MX2020012700A - Composiciones farmaceuticas para el tratamiento de la deficiencia de esfingomielinasa acida. - Google Patents

Composiciones farmaceuticas para el tratamiento de la deficiencia de esfingomielinasa acida.

Info

Publication number
MX2020012700A
MX2020012700A MX2020012700A MX2020012700A MX2020012700A MX 2020012700 A MX2020012700 A MX 2020012700A MX 2020012700 A MX2020012700 A MX 2020012700A MX 2020012700 A MX2020012700 A MX 2020012700A MX 2020012700 A MX2020012700 A MX 2020012700A
Authority
MX
Mexico
Prior art keywords
acid sphingomyelinase
pharmaceutical compositions
treating acid
sphingomyelinase deficiency
compositions
Prior art date
Application number
MX2020012700A
Other languages
English (en)
Inventor
Claudia Buser
Mark Yang
Ramirez Bernardo Perez-
Grant Trierweiler
Sangeeta Benjwal
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 MX2020012700A publication Critical patent/MX2020012700A/es

Links

Classifications

    • 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)
    • 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

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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Inorganic Chemistry (AREA)
  • Biophysics (AREA)
  • Oil, Petroleum & Natural Gas (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)

Abstract

La presente invención proporciona composiciones, tales como composiciones líquidas acuosas y composiciones liofilizadas, que comprenden una esfingomielinasa ácida humana recombinante. También se proporcionan métodos para usar las composiciones para el tratamiento de pacientes que tienen una deficiencia de esfingomielinasa ácida.
MX2020012700A 2018-05-25 2019-05-24 Composiciones farmaceuticas para el tratamiento de la deficiencia de esfingomielinasa acida. MX2020012700A (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
MX2020012700A true MX2020012700A (es) 2021-04-28

Family

ID=66912954

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020012700A MX2020012700A (es) 2018-05-25 2019-05-24 Composiciones farmaceuticas para el tratamiento de la deficiencia de esfingomielinasa acida.

Country Status (14)

Country Link
US (1) US20210145941A1 (es)
EP (1) EP3802805A1 (es)
JP (2) JP7497300B2 (es)
KR (1) KR20210015903A (es)
CN (1) CN112424347A (es)
AU (1) AU2019275109A1 (es)
BR (1) BR112020023829A2 (es)
CA (1) CA3101688A1 (es)
CO (1) CO2020014399A2 (es)
IL (1) IL278964A (es)
MX (1) MX2020012700A (es)
SG (1) SG11202011488WA (es)
UY (1) UY38238A (es)
WO (1) WO2019227029A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (11)

* 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
JP4871124B2 (ja) 2003-06-20 2012-02-08 アレス トレーディング ソシエテ アノニム 凍結乾燥ｆｓｈ／ｌｈ製剤
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
SI1986612T1 (sl) 2006-02-07 2013-01-31 Shire Human Genetic Therapies, Inc. Stabilizirana spojina glukocerebrosidaze
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
CA2764180A1 (en) * 2009-06-18 2010-12-23 Wyeth Llc Lyophilized formulations for small modular immunopharmaceuticals
HRP20211992T1 (hr) 2009-08-28 2022-04-01 Icahn School Of Medicine At Mount Sinai Zamjenska enzimska terapija povećanjem doze za liječenje nedostatka kiseline sfingomijelinaze
US9320711B2 (en) * 2010-06-25 2016-04-26 Shire Human Genetic Therapies, Inc. Methods and compositions for CNS delivery of heparan N-sulfatase
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
FI126979B (en) 2016-02-29 2017-09-15 Faron Pharmaceuticals Oy Lyophilized pharmaceutical formulation and use thereof
JP7216075B2 (ja) * 2017-08-24 2023-01-31 サノフイ 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置

Also Published As

Publication number Publication date
JP7497300B2 (ja) 2024-06-10
BR112020023829A2 (pt) 2021-05-11
AU2019275109A1 (en) 2021-01-21
JP2024069459A (ja) 2024-05-21
EP3802805A1 (en) 2021-04-14
US20210145941A1 (en) 2021-05-20
JP2021525083A (ja) 2021-09-24
WO2019227029A1 (en) 2019-11-28
KR20210015903A (ko) 2021-02-10
CA3101688A1 (en) 2019-11-28
CO2020014399A2 (es) 2020-12-10
CN112424347A (zh) 2021-02-26
IL278964A (en) 2021-01-31
UY38238A (es) 2019-12-31
SG11202011488WA (en) 2020-12-30

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