Rare Disease Report

Orphan Drug Designation Granted to Pediatric Ulcerative Colitis Therapy

SEPTEMBER 08, 2017
Mathew Shanley
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to INN-108, Innovate Biopharmaceuticals’ in-development therapy for pediatric ulcerative colitis.

INN-108 is a novel, oral small molecule comprised of mesalamine (5-ASA) and 4aminophenylacetic acid (4-AAA), 2 drugs that are currently-approved agent for ulcerative colitis and the immunomodulatory agent approved in Japan for rheumatoid arthritis, respectively.  

While ulcerative colitis is a common inflammatory bowel disease in adults, it is extremely rare in children. Patients experience inflammation and ulcers in the colon and rectum, resulting in symptoms like abdominal pain, diarrhea and bloody stools.

Currently, more than 80% of people with mild-to-moderate ulcerative colitis are being treated with anti-inflammatory medications like mesalamine, but in more severe cases, corticosteroids and/or immunomodulatory agents are recommended. Despite the high failure rate of treatment with 5-ASA, there aren’t many alternatives besides steroids before patients are treated with expensive anti-TNF biologics like AbbVie's Humira and J&J's Remicade.

INN-108 has successfully completed a Phase 1 clinical trial in the U.S. including 24 mild-to-moderate ulcerative colitis patients. Phase 2 trials are expected to start in early 2018. Innovate is also developing a liquid oral formulation of the drug that will ease administration to children.

The Orphan Drug Designation will provide Innovate with several exemptions for taxes and filing fees for the drug, and a pediatric rare disease indication could qualify the company to obtain a Pediatric Rare Disease Priority Review Voucher, should the INN-108 get approved.

Priority Review Vouchers can be used by the company to reduce the time to review a new drug application, and can be transferred to another company. Earlier this year, Sarepta sold one such voucher to Gilead Sciences for $125-million.

In August, the FDA granted the same designation to Index Pharmaceuticals’ cobitolimod, and in 2011, approved Remicade (infliximab), an anti-tumor necrosis factor (TNF)-alpha antibody, for the treatment of children with mild ulcerative colitis.

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