AbbVie gets positive EMA committee opinion for Upadacitinib to treat adults with moderate to severe Crohn's disease

"The recent CHMP recommendation to approve upadacitinib for use in Crohn's disease is a momentous step, bringing us closer to offering a first-of-its-kind, once-daily oral treatment that can make a difference for people living with this disease," said Roopal Thakkar, M.D., senior vice president, development, regulatory affairs and chief medical officer, AbbVie. "We remain steadfast in our commitment to researching and developing treatment options as part of a diverse portfolio of therapies for those living with inflammatory bowel diseases."

AbbVie's application for the approval of upadacitinib in Crohn's disease is supported by data from two induction studies, U-EXCEED and U-EXCEL, and one maintenance study, U-ENDURE. Patients receiving upadacitinib were treated with 45 mg once daily for the induction studies, and were randomized to receive either 15 mg or 30 mg once-daily doses for the maintenance study. Across all three Phase 3 studies, a significantly greater proportion of patients treated with upadacitinib achieved the co-primary endpoints of clinical remission per SF/AP (defined as average daily stool frequency [SF] ≤2.8 and abdominal pain [AP] score ≤1.0 and neither greater than baseline) and endoscopic response (defined as decrease in simple endoscopic score for Crohn's disease [SES-CD] >50% from baseline of the induction) compared to placebo.

In all three studies, a statistically significant greater proportion of patients treated with upadacitinib achieved the key secondary endpoint of endoscopic remission (defined as SES-CD ≤4 and at least a 2-point reduction vs. baseline and no subscore >1). Additionally, more upadacitinib-treated patients achieved SES-CD ulcerated surface subscore of 0 at weeks 12 and 52 (nominal p-value<0.001) in patients with SES-CD ulcerated surface subscore ≥1 at baseline. Absence or disappearance of ulceration coupled with improvements seen by endoscopy are associated with mucosal healing.

The safety profile of upadacitinib in Crohn's disease was generally consistent with the known safety profile of upadacitinib.

"The impact of Crohn's disease extends beyond the gut to include systemic symptoms such as fatigue, bowel symptoms and social and emotional functioning. Treatment options that achieve critical endpoints such as clinical remission and endoscopic response can make a difference in managing the challenging symptoms of this condition and health-related outcomes related to quality of life," said Jean-Frédéric Colombel, M.D., professor of medicine and director of Inflammatory Bowel Disease Center, Icahn School of Medicine, Mount Sinai and study investigator. "Upadacitinib could be a promising treatment option for patients who live with uncontrolled moderate to severe Crohn's disease. I look forward to the European Commission's final decision."

RINVOQ is approved in the EU for the treatment of adults with radiographic axial spondylarthritis, non-radiographic axial spondylarthritis, psoriatic arthritis, rheumatoid arthritis, moderately to severely active ulcerative colitis and adults and adolescents with atopic dermatitis.

Use of upadacitinib in Crohn's disease is approved in Great Britain as of January 2023. Its safety and efficacy remain under evaluation in the European Union.

Crohn's disease is a chronic, systemic disease that manifests as inflammation within the gastrointestinal tract, causing persistent diarrhea and abdominal pain. It is a progressive disease, meaning it gets worse over time in a substantial proportion of patients or may develop complications that require urgent medical care, including surgery. Because the signs and symptoms of Crohn's disease are unpredictable, it causes a significant burden on people living with the disease—not only physically, but also emotionally and economically.