Solid Biosciences has announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.
Obtaining ODD status for SGT-003, along with Fast Track Designation granted last month, furthers our efforts to meet the ongoing challenge of treating this devastating disease as expeditiously as possible. These designations are important milestones for Solid, supporting the continued development of next-generation therapies for Duchenne.”Bo Cumbo, President and Chief Executive Officer at Solid Biosciences
The company is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical trial sites for the planned Phase 1/2 clinical trial of SGT-003 and expects to commence patient screening shortly thereafter. This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single IV infusion of SGT-003 in participants with Duchenne muscular dystrophy. Currently, there are two clinical trial sites in the United States. Patient dosing in the trial is expected to commence in the mid-to-late first quarter of 2024.