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Idorsia shows 42-month lucerastat renal outcomes in Fabry disease at WORLDSymposium 2026
Idorsia Ltd announces that the results of lucerastat treatment over 42-months and the kidney biopsy data will be presented at the 22nd Annual WORLDSymposium taking place from February 2-6, 2026.
The following two posters will be presented on February 5, 15:30 PST (February 6, 00:30 CET):
- Lucerastat, an investigational oral substrate reduction therapy in Fabry disease: Kidney biopsy results from the MODIFY open-label extension study (#125, Kiosk 14-B)
- Lucerastat effect on kidney function in patients with Fabry disease: Results from the ongoing open label-extension (OLE) of the phase 3 clinical program (#361. Kiosk 39-B)
The posters present long-term results from the Phase 3 MODIFY study and its open-label extension when ongoing participants had received lucerastat for at least 42 months, with some exceeding six years of continuous therapy. These data provide important insight into the durability of lucerastat’s clinical effects and tolerability over extended treatment.
Idorsia also conducted a kidney biopsy sub-study within the open-label extension, enrolling male patients with classic Fabry disease who had received at least two years of lucerastat monotherapy. This analysis evaluated Gb3 accumulation in key kidney cell types using established quantitative methods, generating data that may help further characterize lucerastat’s potential impact on renal disease biology.
About Fabry disease
Fabry disease is a rare, X-linked lysosomal storage disorder caused by mutations in the GLA gene, resulting in deficient or absent activity of the enzyme α-galactosidase A (α-Gal A). This enzymatic deficiency leads to the accumulation of globotriaosylceramide (Gb3) and its derivatives in cells throughout the body. Over time, this buildup causes progressive damage across multiple organ systems, including the kidneys, heart, nervous system, skin, eyes, and gastrointestinal tract.
The disease manifests in two main phenotypes: classic Fabry disease, typically presenting in childhood with severe, multisystemic involvement, and late-onset Fabry disease, which may emerge in adulthood with predominant cardiac or renal symptoms. Due to its variable presentation and non-specific symptoms, Fabry disease is frequently underdiagnosed or misdiagnosed, leading to delays in treatment and increased risk of irreversible organ damage.
