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

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric 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)
  • 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)
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 (17)

Country Link
US (2) US11898175B2 (enExample)
EP (1) EP3672622B1 (enExample)
JP (3) JP7216075B2 (enExample)
KR (2) KR20250025051A (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)
NZ (1) NZ762860A (enExample)
PL (1) PL3672622T3 (enExample)
SG (1) SG11202001544VA (enExample)
TW (1) TWI791040B (enExample)
WO (1) WO2019038685A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102094253B1 (ko) * 2009-08-28 2020-03-31 이칸 스쿨 오브 메디슨 엣 마운트 시나이 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
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
WO2006130769A2 (en) * 2005-06-02 2006-12-07 Tercica, Inc. Methods for treatment of growth disorders
HK1207005A1 (en) 2012-03-02 2016-01-22 夏尔人类遗传性治疗公司 Compositions and methods for treating type iii gaucher disease
WO2014093354A1 (en) 2012-12-12 2014-06-19 Teva Pharmaceutical Industries Ltd. Fusion of human growth hormone and albumin, formulation and uses thereof
PL3004896T3 (pl) 2013-06-07 2020-04-30 Genzyme Corporation Marker zaburzeń kwaśnej spfingomielinazy i jego zastosowania

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

Similar Documents

Publication Publication Date Title
US20240218338A1 (en) Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients
Marcucci et al. Gaucher disease and bone manifestations
Schuchman et al. Types A and B niemann-pick disease
von Bismarck et al. Improved pulmonary function by acid sphingomyelinase inhibition in a newborn piglet lavage model
Wang et al. Synergistic protection of bone vasculature and bone mass by desferrioxamine in osteoporotic mice
Langeveld et al. Bone health in patients with inborn errors of metabolism
Campistol et al. Glutaric aciduria type I: unusual biochemical presentation
Paccou et al. Cystic fibrosis-related bone disease
Hashemieh et al. Prevalence of osteoporosis among thalassemia patients from Zafar adult thalassemia clinic, Iran
Conway et al. A cross-sectional study of bone mineral density in children and adolescents attending a Cystic Fibrosis Centre
Wesolek et al. Assessment of progranulin and FAM19A5 protein blood levels in patients with metabolic syndrome
RU2795570C2 (ru) Лечение патологических состояний кости у пациентов с дефицитом кислой сфингомиелиназы
Kasapkara et al. The evaluation of skeletal manifestations in patients with Gaucher disease
WO2022173987A1 (en) Alkaline phosphatase polypeptides and methods of use thereof
Lee et al. Clinical characteristics and effects of enzyme replacement therapy with elosulfase alfa in Korean patients with mucopolysaccharidosis type IVA
Elkin Arthritis, vasculitis and bone disease
Zhu et al. Serum myostatin levels are elevated in overweight patients
Wang et al. A study on the nutritional status and body composition of children with Hutchinson–Gilford progeria syndrome
Manoli et al. Osteoporosis in frail patients
Laron et al. Recommended by ISPED and SIMA
MISHRA KRISHNANU DUTTA¹, TRISHNA DAS* 1, DIPANKAR SAHA¹, TAPASH CHAKRABORTY¹
Özay et al. Bone Mineral Density and Lipid Metabolism After Alendronate and Strontium Ranelate Treatment
Kubaski Diagnosis and therapies for mucopolysaccharidoses
Van Coster et al. P301 Mitochondrial abnormalities in a newborn with lactic acidosis and adrenal calcifications