On 9 August, the US Food and Drug Administration (FDA) granted InDex Pharmaceutical’s Kappaproct (cobitolimod) orphan drug designation for the treatment of ulcerative colitis (UC) in pediatric patients.

This news comes one month after the Swedish drug maker managed to secure a new patent in Europe for Kappaproct for the additional protection to treat inflammatory diseases.  

UC is a chronic inflammatory disease of the colon or large intestine that usually initially manifests in the terminal part of the colon (rectum) and extends either to the left part of the colon or to the entire colon as the disease progresses.

According to GlobalData, there were more than 241,000 prevalent cases of UC globally in 2015 for patients aged 0-19 years, with the US comprising approximately 60% of cases. The current first-line therapy for pediatric UC is mesalamine.

In the US, if a child fails to respond to mesalamine or is diagnosed as being a moderate-to-severe case, physicians are likely to prescribe corticosteroids to induce remission, in combination with either a thiopurine immunomodulator or Janssen’s Remicade (infliximab) to maintain remission.

However, efficacy and safety concerns involving the use of corticosteroids as a maintenance therapy remain, stemming mostly from side effects such as hyperglycemia, hypertension, and growth failure. There are also many adverse reactions associated with thiopurine immunomodulators, such as myelosuppression, hepatotoxicity, and flu-like illness in children, while Remicade has a boxed warning for serious infections and malignancy.

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Kappaproct is under development to treat patients who suffer from moderate-to-severe UC. The drug acts as an immunomodulatory agent and functions similarly to azathioprine, relieving clinical symptoms such as hematochezia, frequent stools, and inflammation. The drug currently boasts a promising safety profile due to its limited systemic absorption, setting it in contrast to conventional immunomodulatory agents.

In the Phase III COLLECT study, Kappaproct demonstrated a strong safety profile in comparison with placebo, marking a substantial opportunity for InDex to fill a large unmet need in the pediatric UC space.

As an orphan drug, Kappaproct will gain seven years of market exclusivity if approved for the treatment of pediatric UC. InDex may also receive tax credits and waivers for application fees. Although Kappaproct did not achieve its primary efficacy endpoint in the Phase III COLLECT study, Kappaproct’s promising safety profile could be crucial for pediatric sufferers of UC.

With an upcoming Phase IIb clinical trial, InDex has another opportunity to demonstrate efficacy in an area of unmet need. Kappaproct is currently the only drug being developed for treatment of active UC. Combined with the high safety profile, this presents an opportunity for Kappaproct to compete with anti-tumour necrosis factor (TNF) drugs and conventional immunomodulators as a better rescue therapy for these patients.