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PT2533050E - Opções de tratamento para a doença de fabry - Google Patents

Opções de tratamento para a doença de fabry Download PDF

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Publication number
PT2533050E
PT2533050E PT121684518T PT12168451T PT2533050E PT 2533050 E PT2533050 E PT 2533050E PT 121684518 T PT121684518 T PT 121684518T PT 12168451 T PT12168451 T PT 12168451T PT 2533050 E PT2533050 E PT 2533050E
Authority
PT
Portugal
Prior art keywords
treatment options
fabry disease
fabry
disease
options
Prior art date
Application number
PT121684518T
Other languages
English (en)
Inventor
David Lockhart
Jeff Castelli
Original Assignee
Amicus Therapeutics Inc
Nat Inst Health
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=38723987&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PT2533050(E) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Amicus Therapeutics Inc, Nat Inst Health filed Critical Amicus Therapeutics Inc
Publication of PT2533050E publication Critical patent/PT2533050E/pt

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
    • A61K31/445Non 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
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D211/00Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D211/40Oxygen atoms
    • C07D211/44Oxygen atoms attached in position 4
    • C07D211/46Oxygen atoms attached in position 4 having a hydrogen atom as the second substituent in position 4
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5094Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for blood cell populations
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/924Hydrolases (3) acting on glycosyl compounds (3.2)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/924Hydrolases (3) acting on glycosyl compounds (3.2)
    • G01N2333/94Hydrolases (3) acting on glycosyl compounds (3.2) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/04Endocrine or metabolic disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/52Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Cell Biology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Pathology (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Ecology (AREA)
  • Analytical Chemistry (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Diabetes (AREA)
  • Vascular Medicine (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PT121684518T 2006-05-16 2007-05-16 Opções de tratamento para a doença de fabry PT2533050E (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US80108906P 2006-05-16 2006-05-16
US85363106P 2006-10-23 2006-10-23

Publications (1)

Publication Number Publication Date
PT2533050E true PT2533050E (pt) 2014-05-26

Family

ID=38723987

Family Applications (1)

Application Number Title Priority Date Filing Date
PT121684518T PT2533050E (pt) 2006-05-16 2007-05-16 Opções de tratamento para a doença de fabry

Country Status (18)

Country Link
US (9) US7851143B2 (pt)
EP (5) EP2955520B1 (pt)
JP (6) JP2009537149A (pt)
AU (1) AU2007253900A1 (pt)
CA (1) CA2652553C (pt)
CY (3) CY1115180T1 (pt)
DK (2) DK2533050T6 (pt)
ES (3) ES2464527T7 (pt)
HK (3) HK1178975A1 (pt)
HU (2) HUE027569T2 (pt)
LT (1) LTC2787345I2 (pt)
LU (1) LU93312I2 (pt)
MX (2) MX2008014679A (pt)
NL (1) NL300843I2 (pt)
PL (2) PL2533050T6 (pt)
PT (1) PT2533050E (pt)
SI (2) SI2787345T1 (pt)
WO (1) WO2007137072A2 (pt)

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PT2533050E (pt) 2006-05-16 2014-05-26 Amicus Therapeutics Inc Opções de tratamento para a doença de fabry
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
DK3470077T3 (da) * 2008-02-12 2020-11-30 Amicus Therapeutics Inc Fremgangsmåde til forudsigelse af respons på behandling af sygdomme med farmakologiske chaperoner
MX2010009875A (es) * 2008-03-12 2010-11-26 Amicus Therapeutics Inc Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de pompe.
ES2568610T3 (es) * 2009-11-17 2016-05-03 Baylor Research Institute Triaosilceramida (Gb3) urinaria como un marcador de cardiopatías
CA2791994A1 (en) * 2010-03-05 2011-09-09 Baylor Research Institute Involvement of androgen/androgen receptor pathway in fabry disease
MX2013010446A (es) * 2011-03-11 2014-03-27 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de la enfermedad de fabry.
PT2707101T (pt) 2011-05-12 2019-05-30 Proteostasis Therapeutics Inc Reguladores da proteostase
CA2860085A1 (en) * 2011-12-22 2013-06-27 Centogene Ip Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
AU2013202368B2 (en) 2012-01-25 2016-06-16 Proteostasis Therapeutics, Inc. Proteasome activity modulating compounds
WO2014127038A1 (en) * 2013-02-12 2014-08-21 Baylor Research Institute Methods and compositions related to calcium homeostasis and fabry disease
CN105828811B (zh) * 2013-12-23 2022-04-08 Bcn肽类股份有限公司 用于在溶酶体贮积症或糖原贮积病的治疗中使用的作为胞吐作用活化化合物的比卡鲁胺类似物或(s)-比卡鲁胺
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
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GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
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US11335461B1 (en) 2017-03-06 2022-05-17 Cerner Innovation, Inc. Predicting glycogen storage diseases (Pompe disease) and decision support
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ES2970419T3 (es) * 2017-05-30 2024-05-28 Amicus Therapeutics Inc Migalastat para el tratamiento de pacientes de Fabry que tienen disfunción renal
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CN112585130A (zh) 2018-06-27 2021-03-30 蛋白质平衡治疗股份有限公司 蛋白酶体活性增强化合物
BR112020026580A2 (pt) 2018-06-27 2021-03-23 Proteostasis Therapeutics, Inc. (Fem) Compostos, composição farmacêutica e métodos para tratar um paciente
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Also Published As

Publication number Publication date
US20180250279A1 (en) 2018-09-06
WO2007137072A3 (en) 2008-10-16
JP2013100307A (ja) 2013-05-23
SI2787345T1 (sl) 2016-08-31
EP2024745A4 (en) 2010-03-17
JP6026268B2 (ja) 2016-11-16
ES2464527T9 (es) 2015-07-24
US9987263B2 (en) 2018-06-05
US20180325881A1 (en) 2018-11-15
NL300843I2 (pt) 2016-12-21
DK2533050T3 (da) 2014-06-02
PL2533050T6 (pl) 2016-05-31
JP7175297B2 (ja) 2022-11-18
EP2787345B8 (en) 2016-04-13
EP2955520B1 (en) 2018-10-31
EP3457135A1 (en) 2019-03-20
AU2007253900A1 (en) 2007-11-29
US20080056994A1 (en) 2008-03-06
US7851143B2 (en) 2010-12-14
EP2787345A1 (en) 2014-10-08
HK1129458A1 (en) 2009-11-27
WO2007137072A2 (en) 2007-11-29
DK2533050T6 (da) 2015-09-21
JP2018138564A (ja) 2018-09-06
EP2533050A1 (en) 2012-12-12
JP2023027053A (ja) 2023-03-01
HK1218955A1 (zh) 2017-03-17
CY2016040I1 (el) 2017-06-28
US20150238474A1 (en) 2015-08-27
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US11241422B2 (en) 2022-02-08
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US10406143B2 (en) 2019-09-10
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US20220160690A1 (en) 2022-05-26
ES2709445T3 (es) 2019-04-16
US20190388409A1 (en) 2019-12-26
CA2652553A1 (en) 2007-11-29
LTPA2016033I1 (lt) 2016-12-12
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PL2533050T3 (pl) 2014-08-29
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US20120220632A1 (en) 2012-08-30
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JP2020196717A (ja) 2020-12-10
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US10383864B2 (en) 2019-08-20

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