Drug updated on 12/11/2024
| Dosage Form | Capsule (oral; 123 mg) |
| Drug Class | Alpha-galactosidase A pharmacological chaperones |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
Latest News
Summary
- This summary is based on the review of three systematic review(s)/meta-analysis(es). [1-3]
- The effectiveness of disease-specific therapies, including migalastat and enzyme replacement therapy (migalastat), on cardiovascular manifestations of Fabry disease remains unclear due to heterogeneity in study designs and outcomes. No conclusive evidence supports a significant impact on cardiovascular outcomes.
- In patients with gastrointestinal (GI) symptoms related to Fabry disease, migalastat is a primary treatment option, though its effectiveness is variable. Symptomatic relief is often required, as some patients do not respond adequately to enzyme replacement therapy.
- There is no safety information available in the reviewed studies.
- There is no population types or subgroups information available in the reviewed studies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Galafold (migalastat) Prescribing Information. | 2024 | Amicus Therapeutics, Inc., Princeton, NJ |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| A systematic literature review on the health-related quality of life and economic burden of Fabry disease | 2024 | Orphanet Journal of Rare Diseases |
| Disease-specific therapy for the treatment of the cardiovascular manifestations of Fabry disease: a systematic review | 2023 | Heart |
| Gastrointestinal Manifestations and Treatment Options in Fabry Disease Patients. A Systematic Review | 2022 | Journal of Gastrointestinal and Liver Diseases |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| A review and recommendations for oral chaperone therapy in adult patients with Fabry disease. | 2024 | Orphanet Journal of Rare Diseases |
| Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study. | 2023 | Frontiers in Medicine |
| An expert consensus on practical clinical recommendations and guidance for patients with classic Fabry disease | 2022 | Molecular Genetics and Metabolism |