AU2019217868A1 - Use of migalastat for treating fabry disease in pregnant patients - Google Patents
Use of migalastat for treating fabry disease in pregnant patients Download PDFInfo
- Publication number
- AU2019217868A1 AU2019217868A1 AU2019217868A AU2019217868A AU2019217868A1 AU 2019217868 A1 AU2019217868 A1 AU 2019217868A1 AU 2019217868 A AU2019217868 A AU 2019217868A AU 2019217868 A AU2019217868 A AU 2019217868A AU 2019217868 A1 AU2019217868 A1 AU 2019217868A1
- Authority
- AU
- Australia
- Prior art keywords
- patient
- migalastat
- pregnancy
- formulation
- treatment
- 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.)
- Abandoned
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Urology & Nephrology (AREA)
- Molecular Biology (AREA)
- Dermatology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2024278298A AU2024278298A1 (en) | 2018-02-06 | 2024-12-11 | Use of migalastat for treating fabry disease in pregnant patients |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862626955P | 2018-02-06 | 2018-02-06 | |
| US62/626,955 | 2018-02-06 | ||
| PCT/US2019/016841 WO2019157047A1 (en) | 2018-02-06 | 2019-02-06 | Use of migalastat for treating fabry disease in pregnant patients |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024278298A Division AU2024278298A1 (en) | 2018-02-06 | 2024-12-11 | Use of migalastat for treating fabry disease in pregnant patients |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2019217868A1 true AU2019217868A1 (en) | 2020-10-01 |
Family
ID=65494618
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019217868A Abandoned AU2019217868A1 (en) | 2018-02-06 | 2019-02-06 | Use of migalastat for treating fabry disease in pregnant patients |
| AU2024278298A Pending AU2024278298A1 (en) | 2018-02-06 | 2024-12-11 | Use of migalastat for treating fabry disease in pregnant patients |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024278298A Pending AU2024278298A1 (en) | 2018-02-06 | 2024-12-11 | Use of migalastat for treating fabry disease in pregnant patients |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11357784B2 (enExample) |
| EP (2) | EP4717319A3 (enExample) |
| JP (2) | JP7555818B2 (enExample) |
| KR (1) | KR20200128676A (enExample) |
| CN (1) | CN111971044A (enExample) |
| AU (2) | AU2019217868A1 (enExample) |
| CA (1) | CA3090496C (enExample) |
| ES (1) | ES3065184T3 (enExample) |
| WO (1) | WO2019157047A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX351004B (es) | 2006-05-16 | 2017-09-26 | Nat Institutes Of Health | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry. |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| AU2018277756A1 (en) | 2017-05-30 | 2020-01-23 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| EP4717319A3 (en) | 2018-02-06 | 2026-06-10 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pregnant patients |
| LT3840753T (lt) | 2018-08-20 | 2024-11-11 | Amicus Therapeutics, Inc. | Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją |
| KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| JP5270161B2 (ja) * | 2004-09-24 | 2013-08-21 | ベス イスラエル デアコネス メディカル センター | 妊娠合併症を診断および処置する方法 |
| PL2027137T3 (pl) | 2005-06-08 | 2015-08-31 | Amicus Therapeutics Inc | Oczyszczanie imino- i aminocukrów |
| MX351004B (es) | 2006-05-16 | 2017-09-26 | Nat Institutes Of Health | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry. |
| EP2142197A4 (en) | 2007-03-30 | 2010-11-10 | Amicus Therapeutics Inc | METHOD FOR THE TREATMENT OF MORBUS FABRY WITH PHARMACOLOGICAL CHAPTERONES |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| AU2008245578A1 (en) | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| US8321148B2 (en) | 2008-10-24 | 2012-11-27 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| US9206457B2 (en) | 2009-05-26 | 2015-12-08 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
| EP2502068B1 (en) | 2009-11-17 | 2016-02-10 | Baylor Research Institute | Urinary triaosylceramide (gb3) as a marker of cardiac disease |
| WO2012125402A2 (en) | 2011-03-11 | 2012-09-20 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of fabry disease |
| US10155027B2 (en) | 2012-07-17 | 2018-12-18 | Amicus Therapeutics, Inc. | Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease |
| EP2874648A4 (en) | 2012-07-17 | 2015-12-30 | Amicus Therapeutics Inc | ALPHA GALACTOSIDASE A AND 1-DEOXYGALACTONO JIRIMYCIN CO FORMULATION |
| EP3432882B1 (en) | 2016-03-22 | 2021-09-01 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
| WO2018017721A1 (en) | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| US11357834B2 (en) | 2017-01-10 | 2022-06-14 | Amicus Therapeutics, Inc. | Recombinant α-galactosidase A for treatment of Fabry disease |
| AU2018277756A1 (en) | 2017-05-30 | 2020-01-23 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| WO2019046244A1 (en) | 2017-08-28 | 2019-03-07 | Amicus Therapeutics, Inc. | METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE |
| AU2019217603A1 (en) | 2018-02-06 | 2020-09-24 | Amicus Therapeutics, Inc. | Treatment of patients with classic Fabry disease |
| EP4717319A3 (en) | 2018-02-06 | 2026-06-10 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pregnant patients |
| LT3840753T (lt) | 2018-08-20 | 2024-11-11 | Amicus Therapeutics, Inc. | Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją |
| US20220313670A1 (en) | 2019-06-11 | 2022-10-06 | Amicus Therapeutics, Inc. | Methods Of Treating Fabry Disease In Patients Having Renal Impairment |
| KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
| HRP20260399T1 (hr) | 2020-02-10 | 2026-05-08 | Amicus Therapeutics, Inc. | Postupci liječenja fabryjeve bolesti |
-
2019
- 2019-02-06 EP EP25221383.0A patent/EP4717319A3/en active Pending
- 2019-02-06 WO PCT/US2019/016841 patent/WO2019157047A1/en not_active Ceased
- 2019-02-06 EP EP19706178.1A patent/EP3749307B1/en active Active
- 2019-02-06 KR KR1020207025691A patent/KR20200128676A/ko active Pending
- 2019-02-06 US US16/967,827 patent/US11357784B2/en active Active
- 2019-02-06 ES ES19706178T patent/ES3065184T3/es active Active
- 2019-02-06 CN CN201980024425.7A patent/CN111971044A/zh active Pending
- 2019-02-06 AU AU2019217868A patent/AU2019217868A1/en not_active Abandoned
- 2019-02-06 JP JP2020542621A patent/JP7555818B2/ja active Active
- 2019-02-06 CA CA3090496A patent/CA3090496C/en active Active
-
2022
- 2022-06-13 US US17/838,820 patent/US20220387462A1/en active Pending
-
2024
- 2024-07-12 JP JP2024112288A patent/JP2024161361A/ja active Pending
- 2024-12-11 AU AU2024278298A patent/AU2024278298A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US11357784B2 (en) | 2022-06-14 |
| JP2024161361A (ja) | 2024-11-19 |
| KR20200128676A (ko) | 2020-11-16 |
| CA3090496C (en) | 2024-03-26 |
| EP4717319A3 (en) | 2026-06-10 |
| WO2019157047A1 (en) | 2019-08-15 |
| CN111971044A (zh) | 2020-11-20 |
| JP7555818B2 (ja) | 2024-09-25 |
| EP3749307A1 (en) | 2020-12-16 |
| US20210038624A1 (en) | 2021-02-11 |
| CA3090496A1 (en) | 2019-08-15 |
| EP4717319A2 (en) | 2026-04-01 |
| AU2024278298A1 (en) | 2025-01-09 |
| EP3749307B1 (en) | 2025-12-10 |
| ES3065184T3 (en) | 2026-05-04 |
| US20220387462A1 (en) | 2022-12-08 |
| JP2021512906A (ja) | 2021-05-20 |
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| WO2019046244A1 (en) | METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE | |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| MK5 | Application lapsed section 142(2)(e) - patent request and compl. specification not accepted |