Oral medication for Crohn's disease approved by FDA

Patients with moderate-to-severe Crohn’s disease now have a new treatment option to suppress their intestinal inflammation and help them maintain their relief.

Crohn’s disease, a chronic inflammatory bowel disorder affecting millions worldwide, has long presented a significant challenge for both patients and healthcare providers. Existing treatment options often fall short in achieving sustained remission and controlling symptoms. The results of a phase 3 trial now herald a promising breakthrough in managing this complex condition.

Upadacitinib — a breakthrough, once-daily oral medication — is a selective Janus kinase (JAK) inhibitor that precisely targets the underlying pathways of inflammation. It thereby mitigates symptoms and potentially minimises the risk of adverse events associated with immunosuppression.

The study spanned 43 countries and 277 sites, and enrolled a diverse group of patients 18 to 75 years old who had moderate-to-severe Crohn’s disease for at least three months. Participants were randomly assigned to receive 45 mg of upadacitinib or placebo (2:1) for induction therapy to assess its effectiveness in achieving remission in two double-blind phase 3 trials, U-EXCEL and U-EXCEED. The medication was taken once daily for 12 weeks.

Patients who had a clinical response to upadacitinib induction therapy were randomly assigned in the U-ENDURE maintenance trial to receive 15 mg or 30 mg of upadacitinib, or placebo (1:1:1) once daily for 52 weeks. The maintenance phase evaluated the drug’s ability to sustain remission and prevent relapse. (U-EXCEED had an additional 12-week open-label, active single-group induction period to allow for the accrual of enough patients for entry into U-ENDURE.)

Data from the phase 3 trial, published in The New England Journal of Medicine, showed that upadacitinib demonstrated superior efficacy over placebo, with a statistically significant proportion of patients achieving clinical remission after induction therapy. During the maintenance phase, the therapy substantially reduced the rate of disease relapse, illustrating its potential as a long-term treatment option for Crohn’s disease. As a result, upadacitinib garnered US Food and Drug Administration (FDA) approval on 18 May.

“Upadacitinib addresses an unmet need in the treatment of Crohn’s disease and allows patients access to an oral medication that could put them in clinical and endoscopic remission,” said corresponding author Dr Jean-Frederic Colombel, Co-Director of the Susan and Leonard Feinstein Inflammatory Bowel Disease Clinical Center at The Mount Sinai Hospital.

“This therapy has the potential to transform the lives of individuals battling this chronic condition and offers a new hope for improved quality of life.”

Image credit: iStock.com/Vachiravit Vasuponsritara