BR112021024886A2 - Métodos de tratar a doença de fabry em pacientes com insuficiência renal - Google Patents

Métodos de tratar a doença de fabry em pacientes com insuficiência renal

Info

Publication number
BR112021024886A2
BR112021024886A2 BR112021024886A BR112021024886A BR112021024886A2 BR 112021024886 A2 BR112021024886 A2 BR 112021024886A2 BR 112021024886 A BR112021024886 A BR 112021024886A BR 112021024886 A BR112021024886 A BR 112021024886A BR 112021024886 A2 BR112021024886 A2 BR 112021024886A2
Authority
BR
Brazil
Prior art keywords
disease
methods
renal failure
patients
treating fabry
Prior art date
Application number
BR112021024886A
Other languages
English (en)
Inventor
Franklin Johnson
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
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of BR112021024886A2 publication Critical patent/BR112021024886A2/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
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-galactosidase (3.2.1.22)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)

Abstract

métodos de tratar a doença de fabry em pacientes com insuficiência renal. são fornecidos métodos para o tratamento da doença de fabry em um paciente com insuficiência renal. certos métodos compreendem administrar ao paciente cerca de 100 mg a cerca de 300 mg equivalentes de base livre de migalastate ou sal deste a uma frequência superior a uma vez a cada dois dias, tal como uma vez a cada quatro ou sete dias. certos métodos compreendem medir lyso-gb3 e/ou migalastate em uma ou mais amostras de plasma do paciente.
BR112021024886A 2019-06-11 2020-06-11 Métodos de tratar a doença de fabry em pacientes com insuficiência renal BR112021024886A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962859904P 2019-06-11 2019-06-11
PCT/US2020/037174 WO2020252129A1 (en) 2019-06-11 2020-06-11 Methods of treating fabry disease in patients having renal impairment

Publications (1)

Publication Number Publication Date
BR112021024886A2 true BR112021024886A2 (pt) 2022-01-25

Family

ID=71950725

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021024886A BR112021024886A2 (pt) 2019-06-11 2020-06-11 Métodos de tratar a doença de fabry em pacientes com insuficiência renal

Country Status (15)

Country Link
US (1) US20220313670A1 (pt)
EP (1) EP3982962A1 (pt)
JP (1) JP2022536687A (pt)
KR (1) KR20220019796A (pt)
CN (1) CN114423427A (pt)
AR (1) AR120055A1 (pt)
AU (1) AU2020291002A1 (pt)
BR (1) BR112021024886A2 (pt)
CA (1) CA3141226A1 (pt)
CL (1) CL2021003280A1 (pt)
EA (1) EA202290024A1 (pt)
IL (1) IL288677A (pt)
MX (1) MX2021015352A (pt)
TW (1) TW202112372A (pt)
WO (1) WO2020252129A1 (pt)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2007253900A1 (en) 2006-05-16 2007-11-29 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
AU2009214648B2 (en) 2008-02-12 2014-11-13 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of 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
EP3895708A3 (en) 2019-08-07 2022-01-19 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US20100113517A1 (en) 2007-03-30 2010-05-06 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
EP2150254A4 (en) * 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
AU2009214648B2 (en) 2008-02-12 2014-11-13 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
KR20140011367A (ko) * 2011-03-11 2014-01-28 아미쿠스 세라퓨틱스, 인코포레이티드 파브리 병을 치료하기 위한 투여 요법
CA3031249A1 (en) * 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naive and ert-experienced patients
MX2019008076A (es) * 2017-01-05 2019-08-29 Protalix Ltd Regimen terapeutico para el tratamiento de fabry usando alfa-galactosidasa estabilizada.
AR111971A1 (es) * 2017-05-30 2019-09-04 Amicus Therapeutics Inc Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal
AU2018277756A1 (en) * 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment

Also Published As

Publication number Publication date
CN114423427A (zh) 2022-04-29
IL288677A (en) 2022-02-01
CA3141226A1 (en) 2020-12-17
EP3982962A1 (en) 2022-04-20
KR20220019796A (ko) 2022-02-17
AU2020291002A1 (en) 2022-01-06
MX2021015352A (es) 2022-04-06
EA202290024A1 (ru) 2022-03-14
AR120055A1 (es) 2022-02-02
US20220313670A1 (en) 2022-10-06
TW202112372A (zh) 2021-04-01
JP2022536687A (ja) 2022-08-18
CL2021003280A1 (es) 2022-10-07
WO2020252129A1 (en) 2020-12-17

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