FDA Approves Upadacitinib for Ulcerative Colitis

The decision represents the first ever upadacitinib for a gastroenterological disease.

The US Food and Drug Administration has approved upadacitinib (RINVOQ) as a treatment for adults with moderately to severely active ulcerative colitis.

The approval, awarded to AbbVie, represents a key decision for patients who have had an inadequate response or intolerance to 1 or more tumor necrosis factor (TNF) blockers.

The approval, which represents the first approval of upadacitinib in gastroenterology, is based on a trio of phase 3 randomized, double-blind, placebo-controlled clinical trials. The trials included the U-ACHIEVE and U-ACCOMPLISH induction studies in which the investigators tested upadacitinib 45 mg once daily for 8 weeks, followed by 15 mg or 30 mg once daily in the maintenance trial for 52 weeks.

The investigators found patients treated with the study drug achieved clinical remission more frequently through weeks 8 and 52 compared to placebo. The primary endpoint was stool frequency subscore (SFS) ≤ 1 and not greater than Baseline, rectal bleeding subscore (RBS) = 0, endoscopy subscore (ES) of ≤ 1 without friability

The drug also met all ranked secondary endpoints, including endoscopic improvement and histologic-endoscopic mucosal improvement (HEMI), as well as corticosteroid-free clinical remission in the maintenance study.

"Ulcerative colitis patients live with unpredictable symptoms such as increased stool frequency and bleeding, which can make daily activities difficult," said Maria T. Abreu, MD, Professor of Medicine, Professor of Microbiology and Immunology, University of Miami Miller School of Medicine and Director, Crohn's & Colitis Center, University of Miami Health System, in a statement. "In clinical trials, RINVOQ showed its ability to rapidly control symptoms in just eight weeks for many patients and sustained responses at one year. I believe these types of improvements can make a positive difference for my patients."