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

Info

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
Authority
AU
Australia
Prior art keywords
patient
leu
pro
ala
gly
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2018319565A
Other languages
English (en)
Other versions
AU2018319565A1 (en
Inventor
Ana Cristina SCHEIDT-PUGA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
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 AU2018319565A1 publication Critical patent/AU2018319565A1/en
Assigned to GENZYME CORPORATION reassignment GENZYME CORPORATION Request for Assignment Assignors: SANOFI
Application granted granted Critical
Publication of AU2018319565B2 publication Critical patent/AU2018319565B2/en
Priority to AU2025203384A priority Critical patent/AU2025203384A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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)
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

Family

ID=63556374

Family Applications (2)

Application Number Title Priority Date Filing Date
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

Family Applications After (1)

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

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 (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
CN118453843A (zh) 2024-08-09
JP2023052459A (ja) 2023-04-11
BR112020003541A2 (pt) 2020-09-01
WO2019038685A2 (en) 2019-02-28
MX2020002106A (es) 2020-07-14
AU2018319565A1 (en) 2020-04-09
IL272757B2 (en) 2024-07-01
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

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
RU2731616C2 (ru) Ферментная заместительная терапия с увеличением дозы для лечения недостаточности кислой сфингомиелиназы
Sabir et al. The evolving therapeutic landscape of genetic skeletal disorders
Liu et al. Elevated plasma calcifediol is associated with aggressive periodontitis
Hashemieh et al. Prevalence of osteoporosis among thalassemia patients from Zafar adult thalassemia clinic, Iran
Scott Asfotase alfa in perinatal/infantile-onset and juvenile-onset hypophosphatasia: a guide to its use in the USA
US20240382568A1 (en) Alkaline phosphatase polypeptides and methods of use thereof
Andiran et al. Cyclic pamidronate therapy in children with osteogenesis imperfecta: results of treatment and follow-up after discontinuation.
RU2795570C2 (ru) Лечение патологических состояний кости у пациентов с дефицитом кислой сфингомиелиназы
BR122025022093A2 (pt) Uso de uma esfingomielinase ácida humana recombinante (rhasm) no tratamento de condições ósseas anormais em pacientes com deficiência de esfingomielinase ácida
Negri et al. Bone densitometry in a patient with hypophosphatemic osteomalacia
Elkin Arthritis, vasculitis and bone disease
Younes et al. Serum Uric Acid Level Is Positively Associated With Higher Bone Mineral Density at Multiple Skeletal Sites Among Healthy Qataris
Wang et al. A study on the nutritional status and body composition of children with Hutchinson–Gilford progeria syndrome
Lang et al. Metabolic masqueraders of paediatric and adult rheumatic diseases
Sarantis et al. A new COL1A1 mutation in a Greek patient with osteogenesis imperfecta: Response to a low-dose protocol of zoledronic acid and two-year follow-up
Laron et al. Recommended by ISPED and SIMA
Hagiwara et al. FRI0472 COMPARISON OF THE THERAPEUTIC EFFICACY OF DAILY VERSUS WEEKLY DOSE OF TERIPARATIDE FOR PREVENTING BONE FRACTURE IN PATIENTS WITH OSTEOPOROSIS–A SINGLE CENTRE, PROSPECTIVE OPEN-LABEL STUDY
Misra et al. Obesity and Metabolic Syndrome
BR122024026309A2 (pt) Alfa-glicosidase ácida humana recombinante

Legal Events

Date Code Title Description
PC1 Assignment before grant (sect. 113)

Owner name: GENZYME CORPORATION

Free format text: FORMER APPLICANT(S): SANOFI

FGA Letters patent sealed or granted (standard patent)