The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation, to SRK-015 for the treatment of SMA.
Scholar Rock initiated a Phase 2 proof-of-concept clinical trial, TOPAZ, to assess the safety and efficacy of SRK-015 in patients with Type 2 and Type 3 SMA. Enrolment was completed in January 2020.
The trial includes three cohorts, each representing a distinct subpopulation of patients:
More information