PY1939780A - Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida - Google Patents

Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida

Info

Publication number
PY1939780A
PY1939780A PY201901939780A PY1939780A PY1939780A PY 1939780 A PY1939780 A PY 1939780A PY 201901939780 A PY201901939780 A PY 201901939780A PY 1939780 A PY1939780 A PY 1939780A PY 1939780 A PY1939780 A PY 1939780A
Authority
PY
Paraguay
Prior art keywords
treatment
acid sphingomyelinase
pharmaceutical compositions
sphingomyelinase deficiency
compositions
Prior art date
Application number
PY201901939780A
Other languages
English (en)
Spanish (es)
Inventor
Pérez-Ramírez Bernardo
Buser Claudia
Trierweiler Grant
Yang Mark
Benjwal Sangeeta
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 PY1939780A publication Critical patent/PY1939780A/es

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)
PY201901939780A 2018-05-25 2019-05-23 Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida PY1939780A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862676525P 2018-05-25 2018-05-25

Publications (1)

Publication Number Publication Date
PY1939780A true PY1939780A (es) 2019-12-04

Family

ID=66912954

Family Applications (1)

Application Number Title Priority Date Filing Date
PY201901939780A PY1939780A (es) 2018-05-25 2019-05-23 Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida

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
EP4284442A4 (en) * 2021-02-01 2025-04-23 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR TREATING NIEMANN PICK DISEASE TYPE A
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 (15)

* 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
EP1450847B1 (en) 2001-11-13 2010-09-29 Genentech, Inc. APO2 ligand/ TRAIL formulations and uses thereof
EP1638595B1 (en) 2003-06-20 2013-03-20 Ares Trading S.A. Freeze-dried fsh / lh formulations
CN101214249A (zh) * 2003-11-25 2008-07-09 纽约大学西奈山医学院 尼曼-皮克病的基于陪伴分子的治疗
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
HRP20120994T1 (hr) 2006-02-07 2012-12-31 Shire Human Genetic Therapies, Inc. Stabilizirana smjesa glukocerebrozidaze
AU2007226582B2 (en) * 2006-03-13 2012-07-05 Revvity Health Sciences, Inc. Substrates and internal standards for mass spectroscopy detection
JP2009530284A (ja) * 2006-03-13 2009-08-27 エンサイシブ・ファーマシューティカルズ・インコーポレイテッド 拡張期心不全を治療するための方法と組成物
JP2012530721A (ja) 2009-06-18 2012-12-06 ワイス・エルエルシー 小モジュール免疫薬のための凍結乾燥製剤
SI3998078T1 (sl) 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
JP6073783B2 (ja) * 2010-06-25 2017-02-01 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物
PL3241547T3 (pl) 2011-03-31 2021-02-08 Medy-Tox Inc. Liofilizowany preparat toksyny botulinowej
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 its use
KR20250025051A (ko) * 2017-08-24 2025-02-20 사노피 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

Also Published As

Publication number Publication date
KR102852946B1 (ko) 2025-09-02
JP7832245B2 (ja) 2026-03-17
JP2024069459A (ja) 2024-05-21
JP7497300B2 (ja) 2024-06-10
BR112020023829A2 (pt) 2021-05-11
AU2019275109A1 (en) 2021-01-21
MX2020012700A (es) 2021-04-28
CA3101688A1 (en) 2019-11-28
WO2019227029A1 (en) 2019-11-28
SG11202011488WA (en) 2020-12-30
AU2025205577A1 (en) 2025-08-07
CN112424347A (zh) 2021-02-26
EP3802805A1 (en) 2021-04-14
UY38238A (es) 2019-12-31
US20210145941A1 (en) 2021-05-20
CO2020014399A2 (es) 2020-12-10
JP2021525083A (ja) 2021-09-24
MX2025002733A (es) 2025-04-02
IL278964A (en) 2021-01-31
AU2019275109B2 (en) 2025-04-17
KR20210015903A (ko) 2021-02-10
AU2019275109C1 (en) 2025-11-13

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