2355Treatment of cardiac manifestations in Fabry disease with the oral drug Migalastat: First 12 months results from a cohort of amenable all-comers

Muentze, J.; Gensler, Daniel; Salinger, Tim; Oder, Daniel; Wanner, Christoph; Frantz, Stefan; Nordbeck, Peter

doi:10.1093/eurheartj/ehy565.2355

Cited by 2 publications

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“…The efficacy of migalastat in the real-world setting has been evaluated in an ongoing prospective single-centre study in patients with migalastat-amenable mutations of α-galactosidase A [45]. Initial follow-up after 3–6 months’ treatment in 17 evaluable patients suggested beneficial effects of migalastat on cardiac morphology, with significant reductions from baseline in the myocardial mass index (from 129.38 to 119.88 g/m 2 ; p = 0.02).…”

Section: Therapeutic Efficacy Of Migalastatmentioning

confidence: 99%

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Migalastat: A Review in Fabry Disease

McCafferty

Scott

2019

Drugs

141

105

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Fabry disease is a rare lysosomal disorder characterized by deficient or absent α-galactosidase A activity resulting from mutations in the GLA gene. Migalastat (Galafold™), a pharmacological chaperone, stabilizes and facilitates trafficking of amenable mutant forms of α-galactosidase A enzyme from the endoplasmic reticulum to lysosomes and increases its lysosomal activity. Oral migalastat is the first pharmacological chaperone approved for treating patients [aged ≥ 18 years (USA and Canada) or ≥ 16 years in other countries] with Fabry disease who have a migalastat-amenable GLA mutation. In the FACETS trial in enzyme replacement therapy (ERT)-naive patients with GLA mutations amenable or non-amenable to migalastat, there was no significant difference between the migalastat and placebo groups for the proportion of patients achieving a ≥ 50% reduction in the number of globotriaosylceramide (GL-3) inclusions/kidney interstitial capillary (KIC) at 6 months [primary endpoint; intent-to-treat (ITT) population]. In the modified ITT population (i.e. patients with migalastat-amenable GLA mutations), relative to placebo, migalastat treatment significantly reduced the mean number of GL-3 inclusions/KIC and plasma lyso-globotriaosylsphingosine levels at 6 months. Among evaluable patients, migalastat maintained renal function and reduced cardiac mass after ≤ 24 months’ therapy. In the ATTRACT trial in ERT-experienced patients, renal function was maintained during 18 months of migalastat or ERT; however, migalastat significantly reduced cardiac mass compared with ERT. Migalastat was generally well tolerated in both of these trials. Given its convenient oral regimen and the limited therapeutic options available, migalastat is an important treatment option for Fabry disease in patients with migalastat-amenable GLA mutations.

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Section: Therapeutic Efficacy Of Migalastatmentioning

confidence: 99%

“…Lyso-Gb 3 levels in leukocytes did not alter to a significant degree after 3–6 or 12 months of treatment. Renal function was maintained after 3-6 months and GFR CKD-EPI did not significantly change after 12 months of migalastat treatment [45].…”

Section: Therapeutic Efficacy Of Migalastatmentioning

confidence: 99%