InDex secures FDA orphan-drug designation for cobitolimod

"We are pleased that the FDA has granted orphan-drug designation for cobitolimod for treatment of ulcerative colitis in children, which may provide seven years of market exclusivity in this indication on the US market," said Peter Zerhouni, CEO of InDex Pharmaceuticals.

"The decision shows the medical need for new therapeutic options for this patient population and gives InDex additional opportunities to interact with the FDA."

Cobitolimod is a new type of drug that can help patients with moderate to severe ulcerative colitis back to a normal life. It is a so-called Toll-like receptor 9 (TLR9) agonist, that can provide an anti-inflammatory effect locally in the large intestine, which may induce mucosal healing and relief of the clinical symptoms in ulcerative colitis.

Cobitolimod has achieved clinical proof-of-concept in moderate to severe active ulcerative colitis, with a very favorable safety profile.

Data from four placebo-controlled clinical trials indicate that cobitolimod has statistically significant effects on those endpoints that are most relevant in this disease, both from a regulatory and clinical perspective. These endpoints include the key clinical symptoms such as blood in stool, number of stools, and mucosal healing, respectively.

Cobitolimod is also known as Kappaproct and DIMS0150.