FDA Grants Orphan Drug Status to AbbVie’s Therapy for Pediatric Crohn’s Disease

FDA Grants Orphan Drug Status to AbbVie’s Therapy for Pediatric Crohn’s Disease

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to AbbVie’s investigational drug risankizumab for the treatment of pediatric patients with Crohn’s disease.

Risankizumab (ABBV-066) selectively blocks IL-23, a key protein involved in inflammatory processes that have been linked to a number of chronic immune-mediated diseases.

Risankizumab is part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading the drug’s development and commercialization worldwide. The therapeutic potential of risankizumab is being evaluated in immunological disorders, including Crohn’s disease, psoriasis, and psoriatic arthritis.

“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,” Michael Severino, MD, AbbVie’s executive vice president of research and development and chief scientific officer, said in a press release.

“This designation reinforces the need for additional treatment options for young people living with this sometimes debilitating disease. Our expertise in immunology provides a unique perspective and we look forward to continuing to evaluate risankizumab as a potential new therapy for patients,” he said.

The FDA Orphan Drug designation provides certain incentives to drug companies for medications intended for the treatment, diagnosis or prevention of rare diseases.

Earlier this year, results from a Phase 2 clinical trial (NCT02031276) evaluating risankizumab’s effectiveness, safety and drug properties showed that the investigational drug was more effective than a placebo in patients with moderate to severely active Crohn’s disease.

In the trial, patients were randomly treated for 12 weeks either with a placebo or risankizumab. At 12 weeks, 24 percent of patients treated with 200 mg of risankizumab, and 37 percent treated with 600 mg were symptom-free and in remission, as defined by the Crohn’s Disease Activity Index of less than 150 or a reduction from baseline of at least 100 points, compared to placebo-treated patients.

The study, “IL-23 Inhibitor Risankizumab Induces Remission in Phase II Study in Patients with Moderate-to-Severe Crohn’s Disease,” was presented during the Digestive Disease Week Conference in San Diego, California.

Crohn’s disease, a type of inflammatory bowel disease (IBD) that affects the gastrointestinal tract, can cause diarrhea, abdominal pain, rectal bleeding, and weight loss.