JP6381521B2 - ポンペ病の処置のための投与計画 - Google Patents

ポンペ病の処置のための投与計画 Download PDF

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JP6381521B2
JP6381521B2 JP2015510446A JP2015510446A JP6381521B2 JP 6381521 B2 JP6381521 B2 JP 6381521B2 JP 2015510446 A JP2015510446 A JP 2015510446A JP 2015510446 A JP2015510446 A JP 2015510446A JP 6381521 B2 JP6381521 B2 JP 6381521B2
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deoxynojirimycin
administration
enzyme replacement
acid
glucosidase enzyme
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JP2015519329A (ja
JP2015519329A5 (enExample
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ダグラス スチュアート グリーン，
ダグラス スチュアート グリーン，
ケネス ジョセフ バレンツァノ，
ケネス ジョセフ バレンツァノ，
Original Assignee
アミカス セラピューティックス インコーポレイテッド
アミカス セラピューティックス インコーポレイテッド
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Hydrogenated Pyridines (AREA)
JP2015510446A 2012-05-03 2013-05-02 ポンペ病の処置のための投与計画 Active JP6381521B2 (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201261642311P 2012-05-03 2012-05-03
US61/642,311 2012-05-03
US201261664011P 2012-06-25 2012-06-25
US61/664,011 2012-06-25
US201261697179P 2012-09-05 2012-09-05
US61/697,179 2012-09-05
US201361749234P 2013-01-04 2013-01-04
US201361749132P 2013-01-04 2013-01-04
US61/749,234 2013-01-04
US61/749,132 2013-01-04
PCT/US2013/039215 WO2013166249A1 (en) 2012-05-03 2013-05-02 Dosing regimens for the treatment of pompe disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2018007672A Division JP6612370B2 (ja) 2012-05-03 2018-01-19 ポンペ病の処置のための投与計画

Publications (3)

Publication Number Publication Date
JP2015519329A JP2015519329A (ja) 2015-07-09
JP2015519329A5 JP2015519329A5 (enExample) 2018-03-08
JP6381521B2 true JP6381521B2 (ja) 2018-08-29

Family

ID=49514881

Family Applications (6)

Application Number Title Priority Date Filing Date
JP2015510446A Active JP6381521B2 (ja) 2012-05-03 2013-05-02 ポンペ病の処置のための投与計画
JP2018007672A Active JP6612370B2 (ja) 2012-05-03 2018-01-19 ポンペ病の処置のための投与計画
JP2019196583A Active JP6876111B2 (ja) 2012-05-03 2019-10-29 ポンペ病の処置のための投与計画
JP2021010931A Active JP7046241B2 (ja) 2012-05-03 2021-01-27 ポンペ病の処置のための投与計画
JP2022044812A Withdrawn JP2022101544A (ja) 2012-05-03 2022-03-22 ポンペ病の処置のための投与計画
JP2024034587A Withdrawn JP2024081667A (ja) 2012-05-03 2024-03-07 ポンペ病の処置のための投与計画

Family Applications After (5)

Application Number Title Priority Date Filing Date
JP2018007672A Active JP6612370B2 (ja) 2012-05-03 2018-01-19 ポンペ病の処置のための投与計画
JP2019196583A Active JP6876111B2 (ja) 2012-05-03 2019-10-29 ポンペ病の処置のための投与計画
JP2021010931A Active JP7046241B2 (ja) 2012-05-03 2021-01-27 ポンペ病の処置のための投与計画
JP2022044812A Withdrawn JP2022101544A (ja) 2012-05-03 2022-03-22 ポンペ病の処置のための投与計画
JP2024034587A Withdrawn JP2024081667A (ja) 2012-05-03 2024-03-07 ポンペ病の処置のための投与計画

Country Status (15)

Country Link
US (1) US20150086530A1 (enExample)
EP (3) EP3871688B1 (enExample)
JP (6) JP6381521B2 (enExample)
CY (1) CY1124135T1 (enExample)
DK (1) DK2844279T3 (enExample)
ES (2) ES2859761T3 (enExample)
HR (1) HRP20210398T1 (enExample)
HU (1) HUE053565T2 (enExample)
LT (1) LT2844279T (enExample)
PL (1) PL2844279T3 (enExample)
PT (1) PT2844279T (enExample)
RS (1) RS61598B1 (enExample)
SI (1) SI2844279T1 (enExample)
SM (1) SMT202100136T1 (enExample)
WO (1) WO2013166249A1 (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12004868B2 (en) 2011-06-03 2024-06-11 Signpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac IKr channel
US10238602B2 (en) 2011-06-03 2019-03-26 Signpath Pharma, Inc. Protective effect of DMPC, DMPG, DMPC/DMPG, LysoPG and LysoPC against drugs that cause channelopathies
US10117881B2 (en) 2011-06-03 2018-11-06 Signpath Pharma, Inc. Protective effect of DMPC, DMPG, DMPC/DMPG, LYSOPG and LYSOPC against drugs that cause channelopathies
US8753674B2 (en) 2011-06-03 2014-06-17 Signpath Pharma Inc. Liposomal mitigation of drug-induced long QT syndrome and potassium delayed-rectifier current
US10449193B2 (en) 2011-06-03 2019-10-22 Signpath Pharma Inc. Protective effect of DMPC, DMPG, DMPC/DMPG, lysoPG and lysoPC against drugs that cause channelopathies
US10349884B2 (en) 2011-06-03 2019-07-16 Sighpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac ikr channel
RS61598B1 (sr) * 2012-05-03 2021-04-29 Amicus Therapeutics Inc Režimi doziranja za tretman pompeove bolesti
JP6895252B2 (ja) * 2013-12-18 2021-06-30 サインパス ファルマ, インク.Signpath Pharma, Inc. 心筋ｉｋｒチャネルの薬剤誘発性阻害のリポソームによる軽減
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
EP3151837B1 (en) 2014-06-03 2023-03-15 Signpath Pharma, Inc. Protective effect of dmpc, dmpg, dmpc/dmpg, egpg, lysopg and lysopc against drugs that cause channelopathies
JP6851964B2 (ja) 2014-09-30 2021-03-31 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
ES3063549T3 (en) 2015-12-30 2026-04-17 Amicus Therapeutics Inc Augmented acid alpha-glucosidase for the treatment of pompe disease
IL299470B2 (en) 2015-12-30 2026-05-01 Amicus Therapeutics Inc Improved acid alpha-glucosidase for the treatment of Pompe disease
KR102618519B1 (ko) 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
EP3448374B1 (en) 2016-04-27 2025-02-12 Signpath Pharma Inc. Prevention of drug-induced atrio-ventricular block
WO2018209300A1 (en) * 2017-05-12 2018-11-15 Duke University Methods for the use of low-dose immune modulators transiently for treating patients undergoing protein replacement therapy
CN119113091A (zh) * 2017-05-15 2024-12-13 阿米库斯治疗学公司 重组人类酸性α-葡萄糖苷酶
WO2022227287A1 (zh) * 2021-04-25 2022-11-03 中南大学 Dnj及其衍生物在制备预防和/或治疗肺动脉高压药物中的应用

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1436316B1 (en) 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. Subcellular targeting of therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US7658916B2 (en) 2002-04-05 2010-02-09 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density
PT2332972T (pt) 2003-06-24 2018-04-17 Genzyme Corp Novos promotores de beta-actina e rps21 e suas utilizações
US20080014188A1 (en) 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof
DE602005020745D1 (de) 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
EP3782655A1 (en) * 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
AU2008245578A1 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
RS61598B1 (sr) * 2012-05-03 2021-04-29 Amicus Therapeutics Inc Režimi doziranja za tretman pompeove bolesti

Also Published As

Publication number Publication date
HRP20210398T1 (hr) 2021-05-28
JP2020045346A (ja) 2020-03-26
LT2844279T (lt) 2021-03-25
SMT202100136T1 (it) 2021-05-07
JP2024081667A (ja) 2024-06-18
HUE053565T2 (hu) 2021-07-28
EP2844279A4 (en) 2015-12-16
EP4397364A3 (en) 2024-09-04
JP2021088561A (ja) 2021-06-10
SI2844279T1 (sl) 2021-04-30
JP2015519329A (ja) 2015-07-09
EP2844279A1 (en) 2015-03-11
CY1124135T1 (el) 2022-05-27
RS61598B1 (sr) 2021-04-29
JP2018109003A (ja) 2018-07-12
DK2844279T3 (da) 2021-03-15
EP3871688B1 (en) 2024-03-06
EP2844279B1 (en) 2020-12-09
JP6612370B2 (ja) 2019-11-27
JP6876111B2 (ja) 2021-05-26
PL2844279T3 (pl) 2021-08-16
JP7046241B2 (ja) 2022-04-01
WO2013166249A1 (en) 2013-11-07
ES2980828T3 (es) 2024-10-03
US20150086530A1 (en) 2015-03-26
PT2844279T (pt) 2021-03-11
JP2022101544A (ja) 2022-07-06
EP3871688A1 (en) 2021-09-01
ES2859761T3 (es) 2021-10-04
EP4397364A2 (en) 2024-07-10

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