FDA Grants Orphan Drug Designation to Pediatric Crohn's Disease Treatment

The FDA has granted orphan drug designation to AbbVie for risankizumab (ABBV-066) for the treatment of Crohn’s disease in pediatric patients. In addition the drug is being evaluated for the treatment of other immunological disorders, including psoriasis, psoriatic arthritis, and asthma.

Orphan drug designation is granted to products that could potentially treat, diagnose, or prevent a rare disease or disorder that affects less than 200,000 people in the United States. In a study conducted by Boehringer Ingelheim, the most common adverse effects associated with risankizumab were nausea, abdominal pain, vomiting, anemia, and headache. Together, Boehringer Ingelheim and AbbVie are investigating the development and commercialization of the drug.

"AbbVie is committed to providing innovative treatment options to patients, particularly in areas with unmet medical needs such as Crohn's disease in children — a disease that may impact physical and social development," said Michael Severino, MD, executive vice president of research and development and chief scientific officer of AbbVie, in a press release. "This designation reinforces the need for additional treatment options for young people living with this sometimes debilitating disease.”

Trial results released earlier in 2016 showed that risankizumab achieved higher rates of clinical response than a placebo when tested in patients with Crohn’s disease. Furthermore, there were numerically fewer severe or serious adverse events associated with risankizumab compared to the placebo.