FDA Orphan Drug and Rare Pediatric Disease designations for tegacorat

German family-owned drugmaker Grünenthal today announced that its investigational compound tegacorat (GRM-01) received Orphan Drug and Rare Pediatric Disease designations from the US Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD).

Tegacorat, a non-steroidal selective glucocorticoid receptor agonist and modulator (SEGRAM), is an orally available investigational compound being developed to provide an alternative to glucocorticoid-based treatments such as prednisone, the current standard of care in DMD.