New oral therapy for Fabry patients in development

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New oral therapy for Fabry patients in development

Idorsia Pharmaceuticals, a Swiss biotech company, is developing lucerastat, a new oral therapy for patients with Fabry Disease. The Phase 3 clinical study MODIFY has just enrolled its first patients and will investigate the use of lucerastat monotherapy for the treatment of adult patients with Fabry disease, irrespective of their genetic mutation type.

The MODIFY study will enrol patients having Fabry neuropathic pain despite long-term use of enzyme replacement therapy (ERT) and willing to discontinue ERT for the duration of the study, as well as patients having Fabry neuropathic pain while not receiving ERT. Following a screening period of 6-7 weeks, patients enrolled into MODIFY will receive lucerastat treatment or placebo for six months before being offered the opportunity to enter an open label extension study. The primary endpoint of the study is a reduction in neuropathic pain, which Fabry patients report as significantly impacting their daily activities and quality of life, despite existing treatments.

If you are an adult patient with Fabry disease and have neuropathic pain, and you are interested in participating, please contact your doctor or contact a clinical site near you.

More information on MODIFY including a list of clinical trial sites where the study will take place can be found on here.

Visit the Fabry Disease page or download a factsheet to find out more about the disease and its symptoms.