Idorsia Advances Fabry Disease Program With Encouraging Phase 3 Results

(RTTNews) - Idorsia Ltd (IDIA.SW) announced the publication of pivotal Phase 3 MODIFY study results and its open-label extension (OLE) in Nature Communications. The study evaluated lucerastat, an oral substrate reduction therapy, in adults with Fabry disease. Findings reinforce lucerastat's potential to address unmet needs, particularly in patients with renal impairment.

The MODIFY study (NCT03425539) was a multicenter, double-blind, randomized, placebo-controlled trial involving 118 patients across 14 countries. Participants were randomized in a 2:1 ratio to receive either lucerastat or placebo. At the end of the double-blind period, 107 patients entered the OLE (NCT03737214) to assess long-term safety, tolerability, and efficacy on renal function.

Although the study did not meet its primary endpoint of reducing neuropathic pain over six months, lucerastat demonstrated a strong pharmacodynamic effect. Plasma and urinary Gb3 levels were significantly reduced compared to placebo, and these reductions were sustained in the OLE. Patients who switched from placebo to lucerastat showed similar biomarker improvements.

An interim analysis of the OLE revealed a notable improvement in renal outcomes. Patients treated with lucerastat for at least 12 months experienced a reduction in the rate of eGFR decline compared to the two years prior to enrollment. In those with impaired renal function or rapidly deteriorating eGFR at baseline, lucerastat was associated with a marked attenuation of kidney function loss, suggesting a potential disease-modifying effect. Cardiac function also remained stable, with no worsening of the left ventricular mass index.

Long-term data from the OLE, with patients treated for up to 42 months and some for over six years, confirmed lucerastat's favorable safety profile, with no treatment-related serious adverse events. A kidney biopsy sub-study further supported lucerastat's impact, showing encouraging results in male patients with classic Fabry disease treated for more than three years.

These findings have informed the design of a new Phase 3 program, with Idorsia working closely with the US FDA to define the optimal regulatory pathway. As the OLE study concludes, Idorsia is establishing a post-trial access program to ensure continuity of care for participants still receiving lucerastat.

IDIA.SW closed trading on the SIX Swiss Exchange at CHF 3.7750 on January 9, 2026. The share price declined by CHF 0.2850, representing a 7.02% drop compared to the previous session.

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