The U.S. Food and Drug Administration (FDA) has granted an orphan-drug designation to cobitolimod for the treatment of ulcerative colitis in pediatric patients.
Ulcerative colitis is a fairly common inflammatory bowel disease in adults, but in children it is extremely uncommon. In patients, inflammation and ulcers of the colon and rectum lead to symptoms such as abdominal pain, diarrhea, and bloody stools.
For children with mild ulcerative colitis, anti-inflammatory medications such sulfasalazine and mesalamine are advised. For more moderate to severe cases, corticosteroids and/or immunomodulatory agents are suggested.
In 2011, the FDA-approved Remicade (infliximab), an anti-tumor necrosis factor (TNF)-alpha antibody, for the treatment of children with ulcerative colitis.
Cobitolimod is a Toll-like receptor 9 (TLR9) agonist, that has anti‐inflammatory properties. In adults, cobitolimod is being tested in a Phase 2b study involving adults with moderate to severe ulcerative colitis. It is not currently known when a study will begin for testing the drug in children with ulcerative colitis.
The treatment is being developed by Index Pharmaceuticals, and the Orphan Drug Designation will provide the company with several tax and filing fee exemptions for the drug. Further, a pediatric rare disease indication may qualify the company to obtain a Pediatric Rare Disease Priority Review Voucher if the drug gets approved. Such vouchers can be used by the company to reduce the time to review a new drug application. The voucher can also be transferred to another company. Earlier this year, Sarepta
sold their voucher for $125 million to Gilead Sciences.
Below is a short video explaining ulcerative colitis:
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