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
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
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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Ceased legal-status Critical Current
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    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7012—Compounds 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • A61K47/38—Cellulose; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0002—Galenical forms characterised by the drug release technique; Application systems commanded by energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/06—Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2009—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2013—Organic compounds, e.g. phospholipids, fats
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2022—Organic macromolecular compounds
    • A61K9/2031—Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyethylene oxide, poloxamers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2022—Organic macromolecular compounds
    • A61K9/205—Polysaccharides, e.g. alginate, gums; Cyclodextrin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2022—Organic macromolecular compounds
    • A61K9/205—Polysaccharides, e.g. alginate, gums; Cyclodextrin
    • A61K9/2054—Cellulose; Cellulose derivatives, e.g. hydroxypropyl methylcellulose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2022—Organic macromolecular compounds
    • A61K9/2063—Proteins, e.g. gelatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841—Filling excipients; Inactive ingredients
    • A61K9/485—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841—Filling excipients; Inactive ingredients
    • A61K9/4858—Organic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841—Filling excipients; Inactive ingredients
    • A61K9/4866—Organic macromolecular compounds
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • 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
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35—Heterocyclic 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations 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)
  • Inorganic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Biophysics (AREA)
  • Nutrition Science (AREA)
  • Physiology (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
KR20140003380A (ko) 2010-07-13 2014-01-09 울트라제닉스 파마수티컬 인코포레이티드 시알산 결함 치료용 방법들 및 제형들
AU2012328874B2 (en) 2011-10-24 2017-08-31 Ultragenyx Pharmaceutical Inc. Sialic acid analogs
CN104271125A (zh) * 2012-01-18 2015-01-07 奥特吉尼克斯制药公司 用于治疗唾液酸缺乏的方法和制剂
US20140057856A1 (en) * 2012-08-22 2014-02-27 The Regents Of The Universtiy Of California Compositions and Methods For Enhancing Sialic Acid Levels In Tissue
MX2017006167A (es) 2014-11-12 2018-03-23 Siamab Therapeutics Inc Compuestos que interactúan con glicanos y métodos de uso.
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
WO2018094143A1 (en) 2016-11-17 2018-05-24 Siamab Therapeutics, Inc. Glycan-interacting compounds and methods of use
KR20240044544A (ko) 2017-03-03 2024-04-04 씨젠 인크. 글리칸-상호작용 화합물 및 사용 방법
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
CN121793948A (zh) * 2023-09-01 2026-04-03 豪夫迈·罗氏有限公司 用于制备半固体组合物的新颖方法及其用途

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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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US9241896B2 (en) 2008-12-19 2016-01-26 Ultragenyx Pharmaceutical Inc. Methods and formulations for treating sialic acid deficiencies
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KR20140003380A (ko) 2010-07-13 2014-01-09 울트라제닉스 파마수티컬 인코포레이티드 시알산 결함 치료용 방법들 및 제형들
AU2012328874B2 (en) 2011-10-24 2017-08-31 Ultragenyx Pharmaceutical Inc. Sialic acid analogs
CN104271125A (zh) 2012-01-18 2015-01-07 奥特吉尼克斯制药公司 用于治疗唾液酸缺乏的方法和制剂

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

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