IL272757B2 - A recombinant human acid sphingomyelinase for use for treating an abnormal bone conditions, decreasing bone marrow burden and/or improving bone mineral density - Google Patents

A recombinant human acid sphingomyelinase for use for treating an abnormal bone conditions, decreasing bone marrow burden and/or improving bone mineral density

Info

Publication number
IL272757B2
IL272757B2 IL272757A IL27275720A IL272757B2 IL 272757 B2 IL272757 B2 IL 272757B2 IL 272757 A IL272757 A IL 272757A IL 27275720 A IL27275720 A IL 27275720A IL 272757 B2 IL272757 B2 IL 272757B2
Authority
IL
Israel
Prior art keywords
patient
rhasm
bone
doses
acid sphingomyelinase
Prior art date
Application number
IL272757A
Other languages
English (en)
Hebrew (he)
Other versions
IL272757A (en
IL272757B1 (en
Original Assignee
Sanofi Sa
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 Sanofi Sa, Genzyme Corp filed Critical Sanofi Sa
Publication of IL272757A publication Critical patent/IL272757A/en
Publication of IL272757B1 publication Critical patent/IL272757B1/en
Publication of IL272757B2 publication Critical patent/IL272757B2/en

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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
IL272757A 2017-08-24 2018-08-22 A recombinant human acid sphingomyelinase for use for treating an abnormal bone conditions, decreasing bone marrow burden and/or improving bone mineral density IL272757B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762549732P 2017-08-24 2017-08-24
EP17306720 2017-12-07
PCT/IB2018/056346 WO2019038685A2 (en) 2017-08-24 2018-08-22 TREATMENT OF ABNORMAL BONE CONDITIONS IN PATIENTS WITH ACIDIC SPHINGOMYELASE DISEASE

Publications (3)

Publication Number Publication Date
IL272757A IL272757A (en) 2020-04-30
IL272757B1 IL272757B1 (en) 2024-03-01
IL272757B2 true IL272757B2 (en) 2024-07-01

Family

ID=63556374

Family Applications (1)

Application Number Title Priority Date Filing Date
IL272757A IL272757B2 (en) 2017-08-24 2018-08-22 A recombinant human acid sphingomyelinase for use for treating an abnormal bone conditions, decreasing bone marrow burden and/or improving bone mineral density

Country Status (16)

Country Link
US (2) US11898175B2 (enExample)
EP (1) EP3672622B1 (enExample)
JP (3) JP7216075B2 (enExample)
KR (2) KR102769578B1 (enExample)
CN (2) CN118453843A (enExample)
AU (2) AU2018319565B2 (enExample)
BR (1) BR112020003541A2 (enExample)
CA (1) CA3073648A1 (enExample)
CO (1) CO2020001801A2 (enExample)
ES (1) ES2970423T3 (enExample)
IL (1) IL272757B2 (enExample)
MX (2) MX2020002106A (enExample)
PL (1) PL3672622T3 (enExample)
SG (1) SG11202001544VA (enExample)
TW (1) TWI791040B (enExample)
WO (1) WO2019038685A2 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2773133A1 (en) * 2009-08-28 2011-03-03 Edward H. Schuchman Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
PL3672622T3 (pl) * 2017-08-24 2024-04-02 Genzyme Corporation Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy
PY1939780A (es) * 2018-05-25 2019-12-04 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida
JP7568648B2 (ja) * 2019-05-31 2024-10-16 ジェンザイム・コーポレーション 二次元ｌｃ－ｍｓ／ｍｓシステム
CN112773779B (zh) * 2021-02-04 2022-08-16 上海交通大学医学院附属新华医院 氨溴索、千金藤和汉防已在治疗酸性鞘磷脂酶缺乏症中的应用
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011025996A2 (en) * 2009-08-28 2011-03-03 Mount Sinai School Of Medicine Of New York University Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

Family Cites Families (7)

* 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
CN1302897A (zh) 1999-10-28 2001-07-11 上海博容基因开发有限公司 一种新的多肽——人酸性鞘磷酯酶相似的磷酸二酯酶21和编码这种多肽的多核苷酸
US8158582B2 (en) * 2005-06-02 2012-04-17 Tercica, Inc. Methods for treatment of insulin-like growth factor-1 deficiency
WO2013130963A1 (en) 2012-03-02 2013-09-06 Shire Human Genetic Therapies, Inc. Compositions and methods for treating type iii gaucher disease
AR093908A1 (es) 2012-12-12 2015-06-24 Teva Pharma Fusion de la hormona de crecimiento humana y albumina, formulacion y usos de la misma
MX370570B (es) * 2013-06-07 2019-12-17 Genzyme Corp Marcador para trastornos de la esfingomielinasa acida y sus usos.
PL3672622T3 (pl) * 2017-08-24 2024-04-02 Genzyme Corporation Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011025996A2 (en) * 2009-08-28 2011-03-03 Mount Sinai School Of Medicine Of New York University Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
ANONYMOUS,, ARCHIVE HISTORY FOR NCT02004691, 3 August 2017 (2017-08-03) *
MARGARET M. MCGOVERN ET AL,, DISEASE MANIFESTATIONS AND BURDEN OF ILLNESS IN PATIENTS WITH ACID SPHINGOMYELINASE DEFICIENCY (ASMD), 23 February 2017 (2017-02-23) *
MARGARET M. MCGOVERN ET AL,, NOVEL FIRST-DOSE ADVERSE DRUG REACTIONS DURING A PHASE I TRIAL OF OLIPUDASE ALFA (RECOMBINANT HUMAN ACID SPHINGOMYELINASE) IN ADULTS WITH NIEMANN-PICK DISEASE TYPE B (ACID SPHINGOMYELINASE DEFICIENCY), 2 April 2015 (2015-04-02) *
WASSERSTEIN MELISSA P ET AL,, SUCCESSFUL WITHIN-PATIENT DOSE ESCALATION OF OLIPUDASE ALFA IN ACID SPHINGOMYELINASE DEFICIENCY, 30 May 2015 (2015-05-30) *

Also Published As

Publication number Publication date
CN118453843A (zh) 2024-08-09
JP2023052459A (ja) 2023-04-11
AU2018319565B2 (en) 2025-02-20
BR112020003541A2 (pt) 2020-09-01
WO2019038685A2 (en) 2019-02-28
MX2020002106A (es) 2020-07-14
AU2018319565A1 (en) 2020-04-09
NZ762860A (en) 2025-09-26
TWI791040B (zh) 2023-02-01
RU2020111649A (ru) 2021-09-24
JP2024050732A (ja) 2024-04-10
IL272757A (en) 2020-04-30
ES2970423T3 (es) 2024-05-28
IL272757B1 (en) 2024-03-01
US20240218338A1 (en) 2024-07-04
KR102769578B1 (ko) 2025-02-20
PL3672622T3 (pl) 2024-04-02
CA3073648A1 (en) 2019-02-28
JP7431356B2 (ja) 2024-02-14
EP3672622A2 (en) 2020-07-01
WO2019038685A3 (en) 2019-05-02
MX2023010908A (es) 2023-09-27
SG11202001544VA (en) 2020-03-30
JP2020531527A (ja) 2020-11-05
TW201919689A (zh) 2019-06-01
US20210139868A1 (en) 2021-05-13
JP7216075B2 (ja) 2023-01-31
EP3672622B1 (en) 2023-11-08
CN111344003B (zh) 2024-05-07
KR20250025051A (ko) 2025-02-20
CN111344003A (zh) 2020-06-26
AU2025203384A1 (en) 2025-05-29
KR20200044064A (ko) 2020-04-28
US11898175B2 (en) 2024-02-13
CO2020001801A2 (es) 2020-04-01

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