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CY1124637T1 - Αυξημενη οξινη αλφα-γαυκοσιδαση για την θεραπευτικη αγωγη της νοσου ρομρe - Google Patents

Αυξημενη οξινη αλφα-γαυκοσιδαση για την θεραπευτικη αγωγη της νοσου ρομρe

Info

Publication number
CY1124637T1
CY1124637T1 CY20211100705T CY211100705T CY1124637T1 CY 1124637 T1 CY1124637 T1 CY 1124637T1 CY 20211100705 T CY20211100705 T CY 20211100705T CY 211100705 T CY211100705 T CY 211100705T CY 1124637 T1 CY1124637 T1 CY 1124637T1
Authority
CY
Cyprus
Prior art keywords
disease
romre
gaucosidase
acid alpha
glucosidase
Prior art date
Application number
CY20211100705T
Other languages
English (en)
Inventor
Hung V. Do
Richie Khanna
Russell GOTSCHALL
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
Priority claimed from PCT/US2016/069243 external-priority patent/WO2017117407A1/en
Application filed by Amicus Therapeutics, Inc. filed Critical Amicus Therapeutics, Inc.
Publication of CY1124637T1 publication Critical patent/CY1124637T1/el

Links

Classifications

    • 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
    • A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/05—Phenols
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Παρέχεται μια μέθοδος θεραπευτικής αγωγής της νόσου Pompe η οποία περιλαμβάνει χορήγηση ανασυνδυασμένης ανθρώπινης όξινης α-γλυκοσιδάσης που έχει βέλτιστη γλυκοζυλίωση με υπολείμματα 6-φωσφορικής μαννόζης σε συνδυασμό με μια ποσότητα μιγλουστάτης αποτελεσματική για την μεγιστοποίηση της πρόσληψης ανασυνδυασμένης ανθρώπινης όξινης α-γλυκοσιδάσης, ενώ ελαχιστοποιείται η αναστολή της ενζυματικής δραστικότητας της ανασυνδυασμένης ανθρώπινης όξινης α-γλυκοσιδάσης.
CY20211100705T 2015-12-30 2021-08-06 Αυξημενη οξινη αλφα-γαυκοσιδαση για την θεραπευτικη αγωγη της νοσου ρομρe CY1124637T1 (el)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562272890P 2015-12-30 2015-12-30
US201662300479P 2016-02-26 2016-02-26
US201662315412P 2016-03-30 2016-03-30
US201662402454P 2016-09-30 2016-09-30
US201662428867P 2016-12-01 2016-12-01
US201662431791P 2016-12-08 2016-12-08
PCT/US2016/069243 WO2017117407A1 (en) 2015-12-30 2016-12-29 Augmented acid alpha-glucosidase for the treatment of pompe disease

Publications (1)

Publication Number Publication Date
CY1124637T1 true CY1124637T1 (el) 2022-07-22

Family

ID=60186557

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20211100705T CY1124637T1 (el) 2015-12-30 2021-08-06 Αυξημενη οξινη αλφα-γαυκοσιδαση για την θεραπευτικη αγωγη της νοσου ρομρe

Country Status (16)

Country Link
EP (3) EP4285902A3 (el)
CN (1) CN108472340A (el)
CY (1) CY1124637T1 (el)
DK (2) DK3397273T3 (el)
ES (2) ES2883844T3 (el)
FI (1) FI3957320T3 (el)
HK (1) HK1253014A1 (el)
HR (2) HRP20231465T1 (el)
HU (2) HUE054733T2 (el)
LT (2) LT3397273T (el)
PL (2) PL3957320T3 (el)
PT (2) PT3397273T (el)
RS (2) RS64843B1 (el)
SI (2) SI3957320T1 (el)
SM (2) SMT202300412T1 (el)
TW (2) TWI753874B (el)

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7723296B2 (en) * 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
SI2444102T1 (sl) 2003-01-31 2015-08-31 Mount Sinai School Of Medicine Of New York University Kombinirana terapija za zdravljenje motenj pomanjkanja proteina
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
AU2006247065B8 (en) 2005-05-17 2012-07-12 Amicus Therapeutics, Inc. A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives
AU2009214648B2 (en) 2008-02-12 2014-11-13 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
US20110070643A1 (en) * 2009-05-26 2011-03-24 Do Hung V Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics
EP4397364A3 (en) 2012-05-03 2024-09-04 Amicus Therapeutics, Inc. Dosing regimens for the treatment of pompe disease
WO2014014938A1 (en) 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
SG10202003753PA (en) * 2014-09-30 2020-05-28 Amicus Therapeutics Inc Highly potent acid alpha-glucosidase with enhanced carbohydrates

Also Published As

Publication number Publication date
DK3397273T3 (da) 2021-08-09
HRP20231465T1 (hr) 2024-03-01
ES2883844T3 (es) 2021-12-09
FI3957320T3 (fi) 2023-11-23
TW201726163A (zh) 2017-08-01
TW202245831A (zh) 2022-12-01
LT3397273T (lt) 2021-06-25
RS64843B1 (sr) 2023-12-29
HRP20210894T1 (hr) 2021-07-23
SMT202100452T1 (it) 2021-09-14
ES2965190T3 (es) 2024-04-11
PL3957320T3 (pl) 2024-02-19
DK3957320T5 (da) 2024-08-05
HUE054733T2 (hu) 2021-09-28
HK1253014A1 (zh) 2019-06-06
DK3957320T3 (da) 2023-11-27
SI3397273T1 (sl) 2021-11-30
HUE064049T2 (hu) 2024-02-28
LT3957320T (lt) 2023-12-11
PT3957320T (pt) 2023-11-23
RS62212B1 (sr) 2021-09-30
SI3957320T1 (sl) 2024-01-31
PT3397273T (pt) 2021-08-09
TWI753874B (zh) 2022-02-01
TWI849380B (zh) 2024-07-21
SMT202300412T1 (it) 2024-01-10
EP3397273B1 (en) 2021-05-19
EP4285902A3 (en) 2024-02-21
PL3397273T3 (pl) 2021-12-06
EP3397273A1 (en) 2018-11-07
CN108472340A (zh) 2018-08-31
EP4285902A2 (en) 2023-12-06
EP3957320A1 (en) 2022-02-23
EP3957320B1 (en) 2023-08-23

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