ZA201306735B - Dosing regimens for the treatment of fabry disease - Google Patents

Dosing regimens for the treatment of fabry disease

Info

Publication number
ZA201306735B
ZA201306735B ZA2013/06735A ZA201306735A ZA201306735B ZA 201306735 B ZA201306735 B ZA 201306735B ZA 2013/06735 A ZA2013/06735 A ZA 2013/06735A ZA 201306735 A ZA201306735 A ZA 201306735A ZA 201306735 B ZA201306735 B ZA 201306735B
Authority
ZA
South Africa
Prior art keywords
treatment
dosing regimens
fabry disease
fabry
disease
Prior art date
Application number
ZA2013/06735A
Other languages
English (en)
Inventor
Douglas Stuart Greene
Kenneth Joseph Valenzano
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=46831259&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ZA201306735(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of ZA201306735B publication Critical patent/ZA201306735B/en

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
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • 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)
    • C12Y302/01062Glycosylceramidase (3.2.1.62)
    • 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
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/20Pills, tablets, discs, rods

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Zoology (AREA)
  • Obesity (AREA)
  • Wood Science & Technology (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Hydrogenated Pyridines (AREA)
ZA2013/06735A 2011-03-11 2013-09-09 Dosing regimens for the treatment of fabry disease ZA201306735B (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201161451798P 2011-03-11 2011-03-11
US201161578201P 2011-12-20 2011-12-20
US201261596165P 2012-02-07 2012-02-07
PCT/US2012/028260 WO2012125402A2 (en) 2011-03-11 2012-03-08 Dosing regimens for the treatment of fabry disease

Publications (1)

Publication Number Publication Date
ZA201306735B true ZA201306735B (en) 2014-07-30

Family

ID=46831259

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2013/06735A ZA201306735B (en) 2011-03-11 2013-09-09 Dosing regimens for the treatment of fabry disease

Country Status (16)

Country Link
US (4) US20140219986A1 (cg-RX-API-DMAC7.html)
EP (2) EP2683382B1 (cg-RX-API-DMAC7.html)
JP (5) JP2014528901A (cg-RX-API-DMAC7.html)
KR (1) KR20140011367A (cg-RX-API-DMAC7.html)
CN (2) CN107088225A (cg-RX-API-DMAC7.html)
AU (6) AU2012229330B2 (cg-RX-API-DMAC7.html)
CA (1) CA2829947C (cg-RX-API-DMAC7.html)
CL (1) CL2013002601A1 (cg-RX-API-DMAC7.html)
EA (1) EA031874B1 (cg-RX-API-DMAC7.html)
ES (1) ES2807502T3 (cg-RX-API-DMAC7.html)
HK (1) HK1242998A1 (cg-RX-API-DMAC7.html)
MX (1) MX2013010446A (cg-RX-API-DMAC7.html)
SG (2) SG10201604757RA (cg-RX-API-DMAC7.html)
TW (3) TW201740945A (cg-RX-API-DMAC7.html)
WO (1) WO2012125402A2 (cg-RX-API-DMAC7.html)
ZA (1) ZA201306735B (cg-RX-API-DMAC7.html)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2533050T6 (da) 2006-05-16 2015-09-21 Amicus Therapeutics Inc Behandlingsmuligheder ved Fabrys sygdom
HUE042882T2 (hu) 2008-02-12 2019-07-29 Amicus Therapeutics Inc Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére
UY34317A (es) 2011-09-12 2013-02-28 Genzyme Corp Anticuerpo antireceptor de célula T (alfa)/ß
US9790268B2 (en) 2012-09-12 2017-10-17 Genzyme Corporation Fc containing polypeptides with altered glycosylation and reduced effector function
WO2014164534A2 (en) 2013-03-11 2014-10-09 Genzyme Corporation Site-specific antibody-drug conjugation through glycoengineering
CN106687596A (zh) * 2014-02-17 2017-05-17 葛兰素史密斯克莱知识产权发展有限公司 微生物学方法
RS64072B1 (sr) 2014-03-19 2023-04-28 Genzyme Corp Mesto-specifični glikoinženjering ciljnih delova
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP7001593B2 (ja) 2015-08-11 2022-01-19 コグノア, インコーポレイテッド 人工知能およびユーザ入力を用いて発達進度を判定するための方法および装置
US11972336B2 (en) 2015-12-18 2024-04-30 Cognoa, Inc. Machine learning platform and system for data analysis
EP3432882B1 (en) * 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
KR20240017126A (ko) * 2016-07-19 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 Ert-치료경험이 없는 환자 및 있는 환자에서 파브리병의 치료
WO2018090009A1 (en) 2016-11-14 2018-05-17 Cognoa, Inc. Methods and apparatus for evaluating developmental conditions and providing control over coverage and reliability
JP7324709B2 (ja) * 2017-02-09 2023-08-10 コグノア,インク. デジタル個別化医療のためのプラットフォームとシステム
HUE069837T2 (hu) * 2017-05-30 2025-04-28 Amicus Therapeutics Inc Bizonyos mutációkat hordozó Fabry-kóros betegek kezelése
BR112019025083B1 (pt) 2017-05-30 2022-08-09 Amicus Therapeutics, Inc Métodos de tratamento de pacientes portadores de fabry com insuficiência renal
JP7066157B2 (ja) 2017-06-14 2022-05-13 学校法人 明治薬科大学 ファブリー病治療用医薬の組合せ物及びその利用
EP3749307B1 (en) * 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4299117A3 (en) * 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
AU2020248351B2 (en) 2019-03-22 2025-09-25 Cognoa, Inc. Personalized digital therapy methods and devices
CN113939534B (zh) 2019-04-03 2025-02-28 建新公司 具有降低的断裂的抗αβTCR结合多肽
EA202290024A1 (ru) * 2019-06-11 2022-03-14 Амикус Терапьютикс, Инк. Способы лечения болезни фабри у пациентов с почечной недостаточностью
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin 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
US20240120050A1 (en) * 2022-10-07 2024-04-11 Insight Direct Usa, Inc. Machine learning method for predicting a health outcome of a patient using video and audio analytics
US20240197706A1 (en) * 2022-12-13 2024-06-20 Amicus Therapeutics, Inc. Methods of improving the pharmacokinetics of migalastat

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4551177A (en) * 1984-04-23 1985-11-05 National Starch And Chemical Corporation Compressible starches as binders for tablets or capsules
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
ES2686775T3 (es) * 2003-01-31 2018-10-19 Mount Sinai School Of Medicine Of New York University Terapia de combinación para tratar trastornos de deficiencia de proteínas
US7879354B2 (en) * 2004-01-13 2011-02-01 Mcneil-Ppc, Inc. Rapidly disintegrating gelatinous coated tablets
CN100457106C (zh) * 2005-06-15 2009-02-04 同济大学 1-脱氧野尻霉素在制备治疗糖尿病肾病药物中的应用
DK2533050T6 (da) * 2006-05-16 2015-09-21 Amicus Therapeutics Inc Behandlingsmuligheder ved Fabrys sygdom
US20100113517A1 (en) * 2007-03-30 2010-05-06 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
US9056101B2 (en) * 2007-04-26 2015-06-16 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US20100291060A1 (en) * 2007-08-29 2010-11-18 Shire Human Genetic Therapies, Inc Subcutaneous administration of alpha-galactosidase a

Also Published As

Publication number Publication date
EP2683382B1 (en) 2020-04-22
AU2020201715A1 (en) 2020-04-09
AU2012229330B2 (en) 2016-04-21
CA2829947A1 (en) 2012-09-20
MX2013010446A (es) 2014-03-27
KR20140011367A (ko) 2014-01-28
NZ615726A (en) 2015-01-30
US20140219986A1 (en) 2014-08-07
JP2023093448A (ja) 2023-07-04
CN103974619B (zh) 2017-02-15
CL2013002601A1 (es) 2014-03-28
TW201302197A (zh) 2013-01-16
US20240115708A1 (en) 2024-04-11
EP2683382A2 (en) 2014-01-15
JP2014528901A (ja) 2014-10-30
WO2012125402A2 (en) 2012-09-20
AU2022203011A1 (en) 2022-05-26
AU2012229330A1 (en) 2013-10-03
HK1242998A1 (zh) 2018-07-06
TWI624258B (zh) 2018-05-21
US20180153999A1 (en) 2018-06-07
EP2683382A4 (en) 2014-10-22
CN103974619A (zh) 2014-08-06
AU2016204445A1 (en) 2016-07-21
AU2018201637A1 (en) 2018-04-05
EA031874B1 (ru) 2019-03-29
JP2020033360A (ja) 2020-03-05
TW201634048A (zh) 2016-10-01
TWI624259B (zh) 2018-05-21
WO2012125402A3 (en) 2014-05-01
JP2021098697A (ja) 2021-07-01
AU2024205251A1 (en) 2024-08-22
ES2807502T3 (es) 2021-02-23
TW201740945A (zh) 2017-12-01
SG193379A1 (en) 2013-10-30
CN107088225A (zh) 2017-08-25
EA201301018A1 (ru) 2014-04-30
US20200268890A1 (en) 2020-08-27
EP3698792A1 (en) 2020-08-26
AU2016204445B2 (en) 2018-03-22
JP2017132780A (ja) 2017-08-03
SG10201604757RA (en) 2016-08-30
CA2829947C (en) 2019-01-15

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