AU2019275109C1 - Pharmaceutical compositions for treating acid sphingomyelinase deficiency - Google Patents

Pharmaceutical compositions for treating acid sphingomyelinase deficiency

Info

Publication number
AU2019275109C1
AU2019275109C1 AU2019275109A AU2019275109A AU2019275109C1 AU 2019275109 C1 AU2019275109 C1 AU 2019275109C1 AU 2019275109 A AU2019275109 A AU 2019275109A AU 2019275109 A AU2019275109 A AU 2019275109A AU 2019275109 C1 AU2019275109 C1 AU 2019275109C1
Authority
AU
Australia
Prior art keywords
composition
sodium phosphate
sucrose
methionine
lyophilized
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.)
Active
Application number
AU2019275109A
Other languages
English (en)
Other versions
AU2019275109B2 (en
AU2019275109A1 (en
Inventor
Sangeeta BENJWAL
Claudia BUSER
Bernardo Perez-Ramirez
Grant TRIERWEILER
Mark Yang
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.)
Genzyme Corp
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of AU2019275109A1 publication Critical patent/AU2019275109A1/en
Application granted granted Critical
Publication of AU2019275109B2 publication Critical patent/AU2019275109B2/en
Priority to AU2025205577A priority Critical patent/AU2025205577A1/en
Publication of AU2019275109C1 publication Critical patent/AU2019275109C1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/20—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K9/08—Solutions
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1611—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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • A61K9/1623—Sugars or sugar alcohols, e.g. lactose; Derivatives thereof; Homeopathic globules
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Inorganic Chemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2019275109A 2018-05-25 2019-05-24 Pharmaceutical compositions for treating acid sphingomyelinase deficiency Active AU2019275109C1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025205577A AU2025205577A1 (en) 2018-05-25 2025-07-17 Pharmaceutical compositions for treating acid sphingomyelinase deficiency

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862676525P 2018-05-25 2018-05-25
US62/676,525 2018-05-25
PCT/US2019/033983 WO2019227029A1 (en) 2018-05-25 2019-05-24 Pharmaceutical compositions for treating acid sphingomyelinase deficiency

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2025205577A Division AU2025205577A1 (en) 2018-05-25 2025-07-17 Pharmaceutical compositions for treating acid sphingomyelinase deficiency

Publications (3)

Publication Number Publication Date
AU2019275109A1 AU2019275109A1 (en) 2021-01-21
AU2019275109B2 AU2019275109B2 (en) 2025-04-17
AU2019275109C1 true AU2019275109C1 (en) 2025-11-13

Family

ID=66912954

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2019275109A Active AU2019275109C1 (en) 2018-05-25 2019-05-24 Pharmaceutical compositions for treating acid sphingomyelinase deficiency
AU2025205577A Pending AU2025205577A1 (en) 2018-05-25 2025-07-17 Pharmaceutical compositions for treating acid sphingomyelinase deficiency

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2025205577A Pending AU2025205577A1 (en) 2018-05-25 2025-07-17 Pharmaceutical compositions for treating acid sphingomyelinase deficiency

Country Status (15)

Country Link
US (1) US20210145941A1 (https=)
EP (1) EP3802805A1 (https=)
JP (2) JP7497300B2 (https=)
KR (1) KR102852946B1 (https=)
CN (1) CN112424347A (https=)
AU (2) AU2019275109C1 (https=)
BR (1) BR112020023829A2 (https=)
CA (1) CA3101688A1 (https=)
CO (1) CO2020014399A2 (https=)
IL (1) IL278964A (https=)
MX (2) MX2020012700A (https=)
PY (1) PY1939780A (https=)
SG (1) SG11202011488WA (https=)
UY (1) UY38238A (https=)
WO (1) WO2019227029A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024506860A (ja) * 2021-02-01 2024-02-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ニーマンピック病ａ型を治療するための組成物及び方法
TW202403043A (zh) 2022-03-18 2024-01-16 美商健臻公司 重組人類酸性鞘磷脂酶醫藥組合物及方法

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007084737A2 (en) * 2006-01-20 2007-07-26 Genzyme Corporation Intraventricular enzyme delivery for lysosomal storage diseases
WO2010148337A1 (en) * 2009-06-18 2010-12-23 Wyeth Llc Lyophilized formulations for small modular immunopharmaceuticals
WO2017149199A1 (en) * 2016-02-29 2017-09-08 Faron Pharmaceuticals Oy A lyophilised pharmaceutical formulation and its use

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
ES2351786T3 (es) * 2001-11-13 2011-02-10 Genentech, Inc. Formulaciones con ligando apo2/trail y usos de las mismas.
WO2004112826A1 (en) 2003-06-20 2004-12-29 Ares Trading Sa Freeze-dried fsh / lh formulations
JP2007514661A (ja) * 2003-11-25 2007-06-07 マウント シナイ スクール オブ メディスン オブ ニューヨーク ユニバーシティー ニーマンピック病のシャペロンに基づく治療方法
HRP20120994T1 (hr) 2006-02-07 2012-12-31 Shire Human Genetic Therapies, Inc. Stabilizirana smjesa glukocerebrozidaze
MX2008011842A (es) * 2006-03-13 2008-10-02 Encysive Pharmaceuticals Inc Procedimientos y composiciones para el tratamiento de insuficiencia cardiaca diastolica.
CN102943106A (zh) * 2006-03-13 2013-02-27 珀金埃尔默健康科学股份有限公司 用于质谱法检测的底物和内标
SI3998078T1 (sl) 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
WO2012134240A2 (ko) * 2011-03-31 2012-10-04 (주)메디톡스 보툴리눔 독소의 동결건조제제
JP6480154B2 (ja) 2014-11-06 2019-03-06 持田製薬株式会社 エタネルセプトの凍結乾燥製剤
KR20250025051A (ko) * 2017-08-24 2025-02-20 사노피 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007084737A2 (en) * 2006-01-20 2007-07-26 Genzyme Corporation Intraventricular enzyme delivery for lysosomal storage diseases
WO2010148337A1 (en) * 2009-06-18 2010-12-23 Wyeth Llc Lyophilized formulations for small modular immunopharmaceuticals
WO2017149199A1 (en) * 2016-02-29 2017-09-08 Faron Pharmaceuticals Oy A lyophilised pharmaceutical formulation and its use

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MURRAY JAMES M ET AL: "Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency: The utility of animal models of disease in *

Also Published As

Publication number Publication date
EP3802805A1 (en) 2021-04-14
UY38238A (es) 2019-12-31
WO2019227029A1 (en) 2019-11-28
PY1939780A (es) 2019-12-04
JP2021525083A (ja) 2021-09-24
IL278964A (en) 2021-01-31
AU2025205577A1 (en) 2025-08-07
US20210145941A1 (en) 2021-05-20
MX2025002733A (es) 2025-04-02
JP7497300B2 (ja) 2024-06-10
JP7832245B2 (ja) 2026-03-17
CO2020014399A2 (es) 2020-12-10
CA3101688A1 (en) 2019-11-28
CN112424347A (zh) 2021-02-26
MX2020012700A (es) 2021-04-28
BR112020023829A2 (pt) 2021-05-11
JP2024069459A (ja) 2024-05-21
AU2019275109B2 (en) 2025-04-17
AU2019275109A1 (en) 2021-01-21
SG11202011488WA (en) 2020-12-30
KR20210015903A (ko) 2021-02-10
KR102852946B1 (ko) 2025-09-02

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Legal Events

Date Code Title Description
DA2 Applications for amendment section 104

Free format text: THE NATURE OF THE AMENDMENT IS AS SHOWN IN THE STATEMENT(S) FILED 22 JUL 2025

DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS AS SHOWN IN THE STATEMENT FILED 22 JUL 2025

FGA Letters patent sealed or granted (standard patent)