AU2018319565B2 - Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients - Google Patents

Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients Download PDF

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Publication number
AU2018319565B2
AU2018319565B2 AU2018319565A AU2018319565A AU2018319565B2 AU 2018319565 B2 AU2018319565 B2 AU 2018319565B2 AU 2018319565 A AU2018319565 A AU 2018319565A AU 2018319565 A AU2018319565 A AU 2018319565A AU 2018319565 B2 AU2018319565 B2 AU 2018319565B2
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AU2018319565A1 (en
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Ana Cristina SCHEIDT-PUGA
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Genzyme Corp
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Genzyme Corp
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    • 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)

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  • 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2018319565A 2017-08-24 2018-08-22 Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients Active AU2018319565B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025203384A AU2025203384A1 (en) 2017-08-24 2025-05-12 Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762549732P 2017-08-24 2017-08-24
US62/549,732 2017-08-24
EP17306720 2017-12-07
EP17306720.8 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2025203384A Division AU2025203384A1 (en) 2017-08-24 2025-05-12 Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients

Publications (2)

Publication Number Publication Date
AU2018319565A1 AU2018319565A1 (en) 2020-04-09
AU2018319565B2 true AU2018319565B2 (en) 2025-02-20

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AU2018319565A Active AU2018319565B2 (en) 2017-08-24 2018-08-22 Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients
AU2025203384A Pending AU2025203384A1 (en) 2017-08-24 2025-05-12 Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients

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AU2025203384A Pending AU2025203384A1 (en) 2017-08-24 2025-05-12 Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients

Country Status (17)

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US (2) US11898175B2 (cg-RX-API-DMAC7.html)
EP (1) EP3672622B1 (cg-RX-API-DMAC7.html)
JP (3) JP7216075B2 (cg-RX-API-DMAC7.html)
KR (2) KR102769578B1 (cg-RX-API-DMAC7.html)
CN (2) CN118453843A (cg-RX-API-DMAC7.html)
AU (2) AU2018319565B2 (cg-RX-API-DMAC7.html)
BR (1) BR112020003541A2 (cg-RX-API-DMAC7.html)
CA (1) CA3073648A1 (cg-RX-API-DMAC7.html)
CO (1) CO2020001801A2 (cg-RX-API-DMAC7.html)
ES (1) ES2970423T3 (cg-RX-API-DMAC7.html)
IL (1) IL272757B2 (cg-RX-API-DMAC7.html)
MX (2) MX2020002106A (cg-RX-API-DMAC7.html)
NZ (1) NZ762860A (cg-RX-API-DMAC7.html)
PL (1) PL3672622T3 (cg-RX-API-DMAC7.html)
SG (1) SG11202001544VA (cg-RX-API-DMAC7.html)
TW (1) TWI791040B (cg-RX-API-DMAC7.html)
WO (1) WO2019038685A2 (cg-RX-API-DMAC7.html)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2470199T3 (pl) * 2009-08-28 2019-11-29 Icahn School Med Mount Sinai Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy
UY38238A (es) * 2018-05-25 2019-12-31 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

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 (6)

* 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
EP1901770B1 (en) * 2005-06-02 2014-12-24 Ipsen Biopharmaceuticals, Inc. Gh and igf-1 for treatment of growth disorders
CN104519905A (zh) 2012-03-02 2015-04-15 夏尔人类遗传性治疗公司 用于治疗iii型戈谢病的组合物和方法
EP2931298A4 (en) 2012-12-12 2016-07-06 Teva Pharma FUSION OF HUMAN GROWTH HORMONES AND ALBUMIN, AND FORMULATION THEREOF AND USE THEREOF
EP3004896B1 (en) 2013-06-07 2019-09-18 Genzyme Corporation Marker for acid sphingomyelinase disorders and uses thereof

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 (1)

* Cited by examiner, † Cited by third party
Title
WASSERSTEIN M. P., et al., 2015, "Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency", Molecular Genetics and Metabolism, 116(1), pages 88-97 *

Also Published As

Publication number Publication date
JP2020531527A (ja) 2020-11-05
EP3672622A2 (en) 2020-07-01
JP7216075B2 (ja) 2023-01-31
ES2970423T3 (es) 2024-05-28
IL272757B1 (en) 2024-03-01
TWI791040B (zh) 2023-02-01
CN111344003A (zh) 2020-06-26
KR20250025051A (ko) 2025-02-20
SG11202001544VA (en) 2020-03-30
NZ762860A (en) 2025-09-26
WO2019038685A2 (en) 2019-02-28
WO2019038685A3 (en) 2019-05-02
MX2020002106A (es) 2020-07-14
IL272757B2 (en) 2024-07-01
EP3672622B1 (en) 2023-11-08
MX2023010908A (es) 2023-09-27
CN118453843A (zh) 2024-08-09
KR20200044064A (ko) 2020-04-28
AU2025203384A1 (en) 2025-05-29
US20240218338A1 (en) 2024-07-04
BR112020003541A2 (pt) 2020-09-01
JP2023052459A (ja) 2023-04-11
KR102769578B1 (ko) 2025-02-20
JP2024050732A (ja) 2024-04-10
CN111344003B (zh) 2024-05-07
US20210139868A1 (en) 2021-05-13
RU2020111649A (ru) 2021-09-24
CO2020001801A2 (es) 2020-04-01
IL272757A (en) 2020-04-30
AU2018319565A1 (en) 2020-04-09
TW201919689A (zh) 2019-06-01
CA3073648A1 (en) 2019-02-28
PL3672622T3 (pl) 2024-04-02
US11898175B2 (en) 2024-02-13
JP7431356B2 (ja) 2024-02-14

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