NZ750752B2 - Treatment of fabry disease in ert-naïve and ert-experienced patients - Google Patents
Treatment of fabry disease in ert-naïve and ert-experienced patientsInfo
- Publication number
- NZ750752B2 NZ750752B2 NZ750752A NZ75075217A NZ750752B2 NZ 750752 B2 NZ750752 B2 NZ 750752B2 NZ 750752 A NZ750752 A NZ 750752A NZ 75075217 A NZ75075217 A NZ 75075217A NZ 750752 B2 NZ750752 B2 NZ 750752B2
- Authority
- NZ
- New Zealand
- Prior art keywords
- ert
- medicament
- administered
- lvmi
- months
- Prior art date
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
- A61K31/33—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
- A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
-
- 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
- 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
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P39/00—General protective or antinoxious agents
- A61P39/02—Antidotes
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
Abstract
Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-nave Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.
Claims (16)
1. Use of migalastat or salt f in the manufacture of a medicament for reducing left ventricular mass index (LVMi) and ameliorating proteinuria in a patient having Fabry disease 5 wherein the effective amount is about 123 mg free base equivalent (FBE) and the medicament is to be administered to the patient every other day.
2. The use of claim 1, wherein the patient has left ventricular hypertrophy (LVH) prior to initiating administration.
3. The use of claim 1 or 2, wherein the administration enhances a-galactosidase A 10 activity.
4. The use of any one of claims 1-3, wherein the effective amount of migalastat or salt thereof is about 123 mg of migalastat free base.
5. The use of any one of claims 1-3, n the effective amount of migalastat or salt thereof is about 150 mg of migalastat hydrochloride. 15
6. The use of any one of claims 1-5, wherein the medicament ses an oral dosage form.
7. The use of claim 6, wherein the oral dosage form comprises a tablet, a capsule or a solution.
8. The use of any one of claims 1-7, wherein the medicament is to be administered for at 20 least 18 months.
9. The use of any one of claims 1-8, wherein the medicament n is to be administered for at least 30 months.
10. The use of any one of claims 1-9, wherein the ment when administered provides an average decrease of LVMi in a group of perienced patients of at least about 5 g/m2 25 after 18 months of stration.
11. The use of any one of claims 1-10, wherein the medicament when administered provides an average decrease of LVMi in a group of ERT-experienced ts of about 6.6 g/m2 after 18 months of administration.
12. The use of any one of claims 1-11, wherein the medicament when administered es an average decrease of LVMi in a group of ERT-experienced patients of at least about 2 g/m2 after 30 months of administration.
13. The use of any one of claims 1-12, wherein the ment when administered 5 provides an average decrease of LVMi in a group of ERT-experienced patients of about 3.8 g/m2 after 30 months of administration.
14. The use of any one of claims 1-13, n the medicament when administered provides an average decrease of LVMi in a group of ERT-experienced patients with LVH of at least about 5 g/m2 after 30 months of administration. 10
15. The use of any one of claims 1-14, wherein the medicament when administered provides an average decrease of LVMi in a group of ERT-experienced patients with LVH of about 9 g/m2 after 30 months of stration.
16. The use of any one of claims 1-15, wherein the medicament when administered normalizes LVMi in a patient with LVH prior to initiating administration. cccttctgtaggggcagagaggttctacttcattactgcgtctcctgggaaggccatcag gactgctggctaaagtgggaaccaggactctttgtgagttaagaatttgtgtatttatat gtgtgttatacacattttttaaaaaactgtaacgacatcaggttgagcagtcgtctccgg gtggtgaattatgtgtatttttaaattttatactatattgttatttttcaaatgttcgaa attgaatatgtagattgttgttatcagcagaaaaataaacattattcaaatactctattc agtaaagtaatttattgggcgcctttgtcaagcacgcatttgcctagatgtgactctaca gataaaattcacttggggcctccccttacagacaatcaggcagtggagactgagtgcctg aatggatagaccagcactcagaccactattttcagtatctgtttttcttaactcagggcc ttcaaacgtttttcgccttacggtcacccttagggtcccccgagaccggcccag acagacagatatacaaaaacacatacacagtcatgagcgtccaccatttccccaccaggc caggcggcttcccggcactgagatgggggggaggagggagagagcgcgaggggg gaggggaaagcagagaacgaaagaggcggaggcggcccccgaaccccgctctggtcttca tcatcaccacccctgggtccccagttcccacccacacaccaacctctaacgataccgggt aattttcctccttcttccctcaaacggctatagcgagacggtagacgacgaccagaacta cttctgctcacgtaagcgagtaatcacgtgagcgcctacgtcatgtgagatctcggtcac aactctcggcttaaactcgggatcactaaggtgccgcacttccttctggtatgg ggcgggtcaatatcaagaaaggaagagggtgattggttagcggaacgtcttacg tgactgattattggtctacctctggggataaccgtcccagttgccagagaaacaataacg tcattatttaataagtcatcggtgattggtccgcccctgaggttaatcttaaaagcccag gttacccgcggaaatttatgctgtccggtcaccgtgacaatgcagctgaggaacccagaa ctacatctgggctgcgcgcttgcgcttcgcttcctggccctcgtttcctgggacatccct ggggctagagcactggacaatggattggcaaggacgcctaccatgggctggctgcactgg gagcgcttcatgtgcaaccttgactgccaggaagagccagattcctgcatcaggtatcag atattgggtactcccttccctttgcttttccatgtgtttgggtgtgtttggggaactgga gagtctcaacgggaacagttgagcccgagggagagctcccccacccgactctgctgctgc ttttttatccccagcaaactgtcccgaatcaggactagccctaaactttctctgtgtgac ctttcctgggatgggagtccggccagcggcccctgtttctttctctctctctctctctct cgttctccttctctttctctttctcttctttcctctctctttctctctctccctgcccgg ttctcttttttcactgctccttgcagagcagggccaccccataggcagtgtgcccaaagt agccctgcccggttctattcagacccttcttgtgaacttctgctcttcctctgccgggtg ctaaccgttagaacatctagggtgggtaggaggaatggggaactaagattcgtgccattt tttctccttttggggtcgtggatttctcggcagtatctcgagggagttagagagaccata aggtcgctgagatctctcccacctcgcccatgagcgtggcatcaggctggaaggttgaca tggaggaactttatacatttacacctttgcgtgagggttgaggctggattagataggtat tgaacatatctgaccctcacaatccttatctgtaaattgggattacaaccttttaatttc agggagctgacaaaaaaaatctgaaaaatagttcttatctcacacaggtgagttttcaag acctatttaaagtacatagcacagcgcttgaccattcaactgcgcttacagagc aaatgttcaatgggaaaatgaatgtaaatctacaaatctgaatgaatatgtgtatttttc tggagagaggatatttacctttcttcaaattctcaaagggctctgtgatttaaaaaaggt taggaatcactgatagatgttggtaaaaggtggcagtcacagtacatttctgtgtccata agttattcctatgaatatctttatagataaagtcaggatgttggtcagacatcacagaag aaattggccttgtaagtttcatgtgaccctgtggtacagtatgtgtggcaattttgccca tcacggatttttttttattggtatttgcatctgattataaaactaatgcatgatcattgc aaaaaatgtagataaagaagagcaaaatgaaaataaagatttccccccaccgttccacca cccagaaataatcatggtttaaatgttaatatacaaccttacaattgttttctatataaa tgaaaacatagatttctttatttcattattttccataaaaaatggatcatgtttatgtca tgtttggctaatggcaagaccctggcacccagtctgggctcaaattctgcctcattgtta cttagccctgtgacattgggtaaattacactttttttttttttttttttttgagacgggg tctcgctctgtcgcccaggctggagtgcagtggcacgatctcggctcactgcaagtccgc 2940 ctcctgggttcacgccattcttctgcctcagcctcccgagtagctgggactacaggcgcc 3000 tgccaccacgcctggctctttttttttttttttttttttttagtacagacggggtttcac 3060 catgttagccagggtggtctcaatctcctgacctcgtgattcgcccgcctcagcctccca 3
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| NZ790449A NZ790449B2 (en) | 2017-07-19 | Treatment of fabry disease in ert-naïve and ert-experienced patients |
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US15/213,920 US9999618B2 (en) | 2007-04-26 | 2016-07-19 | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| PCT/US2017/042872 WO2018017721A1 (en) | 2016-07-19 | 2017-07-19 | Treatment of fabry disease in ert-naïve and ert-experienced patients |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| NZ750752A NZ750752A (en) | 2025-07-25 |
| NZ750752B2 true NZ750752B2 (en) | 2025-10-29 |
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