HRP20210398T1 - Režimi doziranja za liječenje pompeove bolesti - Google Patents

Režimi doziranja za liječenje pompeove bolesti Download PDF

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Publication number
HRP20210398T1
HRP20210398T1 HRP20210398TT HRP20210398T HRP20210398T1 HR P20210398 T1 HRP20210398 T1 HR P20210398T1 HR P20210398T T HRP20210398T T HR P20210398TT HR P20210398 T HRP20210398 T HR P20210398T HR P20210398 T1 HRP20210398 T1 HR P20210398T1
Authority
HR
Croatia
Prior art keywords
dnj
pharmaceutically acceptable
acceptable salt
replacement therapy
human recombinant
Prior art date
Application number
HRP20210398TT
Other languages
English (en)
Croatian (hr)
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
Application filed by Amicus Therapeutics, Inc. filed Critical Amicus Therapeutics, Inc.
Publication of HRP20210398T1 publication Critical patent/HRP20210398T1/hr

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Classifications

    • 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
    • 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
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • 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)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (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)
HRP20210398TT 2012-05-03 2013-05-02 Režimi doziranja za liječenje pompeove bolesti HRP20210398T1 (hr)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201261642311P 2012-05-03 2012-05-03
US201261664011P 2012-06-25 2012-06-25
US201261697179P 2012-09-05 2012-09-05
US201361749234P 2013-01-04 2013-01-04
US201361749132P 2013-01-04 2013-01-04
PCT/US2013/039215 WO2013166249A1 (en) 2012-05-03 2013-05-02 Dosing regimens for the treatment of pompe disease
EP13785028.5A EP2844279B1 (en) 2012-05-03 2013-05-02 Dosing regimens for the treatment of pompe disease

Publications (1)

Publication Number Publication Date
HRP20210398T1 true HRP20210398T1 (hr) 2021-05-28

Family

ID=49514881

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20210398TT HRP20210398T1 (hr) 2012-05-03 2013-05-02 Režimi doziranja za liječenje pompeove bolesti

Country Status (15)

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

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US8753674B2 (en) 2011-06-03 2014-06-17 Signpath Pharma Inc. Liposomal mitigation of drug-induced long QT syndrome and potassium delayed-rectifier current
US10349884B2 (en) 2011-06-03 2019-07-16 Sighpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac ikr channel
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
US12004868B2 (en) 2011-06-03 2024-06-11 Signpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac IKr channel
US10532045B2 (en) 2013-12-18 2020-01-14 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
SMT202100136T1 (it) * 2012-05-03 2021-05-07 Amicus Therapeutics Inc Regimi posologici per il trattamento della malattia di pompe
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
AU2015269699B2 (en) 2014-06-03 2020-08-13 Avanti Polar Lipids, Inc. Protective effect of DMPC, DMPG, DMPC/DMPG, EGPG, LysoPG and LysoPC against drugs that cause channelopathies
CN114540327B (zh) 2014-09-30 2026-03-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
TW202417620A (zh) * 2014-09-30 2024-05-01 美商阿米庫斯醫療股份有限公司 高效經修飾之酸性α-葡萄糖苷酶及其製造與使用方法
KR20250007693A (ko) 2015-12-30 2025-01-14 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
HRP20231465T1 (hr) 2015-12-30 2024-03-01 Amicus Therapeutics, Inc. Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
AU2017257496B2 (en) 2016-04-27 2020-05-07 Signpath Pharma, Inc. Prevention of drug-induced atrio-ventricular block
US11944628B2 (en) 2017-05-12 2024-04-02 Duke University Methods for the use of low-dose immune modulators transiently for treating patients undergoing protein replacement therapy
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
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
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation 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
WO2002087510A2 (en) 2001-04-30 2002-11-07 Symbiontics, 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
WO2003086452A2 (en) 2002-04-05 2003-10-23 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
HUE036449T2 (hu) 2003-06-24 2018-07-30 Genzyme Corp Új béta-aktin és RPS21 promóterek és alkalmazásaik
US20080014188A1 (en) 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof
CA2553955C (en) 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
EP3782655A1 (en) * 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
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.
WO2010056746A1 (en) 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
SMT202100136T1 (it) * 2012-05-03 2021-05-07 Amicus Therapeutics Inc Regimi posologici per il trattamento della malattia di pompe

Also Published As

Publication number Publication date
JP2022101544A (ja) 2022-07-06
PT2844279T (pt) 2021-03-11
JP6612370B2 (ja) 2019-11-27
JP2018109003A (ja) 2018-07-12
LT2844279T (lt) 2021-03-25
JP2020045346A (ja) 2020-03-26
SMT202100136T1 (it) 2021-05-07
ES2859761T3 (es) 2021-10-04
EP2844279A1 (en) 2015-03-11
JP2024081667A (ja) 2024-06-18
ES2980828T3 (es) 2024-10-03
US20150086530A1 (en) 2015-03-26
JP6876111B2 (ja) 2021-05-26
EP2844279B1 (en) 2020-12-09
EP2844279A4 (en) 2015-12-16
SI2844279T1 (sl) 2021-04-30
CY1124135T1 (el) 2022-05-27
PL2844279T3 (pl) 2021-08-16
EP3871688B1 (en) 2024-03-06
EP3871688A1 (en) 2021-09-01
JP2015519329A (ja) 2015-07-09
WO2013166249A1 (en) 2013-11-07
HUE053565T2 (hu) 2021-07-28
DK2844279T3 (da) 2021-03-15
JP6381521B2 (ja) 2018-08-29
EP4397364A3 (en) 2024-09-04
RS61598B1 (sr) 2021-04-29
EP4397364A2 (en) 2024-07-10
JP2021088561A (ja) 2021-06-10
JP7046241B2 (ja) 2022-04-01

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