NZ790449B2 - 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
- NZ790449B2 NZ790449B2 NZ790449A NZ79044917A NZ790449B2 NZ 790449 B2 NZ790449 B2 NZ 790449B2 NZ 790449 A NZ790449 A NZ 790449A NZ 79044917 A NZ79044917 A NZ 79044917A NZ 790449 B2 NZ790449 B2 NZ 790449B2
- Authority
- NZ
- New Zealand
- Prior art keywords
- medicament
- ert
- migalastat
- patient
- administered
- 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
formulation comprising an effective amount of migalastat or salt thereof for use in reducing left ventricular mass index (LVMi) in an enzyme replacement therapy (ERT)-experienced patient having Fabry disease, and/or for use in reducing globotriaosylceramide (GL-3) inclusion volume per podocyte and ameliorating proteinuria in a patient having Fabry disease. The effective amount is about 123 mg free base equivalent (FBE) and the formulation is administered to the patient every other day.
Claims (12)
1. Use of migalastat or salt thereof in the manufacture of a medicament for reducing globotriaosylceramide (GL-3) ion volume per podocyte and ameliorating nuria in a 5 patient having Fabry disease, wherein the effective amount is about 123 mg free base equivalent (FBE) and the medicament is to be stered to the patient every other day.
2. The use of claim 1, wherein the patient is an ERT-naïve patient.
3. The use of claim 1 or 2, wherein the administration enhances a-galactosidase A 10
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, wherein the effective amount of migalastat or salt thereof is about 150 mg of migalastat hydrochloride.
6. The use of any one of claims 1-5, wherein the medicament comprises an oral dosage 15 form.
7. The use of claim 6, wherein the oral dosage form ses 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 least 18 months. 20
9. The use of any one of claims 1-8, wherein the medicament is to be administered for at least 30 months.
10. The use of any one of claims 1-9, wherein the medicament when administered es an average reduction of globotriaosylceramide (GL-3) inclusion volume per podocyte in a group of ERT-naïve patients of at least about 30% after 6 months of stration of 25 migalastat or a salt thereof.
11. The use of any one of claims 1-10, wherein the medicament when administered reduces plasma globotriaosylsphingosine (Iyso-Gb3).
12. The use of any one of claims 1-11, n the medicament when administered improves renal function. cccttctgtaggggcagagaggttctacttcattactgcgtctcctgggaaggccatcag gactgctggctaaagtgggaaccaggactctttgtgagttaagaatttgtgtatttatat gtgtgttatacacattttttaaaaaactgtaacgacatcaggttgagcagtcgtctccgg gtggtgaattatgtgtatttttaaattttatactatattgttatttttcaaatgttcgaa attgaatatgtagattgttgttatcagcagaaaaataaacattattcaaatactctattc agtaaagtaatttattgggcgcctttgtcaagcacgcatttgcctagatgtgactctaca attcacttggggcctccccttacagacaatcaggcagtggagactgagtgcctg aatggatagaccagcactcagaccactattttcagtatctgtttttcttaactcagggcc gtggttttcaaacgtttttcgccttacggtcacccttagggtcccccgagaccggcccag acagacagatatacaaaaacacatacacagtcatgagcgtccaccatttccccaccaggc gcagcacaggcggcttcccggcactgagatgggggggaggagggagagagcgcgaggggg gaggggaaagcagagaacgaaagaggcggaggcggcccccgaaccccgctctggtcttca tcatcaccacccctgggtccccagttcccacccacacaccaacctctaacgataccgggt cctccttcttccctcaaacggctatagcgagacggtagacgacgaccagaacta cttctgctcacgtaagcgagtaatcacgtgagcgcctacgtcatgtgagatctcggtcac gtgagcaactctcggcttaaactcgggatcactaaggtgccgcacttccttctggtatgg aaatagggcgggtcaatatcaagaaaggaagagggtgattggttagcggaacgtcttacg tgactgattattggtctacctctggggataaccgtcccagttgccagagaaacaataacg tcattatttaataagtcatcggtgattggtccgcccctgaggttaatcttaaaagcccag cgcggaaatttatgctgtccggtcaccgtgacaatgcagctgaggaacccagaa ctacatctgggctgcgcgcttgcgcttcgcttcctggccctcgtttcctgggacatccct ggggctagagcactggacaatggattggcaaggacgcctaccatgggctggctgcactgg gagcgcttcatgtgcaaccttgactgccaggaagagccagattcctgcatcaggtatcag atattgggtactcccttccctttgcttttccatgtgtttgggtgtgtttggggaactgga gagtctcaacgggaacagttgagcccgagggagagctcccccacccgactctgctgctgc ttttttatccccagcaaactgtcccgaatcaggactagccctaaactttctctgtgtgac ctttcctgggatgggagtccggccagcggcccctgtttctttctctctctctctctctct cgttctccttctctttctctttctcttctttcctctctctttctctctctccctgcccgg ttctcttttttcactgctccttgcagagcagggccaccccataggcagtgtgcccaaagt agccctgcccggttctattcagacccttcttgtgaacttctgctcttcctctgccgggtg gttagaacatctagggtgggtaggaggaatggggaactaagattcgtgccattt tttctccttttggggtcgtggatttctcggcagtatctcgagggagttagagagaccata aggtcgctgagatctctcccacctcgcccatgagcgtggcatcaggctggaaggttgaca tggaggaactttatacatttacacctttgcgtgagggttgaggctggattagataggtat tgaacatatctgaccctcacaatccttatctgtaaattgggattacaaccttttaatttc agggagctgacaaaaaaaatctgaaaaatagttcttatctcacacaggtgagttttcaag gagataacctatttaaagtacatagcacagcgcttgaccattcaactgcgcttacagagc aaatgttcaatgggaaaatgaatgtaaatctacaaatctgaatgaatatgtgtatttttc tggagagaggatatttacctttcttcaaattctcaaagggctctgtgatttaaaaaaggt taggaatcactgatagatgttggtaaaaggtggcagtcacagtacatttctgtgtccata agttattcctatgaatatctttatagataaagtcaggatgttggtcagacatcacagaag gccttgtaagtttcatgtgaccctgtggtacagtatgtgtggcaattttgccca tcacggatttttttttattggtatttgcatctgattataaaactaatgcatgatcattgc aaaaaatgtagataaagaagagcaaaatgaaaataaagatttccccccaccgttccacca cccagaaataatcatggtttaaatgttaatatacaaccttacaattgttttctatataaa tgaaaacatagatttctttatttcattattttccataaaaaatggatcatgtttatgtca tgtttggctaatggcaagaccctggcacccagtctgggctcaaattctgcctcattgtta cttagccctgtgacattgggtaaattacactttttttttttttttttttttgagacgggg tctgtcgcccaggctggagtgcagtggcacgatctcggctcactgcaagtccgc 2940 ctcctgggttcacgccattcttctgcctcagcctcccgagtagctgggactacaggcgcc 3000 tgccaccacgcctggctctttttttttttttttttttttttagtacagacggggtttcac 3060 catgttagccagggtggtctcaatctcctgacctcgtgattcgcccgcctcagcctccca 3
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 |
| NZ750752A NZ750752B2 (en) | 2017-07-19 | Treatment of fabry disease in ert-naïve and ert-experienced patients |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| NZ790449A NZ790449A (en) | 2025-08-29 |
| NZ790449B2 true NZ790449B2 (en) | 2025-12-02 |
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