AU2011279158B2 - Method and formulations for treating sialic acid deficiencies - Google Patents

Method and formulations for treating sialic acid deficiencies Download PDF

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Publication number
AU2011279158B2
AU2011279158B2 AU2011279158A AU2011279158A AU2011279158B2 AU 2011279158 B2 AU2011279158 B2 AU 2011279158B2 AU 2011279158 A AU2011279158 A AU 2011279158A AU 2011279158 A AU2011279158 A AU 2011279158A AU 2011279158 B2 AU2011279158 B2 AU 2011279158B2
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AU
Australia
Prior art keywords
sialic acid
extended release
release formulation
pharmaceutically acceptable
acceptable salt
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
AU2011279158A
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English (en)
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AU2011279158A1 (en
Inventor
Emil Kakkis
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Ultragenyx Pharmaceutical Inc
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Ultragenyx Pharmaceutical Inc
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Publication of AU2011279158A1 publication Critical patent/AU2011279158A1/en
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Publication of AU2011279158B2 publication Critical patent/AU2011279158B2/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7012Compounds having a free or esterified carboxyl group attached, directly or through a carbon chain, to a carbon atom of the saccharide radical, e.g. glucuronic acid, neuraminic acid
    • 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
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • A61K47/38Cellulose; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0002Galenical forms characterised by the drug release technique; Application systems commanded by energy
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/06Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • 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
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2009Inorganic compounds
    • 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
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2013Organic compounds, e.g. phospholipids, fats
    • 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
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2022Organic macromolecular compounds
    • A61K9/2031Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyethylene oxide, poloxamers
    • 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
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2022Organic macromolecular compounds
    • A61K9/205Polysaccharides, e.g. alginate, gums; Cyclodextrin
    • 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
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2022Organic macromolecular compounds
    • A61K9/205Polysaccharides, e.g. alginate, gums; Cyclodextrin
    • A61K9/2054Cellulose; Cellulose derivatives, e.g. hydroxypropyl methylcellulose
    • 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
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2022Organic macromolecular compounds
    • A61K9/2063Proteins, e.g. gelatin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841Filling excipients; Inactive ingredients
    • A61K9/485Inorganic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841Filling excipients; Inactive ingredients
    • A61K9/4858Organic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841Filling excipients; Inactive ingredients
    • A61K9/4866Organic macromolecular compounds
    • 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
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
    • 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
    • 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
    • A61K9/2004Excipients; Inactive ingredients
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate

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)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Inorganic Chemistry (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Biophysics (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2011279158A 2010-07-13 2011-07-13 Method and formulations for treating sialic acid deficiencies Ceased AU2011279158B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US36399510P 2010-07-13 2010-07-13
US61/363,995 2010-07-13
PCT/US2011/043910 WO2012009474A1 (en) 2010-07-13 2011-07-13 Method and formulations for treating sialic acid deficiencies

Publications (2)

Publication Number Publication Date
AU2011279158A1 AU2011279158A1 (en) 2013-02-21
AU2011279158B2 true AU2011279158B2 (en) 2015-07-09

Family

ID=45469794

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2011279158A Ceased AU2011279158B2 (en) 2010-07-13 2011-07-13 Method and formulations for treating sialic acid deficiencies

Country Status (10)

Country Link
US (4) US9511015B2 (enExample)
EP (1) EP2593109A4 (enExample)
JP (1) JP5777712B2 (enExample)
KR (1) KR20140003380A (enExample)
CN (2) CN108606956A (enExample)
AU (1) AU2011279158B2 (enExample)
BR (1) BR112013000776A2 (enExample)
CA (1) CA2805222A1 (enExample)
IL (1) IL224203B (enExample)
WO (1) WO2012009474A1 (enExample)

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US9241896B2 (en) 2008-12-19 2016-01-26 Ultragenyx Pharmaceutical Inc. Methods and formulations for treating sialic acid deficiencies
JP5777712B2 (ja) 2010-07-13 2015-09-09 ウルトラジェニックス ファーマシューティカル インコーポレイテッド シアル酸欠乏症を治療するための方法および製剤
CA2849114C (en) 2011-10-24 2020-12-15 Ultragenyx Pharmaceutical Inc. Sialic acid analogs
BR112014017587A8 (pt) * 2012-01-18 2017-07-04 Ultragenyx Pharmaceutical Inc métodos e formulações para o tratamento de deficiências de ácido siálico
WO2014031837A1 (en) * 2012-08-22 2014-02-27 The Regents Of The University Of California Compositions and methods for enhancing sialic acid levels in tissue
SI3218005T1 (sl) 2014-11-12 2023-06-30 Seagen Inc. Z glikanom delujoče spojine, in postopki uporabe
AR106025A1 (es) 2015-09-14 2017-12-06 Ultragenyx Pharmaceutical Inc Formas cristalinas de ácido siálico o una sal o solvato de este
CA3002097A1 (en) 2015-11-12 2017-05-18 Siamab Therapeutics, Inc. Glycan-interacting compounds and methods of use
EP3541847A4 (en) 2016-11-17 2020-07-08 Seattle Genetics, Inc. COMPOUNDS INTERACTING WITH GLYCANE AND METHODS OF USE
EP3589319A4 (en) 2017-03-03 2021-07-14 Seagen Inc. COMPOUNDS INTERACTING WITH GLYCAN AND METHODS OF USE
US20200327425A1 (en) * 2017-10-31 2020-10-15 Once Labs Inc. Automated feedback-driven prognosis system and method
EP3486326A1 (en) 2017-11-21 2019-05-22 Jennewein Biotechnologie GmbH Method for the purification of n-acetylneuraminic acid from a fermentation broth
US20200371110A1 (en) * 2018-01-12 2020-11-26 The Wistar Institute Of Anatomy And Biology Methods for identifying and treating hiv persistence
CN110548012B (zh) * 2019-08-27 2022-05-13 嘉必优生物技术(武汉)股份有限公司 一种唾液酸片剂及其制备方法
TW202513040A (zh) * 2023-09-01 2025-04-01 瑞士商赫孚孟拉羅股份公司 製備半固體組成物之新穎方法及其用途

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WO2004000366A1 (en) * 2002-06-21 2003-12-31 Novo Nordisk Health Care Ag Pegylated factor vii glycoforms
WO2008150477A2 (en) * 2007-05-31 2008-12-11 Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services National Institutes Of Health N-acetyl mannosamine as a therapeutic agent
WO2009032605A2 (en) * 2007-08-27 2009-03-12 Massachusetts Institute Of Technology Bi-functional polymer-attached inhibitors of influenza virus

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JP5626734B2 (ja) 2009-05-15 2014-11-19 公益財団法人ヒューマンサイエンス振興財団 Gneタンパク質の機能低下に起因する疾患の治療用医薬剤、食品組成物
JP5777712B2 (ja) 2010-07-13 2015-09-09 ウルトラジェニックス ファーマシューティカル インコーポレイテッド シアル酸欠乏症を治療するための方法および製剤
CA2849114C (en) 2011-10-24 2020-12-15 Ultragenyx Pharmaceutical Inc. Sialic acid analogs
BR112014017587A8 (pt) 2012-01-18 2017-07-04 Ultragenyx Pharmaceutical Inc métodos e formulações para o tratamento de deficiências de ácido siálico

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004000366A1 (en) * 2002-06-21 2003-12-31 Novo Nordisk Health Care Ag Pegylated factor vii glycoforms
WO2008150477A2 (en) * 2007-05-31 2008-12-11 Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services National Institutes Of Health N-acetyl mannosamine as a therapeutic agent
WO2009032605A2 (en) * 2007-08-27 2009-03-12 Massachusetts Institute Of Technology Bi-functional polymer-attached inhibitors of influenza virus

Also Published As

Publication number Publication date
US9554987B2 (en) 2017-01-31
KR20140003380A (ko) 2014-01-09
US9511015B2 (en) 2016-12-06
US20130273160A1 (en) 2013-10-17
AU2011279158A1 (en) 2013-02-21
US20130225513A1 (en) 2013-08-29
CN103370069A (zh) 2013-10-23
JP5777712B2 (ja) 2015-09-09
IL224203B (en) 2018-06-28
WO2012009474A1 (en) 2012-01-19
US20170157160A1 (en) 2017-06-08
CA2805222A1 (en) 2012-01-19
US20170136051A1 (en) 2017-05-18
BR112013000776A2 (pt) 2016-05-24
CN108606956A (zh) 2018-10-02
EP2593109A1 (en) 2013-05-22
JP2013531041A (ja) 2013-08-01
EP2593109A4 (en) 2014-01-08

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