PL2533050T6 - Możliwości leczenia choroby Fabry'ego - Google Patents

Możliwości leczenia choroby Fabry'ego

Info

Publication number
PL2533050T6
PL2533050T6 PL12168451T PL12168451T PL2533050T6 PL 2533050 T6 PL2533050 T6 PL 2533050T6 PL 12168451 T PL12168451 T PL 12168451T PL 12168451 T PL12168451 T PL 12168451T PL 2533050 T6 PL2533050 T6 PL 2533050T6
Authority
PL
Poland
Prior art keywords
treatment options
fabry disease
fabry
disease
options
Prior art date
Application number
PL12168451T
Other languages
English (en)
Other versions
PL2533050T3 (pl
Inventor
David Lockhart
Jeff Castelli
Original Assignee
Amicus Therapeutics Inc
Nat Institutes Of 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=PL2533050(T6) "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 Institutes Of Health filed Critical Amicus Therapeutics Inc
Publication of PL2533050T3 publication Critical patent/PL2533050T3/pl
Publication of PL2533050T6 publication Critical patent/PL2533050T6/pl

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
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic 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
    • C07D211/36—Heterocyclic 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
    • C07D211/40—Oxygen atoms
    • C07D211/44—Oxygen atoms attached in position 4
    • C07D211/46—Oxygen atoms attached in position 4 having a hydrogen atom as the second substituent in position 4
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5094—Chemical 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
    • G01N2333/94—Hydrolases (3) acting on glycosyl compounds (3.2) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/04—Endocrine or metabolic disorders
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Cell Biology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Ecology (AREA)
  • Pathology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • General Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • Food Science & Technology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Obesity (AREA)
  • Vascular Medicine (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL12168451T 2006-05-16 2007-05-16 Możliwości leczenia choroby Fabry'ego PL2533050T6 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US80108906P 2006-05-16 2006-05-16
US85363106P 2006-10-23 2006-10-23
EP07797506.8A EP2024745B1 (en) 2006-05-16 2007-05-16 Assays for diagnosing and evaluating treatment options for fabry disease
EP12168451.8A EP2533050B3 (en) 2006-05-16 2007-05-16 Treatment Options For Fabry Disease

Publications (2)

Publication Number Publication Date
PL2533050T3 PL2533050T3 (pl) 2014-08-29
PL2533050T6 true PL2533050T6 (pl) 2016-05-31

Family

ID=38723987

Family Applications (2)

Application Number Title Priority Date Filing Date
PL12168451T PL2533050T6 (pl) 2006-05-16 2007-05-16 Możliwości leczenia choroby Fabry'ego
PL14168748T PL2787345T3 (pl) 2006-05-16 2007-05-16 Możliwości leczenia choroby Fabry’ego

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL14168748T PL2787345T3 (pl) 2006-05-16 2007-05-16 Możliwości leczenia choroby Fabry’ego

Country Status (18)

Country Link
US (9) US7851143B2 (pl)
EP (5) EP3457135A1 (pl)
JP (6) JP2009537149A (pl)
AU (1) AU2007253900A1 (pl)
CA (1) CA2652553C (pl)
CY (3) CY1115180T1 (pl)
DK (2) DK2787345T3 (pl)
ES (3) ES2464527T7 (pl)
HK (3) HK1178975A1 (pl)
HU (2) HUE027569T2 (pl)
LT (1) LTC2787345I2 (pl)
LU (1) LU93312I2 (pl)
MX (2) MX351004B (pl)
NL (1) NL300843I2 (pl)
PL (2) PL2533050T6 (pl)
PT (1) PT2533050E (pl)
SI (2) SI2533050T1 (pl)
WO (1) WO2007137072A2 (pl)

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EP3457135A1 (en) 2006-05-16 2019-03-20 Amicus Therapeutics, Inc. Treatment options for fabry disease
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WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
PL3470077T3 (pl) * 2008-02-12 2021-04-06 Amicus Therapeutics, Inc. Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi
JP2011517556A (ja) * 2008-03-12 2011-06-16 アミカス セラピューティックス インコーポレイテッド ポンペ病のための治療選択肢を診断し評価するための検定
JP5739898B2 (ja) * 2009-11-17 2015-06-24 ベイラー リサーチ インスティテュートＢａｙｌｏｒ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ 心疾患マーカーとしての尿中トリアオシルセラミド（ｇｂ３）
AU2011223706A1 (en) * 2010-03-05 2012-09-20 Baylor Research Institute Involvement of androgen/androgen receptor pathway in Fabry disease
CN103974619B (zh) * 2011-03-11 2017-02-15 阿米库斯治疗学公司 治疗法布里病的给药方案
DK2707101T3 (da) 2011-05-12 2019-05-13 Proteostasis Therapeutics Inc Proteostaseregulatorer
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US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
CN107074971B (zh) * 2014-07-25 2021-11-02 夏尔人类遗传性治疗公司 人4-磷酸衔接蛋白2糖脂转移蛋白样域的晶体结构
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EP4324522A3 (en) * 2017-05-30 2024-05-22 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
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US20210038579A1 (en) * 2018-02-06 2021-02-11 Amicus Therapeutics, Inc. Treatment Of Patients With Classic Fabry Disease
EP3749307A1 (en) 2018-02-06 2020-12-16 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
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US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
PL3470077T3 (pl) 2008-02-12 2021-04-06 Amicus Therapeutics, Inc. Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi
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JP2020503900A (ja) 2017-01-10 2020-02-06 アミカス セラピューティックス インコーポレイテッド ファブリー病の処置のための組換えアルファ−ガラクトシダーゼａ
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SG11202000423XA (en) 2017-07-19 2020-02-27 Amicus Therapeutics Inc Treatment of fabry disease in ert-naïve and ert-experienced patients
EP4176877A1 (en) 2017-08-28 2023-05-10 Amicus Therapeutics, Inc. Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease
EP3749307A1 (en) 2018-02-06 2020-12-16 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
US20210038579A1 (en) 2018-02-06 2021-02-11 Amicus Therapeutics, Inc. Treatment Of Patients With Classic Fabry Disease
CA3110065A1 (en) 2018-08-20 2020-02-27 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
WO2020252129A1 (en) 2019-06-11 2020-12-17 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having renal impairment
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin Methods of treating fabry disease in patients having a mutation in the gla gene

Also Published As

Publication number Publication date
US10406143B2 (en) 2019-09-10
JP2009537149A (ja) 2009-10-29
EP2024745A4 (en) 2010-03-17
LTPA2016033I1 (lt) 2016-12-12
CY2016040I2 (el) 2017-06-28
US9987263B2 (en) 2018-06-05
JP2023027053A (ja) 2023-03-01
CA2652553A1 (en) 2007-11-29
EP2024745A2 (en) 2009-02-18
US20080056994A1 (en) 2008-03-06
PL2533050T3 (pl) 2014-08-29
EP3457135A1 (en) 2019-03-20
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US20220160690A1 (en) 2022-05-26
US11241422B2 (en) 2022-02-08
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