Experimental Pediatric Ulcerative Colitis Drug Picks Up Orphan Drug Status

Experimental Pediatric Ulcerative Colitis Drug Picks Up Orphan Drug Status

shutterstock_168768398AvaxiaBiologics,Inc. recently announced that the US Food and Drug Administration (FDA) granted their lead experimental drug AVX-470 Orphan Drug Designation for the treatment of pediatric ulcerative colitis. The news comes after the company previously announced the results of their Phase 1b clinical trial for AVX-470.

Ulcerative colitis (one of the two most common types of Inflammatory Bowel Disease) is characterized by inflammation of the large intestine. The symptoms for UC include abdominal cramping, diarrhea, and bloody stools, with approximately 25% of the cases occurring in young people who are less than 20 years old. The disease impacts normal childhood development due to deficient dietary intake accompanied with impaired absorption and enteric blood loss occurring at a key stage of childhood development.

AVX-470 is the first specific gut-targeted anti-TNF antibody being developed as a therapeutic for ulcerative colitis clinical applications that can be administered orally, thus offering a significant advantage when compared to other anti-TNF drugs available to treat intestinal bowel disease that need to be injected. Moreover, many curren UC treatments have been shown to cause serious side-effects, such as risk of infection due to their negative impact on the bodies’ immune system, since they do not target the gut specifically. Thus, although this type of treatment is very advantageous in the early stages of the disease, their side effects make them only a second or third-line therapeutical choice. AvaxiaBiologics,Inc. expects that AVX-470 efficacy and gut-specificity will establish this drug as first line of treatment for UC patients.

Barbara S. Fox, Ph.D., Chief Executive Officer commented, “We are pleased to receive Orphan Drug Designation for AVX-470 for treatment of pediatric ulcerative colitis. The drawbacks of current anti-TNF drugs are especially problematic in children. These drugs are injected or infused and may have serious side effects caused by systemic immunosuppression. As a gut-targeted antibody drug, AVX-470 avoids these drawbacks and may allow use of anti-TNF therapy earlier in the treatment of pediatric UC, with the goal of improving patient outcomes.”


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