ES2970423T3 - Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida - Google Patents

Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida Download PDF

Info

Publication number
ES2970423T3
ES2970423T3 ES18768948T ES18768948T ES2970423T3 ES 2970423 T3 ES2970423 T3 ES 2970423T3 ES 18768948 T ES18768948 T ES 18768948T ES 18768948 T ES18768948 T ES 18768948T ES 2970423 T3 ES2970423 T3 ES 2970423T3
Authority
ES
Spain
Prior art keywords
patient
rhasm
doses
bone
dose
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
ES18768948T
Other languages
English (en)
Spanish (es)
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
Application granted granted Critical
Publication of ES2970423T3 publication Critical patent/ES2970423T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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)
ES18768948T 2017-08-24 2018-08-22 Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida Active ES2970423T3 (es)

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 (1)

Publication Number Publication Date
ES2970423T3 true ES2970423T3 (es) 2024-05-28

Family

ID=63556374

Family Applications (1)

Application Number Title Priority Date Filing Date
ES18768948T Active ES2970423T3 (es) 2017-08-24 2018-08-22 Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida

Country Status (16)

Country Link
US (2) US11898175B2 (https=)
EP (1) EP3672622B1 (https=)
JP (3) JP7216075B2 (https=)
KR (2) KR20250025051A (https=)
CN (2) CN111344003B (https=)
AU (2) AU2018319565B2 (https=)
BR (1) BR112020003541A2 (https=)
CA (1) CA3073648A1 (https=)
CO (1) CO2020001801A2 (https=)
ES (1) ES2970423T3 (https=)
IL (1) IL272757B2 (https=)
MX (2) MX2020002106A (https=)
PL (1) PL3672622T3 (https=)
SG (1) SG11202001544VA (https=)
TW (1) TWI791040B (https=)
WO (1) WO2019038685A2 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI3998078T1 (sl) * 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
KR20250025051A (ko) * 2017-08-24 2025-02-20 사노피 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료
PY1939780A (es) * 2018-05-25 2019-12-04 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida
EP3976218B1 (en) * 2019-05-31 2025-02-19 Genzyme Corporation Two-dimensional lc-ms/ms systems
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

Family Cites Families (8)

* 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
JP2008545752A (ja) * 2005-06-02 2008-12-18 テルシカ インコーポレーティッド 成長障害を処置するための方法
SI3998078T1 (sl) * 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
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
LT3004896T (lt) * 2013-06-07 2020-01-10 Genzyme Corporation Rūgštinės sfingomielinazės sutrikimų žymuo ir jo panaudojimas
KR20250025051A (ko) * 2017-08-24 2025-02-20 사노피 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료

Also Published As

Publication number Publication date
AU2018319565A1 (en) 2020-04-09
JP7216075B2 (ja) 2023-01-31
EP3672622B1 (en) 2023-11-08
JP2024050732A (ja) 2024-04-10
CN118453843A (zh) 2024-08-09
MX2023010908A (es) 2023-09-27
US11898175B2 (en) 2024-02-13
CA3073648A1 (en) 2019-02-28
KR102769578B1 (ko) 2025-02-20
EP3672622A2 (en) 2020-07-01
BR112020003541A2 (pt) 2020-09-01
IL272757A (en) 2020-04-30
NZ762860A (en) 2025-09-26
IL272757B1 (en) 2024-03-01
JP7431356B2 (ja) 2024-02-14
CN111344003A (zh) 2020-06-26
IL272757B2 (en) 2024-07-01
AU2018319565B2 (en) 2025-02-20
SG11202001544VA (en) 2020-03-30
TWI791040B (zh) 2023-02-01
US20210139868A1 (en) 2021-05-13
MX2020002106A (es) 2020-07-14
CN111344003B (zh) 2024-05-07
KR20200044064A (ko) 2020-04-28
TW201919689A (zh) 2019-06-01
PL3672622T3 (pl) 2024-04-02
WO2019038685A2 (en) 2019-02-28
RU2020111649A (ru) 2021-09-24
JP2023052459A (ja) 2023-04-11
AU2025203384A1 (en) 2025-05-29
JP2020531527A (ja) 2020-11-05
KR20250025051A (ko) 2025-02-20
WO2019038685A3 (en) 2019-05-02
US20240218338A1 (en) 2024-07-04
CO2020001801A2 (es) 2020-04-01

Similar Documents

Publication Publication Date Title
ES2970423T3 (es) Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida
Pogoryelova et al. GNE myopathy: from clinics and genetics to pathology and research strategies
Schuchman et al. Types A and B niemann-pick disease
AU2016216625B2 (en) Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
ES2751391T3 (es) Procedimientos de tratamiento de trastornos metabólicos
Rang et al. Re-imagining cystic fibrosis care: next generation thinking
Campistol et al. Glutaric aciduria type I: unusual biochemical presentation
Liu et al. Citrate: a key signalling molecule and therapeutic target for bone remodeling disorder
Sylvester et al. Report of the CCFA pediatric bone, growth and muscle health workshop, New York City, November 11–12, 2011, with updates
Ichikawa et al. High dietary phosphate intake induces development of ectopic calcifications in a murine model of familial tumoral calcinosis
Huang et al. Chronic hypoxia induces liver injury via PKCδ/ASMase-driven ceramide accumulation and endoplasmic reticulum stress in rats
CA3222399A1 (en) Nad-augmentation therapy for parkinson's disease
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
Caminiti et al. Nutritional assessment of a population with a history of childhood craniopharyngioma seen at Hospital ‘Prof. Dr. Juan P. Garrahan’
Snezhitskiy et al. From shadows to spotlight: how two cases redefined Barth syndrome awareness in Belarus: two case reports with a literature review
Laron et al. Recommended by ISPED and SIMA
Peschka et al. Annual Conference and Seminar–Arbeitsgemeinschaft für Pädiatrische Stoffwechselstörungen e. V.(APS)
Osterbauer Clinical and Preclinical Characterization of Skeletal Muscle Aging
WO2024228791A9 (en) Treating muscle weakness with alkaline phosphatases
Glorieux Emerging Concepts in Pediatric Bone Disease
Boubaya et al. Bone fragility in sarcoidosis and relationships with calcium metabolism disorders: a cross sectional study on 142 patients