CO2020001801A2 - Tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida - Google Patents
Tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácidaInfo
- Publication number
- CO2020001801A2 CO2020001801A2 CONC2020/0001801A CO2020001801A CO2020001801A2 CO 2020001801 A2 CO2020001801 A2 CO 2020001801A2 CO 2020001801 A CO2020001801 A CO 2020001801A CO 2020001801 A2 CO2020001801 A2 CO 2020001801A2
- Authority
- CO
- Colombia
- Prior art keywords
- patients
- acid sphingomyelinase
- treatment
- abnormal bone
- sphingomyelinase deficiency
- Prior art date
Links
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 title abstract 3
- 230000002159 abnormal effect Effects 0.000 title abstract 2
- 210000000988 bone and bone Anatomy 0.000 title abstract 2
- 101000785978 Homo sapiens Sphingomyelin phosphodiesterase Proteins 0.000 abstract 1
- 208000029725 Metabolic bone disease Diseases 0.000 abstract 1
- 210000001185 bone marrow Anatomy 0.000 abstract 1
- 102000048866 human SMPD1 Human genes 0.000 abstract 1
- 108010092496 olipudase alfa Proteins 0.000 abstract 1
- 229950010922 olipudase alfa Drugs 0.000 abstract 1
- 231100001055 skeletal defect Toxicity 0.000 abstract 1
Classifications
-
- 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)
-
- 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
- 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)
- General Health & Medical Sciences (AREA)
- Physical Education & Sports Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Rheumatology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Epidemiology (AREA)
- Obesity (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (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)
Abstract
La invención proporciona métodos de uso de una esfingomielinasa ácida humana (p. ej. olipudasa alfa) en el tratamiento de una afección ósea anormal en pacientes con deficiencia de esfingomielinasa ácida, tales como baja densidad ósea, alta carga de médula ósea y otras anomalías esqueléticas que se presentan en pacientes con deficiencia de esfingomielinasa ácida.
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 |
|---|---|
| CO2020001801A2 true CO2020001801A2 (es) | 2020-04-01 |
Family
ID=63556374
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2020/0001801A CO2020001801A2 (es) | 2017-08-24 | 2020-02-18 | Tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US11898175B2 (es) |
| EP (1) | EP3672622B1 (es) |
| JP (3) | JP7216075B2 (es) |
| KR (1) | KR20200044064A (es) |
| CN (2) | CN111344003B (es) |
| AU (1) | AU2018319565A1 (es) |
| BR (1) | BR112020003541A2 (es) |
| CA (1) | CA3073648A1 (es) |
| CO (1) | CO2020001801A2 (es) |
| ES (1) | ES2970423T3 (es) |
| IL (1) | IL272757B2 (es) |
| MX (2) | MX2020002106A (es) |
| PL (1) | PL3672622T3 (es) |
| SG (1) | SG11202001544VA (es) |
| TW (1) | TWI791040B (es) |
| WO (1) | WO2019038685A2 (es) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT2470199T (lt) * | 2009-08-28 | 2019-10-10 | Icahn School Of Medicine At Mount Sinai | Pakaitinė fermento terapija su dozės didinimu, skirta rūgštinės sfingomielinazės nepakankamumo gydymui |
| UY38238A (es) * | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| CA3140183A1 (en) * | 2019-05-31 | 2020-12-03 | 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 (7)
| 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 |
| LT2470199T (lt) * | 2009-08-28 | 2019-10-10 | Icahn School Of Medicine At Mount Sinai | Pakaitinė fermento terapija su dozės didinimu, skirta rūgštinės sfingomielinazės nepakankamumo gydymui |
| JP6230160B2 (ja) | 2012-03-02 | 2017-11-15 | シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド | Ｉｉｉ型ゴーシェ病を治療するための組成物および方法 |
| CA2892626A1 (en) | 2012-12-12 | 2014-06-19 | Teva Pharmaceutical Industries Ltd. | Fusion of human growth hormone and albumin, formulation and uses thereof |
| IL284102B2 (en) | 2013-06-07 | 2023-04-01 | Genzyme Corp | A marker for disorders of acid sphingomyelinase and its uses |
-
2018
- 2018-08-22 EP EP18768948.4A patent/EP3672622B1/en active Active
- 2018-08-22 ES ES18768948T patent/ES2970423T3/es active Active
- 2018-08-22 CN CN201880067498.XA patent/CN111344003B/zh active Active
- 2018-08-22 KR KR1020207008213A patent/KR20200044064A/ko not_active Application Discontinuation
- 2018-08-22 JP JP2020511325A patent/JP7216075B2/ja active Active
- 2018-08-22 CN CN202410508209.2A patent/CN118453843A/zh active Pending
- 2018-08-22 TW TW107129257A patent/TWI791040B/zh active
- 2018-08-22 BR BR112020003541-6A patent/BR112020003541A2/pt unknown
- 2018-08-22 PL PL18768948.4T patent/PL3672622T3/pl unknown
- 2018-08-22 MX MX2020002106A patent/MX2020002106A/es unknown
- 2018-08-22 WO PCT/IB2018/056346 patent/WO2019038685A2/en active Application Filing
- 2018-08-22 US US16/641,116 patent/US11898175B2/en active Active
- 2018-08-22 CA CA3073648A patent/CA3073648A1/en active Pending
- 2018-08-22 AU AU2018319565A patent/AU2018319565A1/en active Pending
- 2018-08-22 SG SG11202001544VA patent/SG11202001544VA/en unknown
- 2018-08-22 IL IL272757A patent/IL272757B2/en unknown
-
2020
- 2020-02-18 CO CONC2020/0001801A patent/CO2020001801A2/es unknown
- 2020-02-24 MX MX2023010908A patent/MX2023010908A/es unknown
-
2023
- 2023-01-18 JP JP2023005602A patent/JP7431356B2/ja active Active
-
2024
- 2024-01-10 US US18/409,535 patent/US20240218338A1/en active Pending
- 2024-01-31 JP JP2024012534A patent/JP2024050732A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| BR112020003541A2 (pt) | 2020-09-01 |
| US11898175B2 (en) | 2024-02-13 |
| WO2019038685A2 (en) | 2019-02-28 |
| IL272757A (en) | 2020-04-30 |
| TWI791040B (zh) | 2023-02-01 |
| JP7216075B2 (ja) | 2023-01-31 |
| RU2020111649A (ru) | 2021-09-24 |
| IL272757B2 (en) | 2024-07-01 |
| JP2024050732A (ja) | 2024-04-10 |
| CA3073648A1 (en) | 2019-02-28 |
| CN111344003A (zh) | 2020-06-26 |
| TW201919689A (zh) | 2019-06-01 |
| MX2020002106A (es) | 2020-07-14 |
| CN118453843A (zh) | 2024-08-09 |
| JP7431356B2 (ja) | 2024-02-14 |
| AU2018319565A1 (en) | 2020-04-09 |
| MX2023010908A (es) | 2023-09-27 |
| US20240218338A1 (en) | 2024-07-04 |
| EP3672622B1 (en) | 2023-11-08 |
| WO2019038685A3 (en) | 2019-05-02 |
| EP3672622A2 (en) | 2020-07-01 |
| ES2970423T3 (es) | 2024-05-28 |
| IL272757B1 (en) | 2024-03-01 |
| KR20200044064A (ko) | 2020-04-28 |
| US20210139868A1 (en) | 2021-05-13 |
| JP2020531527A (ja) | 2020-11-05 |
| CN111344003B (zh) | 2024-05-07 |
| JP2023052459A (ja) | 2023-04-11 |
| SG11202001544VA (en) | 2020-03-30 |
| PL3672622T3 (pl) | 2024-04-02 |
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