FDA Grants Orphan Designation to InDex’s Treatment for Pediatric Ulcerative Colitis

InDex Pharmaceuticals received an Orphan Drug designation for its cobitolimod treatment for pediatric ulcerative colitis. Cobitolimod is also known as Kappaproct and DIMS0150.

Cobitolimod is a Toll-like receptor 9 agonist, which provides an anti-inflammatory effect in the large intestine, and possibly induces mucosal healing and relief of clinical symptoms, the company said.

Cobitolimod achieved clinical proof-of-concept in moderate to severe active ulcerative colitis, with a favorable safety profile. Four placebo-controlled clinical trials demonstrated cobitolimod’s effects on those endpoints including blood in stool, number of stools and mucosal healing.