Innovate Biopharmaceuticals recently submitted an application to the U.S. Food and Drug Administration (FDA) seeking orphan drug designation for its potential pediatric ulcerative colitis therapy, INN-108.

The FDA issues orphan drug designation to promising treatments for rare diseases affecting fewer than 200,000 people per year in the U.S. The FDA offers incentives including tax credits for clinical testing, seven years of market exclusivity, fast-tracking for regulatory proceedings, and exemption from prescription drug user fees.

“Orphan drug designation would expedite our development of a therapy that we believe could bring relief to children and teenagers suffering from active ulcerative colitis,” Chris Prior, PhD, Innovate’s CEO, said in a press release.

“There is an unmet need for an effective treatment for this disease and INN-108 is a promising potential therapy. Our FDA submission is a major milestone toward making this therapy available,” Prior added.

Ulcerative colitis is an inflammatory bowel disease that causes long-lasting inflammation and ulcers in the large intestine or colon. The disease typically causes constant diarrhea, often with blood, as well as abdominal and rectal pain, and increased bowel movements. In severe cases, patients experience weight loss and fatigue.

The disease often develops in teenagers and young adults, with estimates of the number of pediatric ulcerative colitis cases in the U.S. ranging from 13,700 to 87,600. The disease more commonly occurs in children and teenagers ages 10 to 17. Complications in children include nutritional deficiencies, slowed growth and development, anemia, and psychological problems.

Current treatment of mild to moderate forms of the disease, which affect more than 80% of ulcerative colitis patients, uses mesalamine, or 5-aminosalicylic acid (5-ASA). However, with failure rates for 5-ASA treatments ranging from 40-50%, new therapies for ulcerative colitis are needed, Innovate says.

INN-108 is a small molecule containing two components: mesalamine/5-ASA and 4-aminophenylacetic acid, an immunomodulatory drug approved in Japan for rheumatoid arthritis. Preliminary data show the combination could be a more efficient treatment than mesalamine/5-ASA alone.

INN-108 has successfully completed two Phase 1 clinical trials in the U.S. for mild to moderate ulcerative colitis in healthy adults and in patients with ulcerative colitis. The drug will begin a Phase 2 trial in 2018. Innovate is developing a liquid oral form of INN-108 for better use in pediatrics.

Also, pending shareholder approval, Innovate will merge with high-performance electronics marketer Monster Digital. The company is expected to begin Phase 3 clinical trials for its late-stage potential therapy for celiac disease in late 2017.