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 apitegromab for the treatment of SMA.
Scholar Rock initiated a Phase 2 proof-of-concept clinical trial, TOPAZ, to assess the safety and efficacy of apitegromab 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:
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