2005-06-08 2006-12-14 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes
US7955262B2 (en) 2005-07-26 2011-06-07 Syneron Medical Ltd. Method and apparatus for treatment of skin using RF and ultrasound energies
AU2006272497B2 (en) 2005-07-27 2012-07-19 University Of Florida Research Foundation, Inc. Small compounds that correct protein misfolding and uses thereof
AU2015230773B2 (en) * 2006-05-16 2017-09-28 Amicus Therapeutics Inc. Assays for diagnosing and evaluating treatment options for fabry disease
EP2533050B3 (en) * 2006-05-16 2015-06-24 Amicus Therapeutics, Inc. Treatment Options For Fabry Disease
DE602007012924D1 (de) 2006-09-22 2011-04-14 Krka Tovarna Zdravil D D Aripiprazolhemifumarat und verfahren zu dessen herstellung
CA2682441A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
SI2252313T1 (sl) 2008-02-12 2015-08-31 Amicus Therapeutics, Inc. Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
CN107088225A (zh) * 2011-03-11 2017-08-25 阿米库斯治疗学公司 治疗法布里病的给药方案
JP2013255488A (ja) * 2012-05-18 2013-12-26 Saitama Medical Univ 拡張期心不全非ヒトモデル動物及びその製造方法、並びに、拡張期心不全予防乃至症状改善活性を有する物質のスクリーニング方法
CA3031249A1 (en) * 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients

Also Published As

Publication number Publication date
CO2018006358A2 (es) 2018-11-30
EP4378524A2 (en) 2024-06-05
KR20190030729A (ko) 2019-03-22
IL264301A (en) 2019-02-28
US20190183869A1 (en) 2019-06-20
CN109661230A (zh) 2019-04-19
EP4324521A3 (en) 2024-04-24
EP4389213A2 (en) 2024-06-26
US20230321065A1 (en) 2023-10-12
TWI875676B (zh) 2025-03-11
AU2025217396A1 (en) 2025-09-04
AR109103A1 (es) 2018-10-31
CA3031249A1 (en) 2018-01-25
NZ750752A (en) 2025-07-25
EP4389213A3 (en) 2024-08-21
JP2018526368A (ja) 2018-09-13
EP4356911A1 (en) 2024-04-24
AU2017298376A1 (en) 2019-03-07
AU2023206175B2 (en) 2025-05-22
EP4378524A3 (en) 2024-08-14
JP2019178140A (ja) 2019-10-17
EP3487502A1 (en) 2019-05-29
WO2018017721A1 (en) 2018-01-25
AU2023206175A1 (en) 2023-08-10
CL2018001677A1 (es) 2019-02-01
TW201806597A (zh) 2018-03-01
JP6752270B2 (ja) 2020-09-09
NZ790449A (en) 2025-08-29
EP4324521A2 (en) 2024-02-21
MX2018007669A (es) 2018-09-12
KR20240017126A (ko) 2024-02-06
US12070453B2 (en) 2024-08-27
TW202408513A (zh) 2024-03-01
IL313907A (en) 2024-08-01
MX2022011334A (es) 2022-10-07
JP2024026079A (ja) 2024-02-28
JP2022033749A (ja) 2022-03-02
KR20230061563A (ko) 2023-05-08

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