Ustekinumab Receives FDA Approval for Ulcerative Colitis

Adult patients with moderate to severely active ulcerative colitis have a new treatment option with the United States Food and Drug Administration(FDA)’s approval of ustekinumab(Stelara).

With the approval, which was awarded to Janssen Pharmaceutical, ustekinumab becomes the first and only approved treatment for ulcerative colitis to demonstrate improvement of the colon.

“The FDA approval of Stelara for UC represents an exciting milestone, offering patients a new option that has demonstrated improvement of the histology and endoscopic appearance of the intestinal lining, while also offering patients the potential for response and remission without the need for steroids,” said William Sandborn, MD, professor of medicine UC San Diego School of Medicine and clinical trial investigator.

Approval of ustekinumab was based on results of the phase 3 UNIFI clinical trial, which demonstrated the therapy’s ability to induce remission in more than 40% of patients at 1 year. The trial had a primary endpoint of clinical remission

The UNIFI trial consisted of an initial induction study and a maintenance study to assess the safety and efficacy of the treatment. During the induction study, patients received ustekinumab 6 mg/kg intravenously. Following the induction phase, patients were monitored for 8 weeks and received ustekinumab 90 mg subcutaneously every 8 weeks for 44 weeks.

Investigators observed 19% of patients receiving ustekinumab achieved clinical remission during the induction study. Investigators also noted ustekinumab provided rapid relief of symptoms in 58% of patients during the 8-week study.

At 1 year 45% of patients receiving ustekinumab were in remission. Additionally, 43% of patients treated with ustekinumab were in remission without receiving corticosteroids.

In regard to the treatment’s impact on intestinal lining, investigators noted improvements, which was measured by a novel histologic-endoscopic mucosal improvement endpoint, in the induction and maintenance studies. In the induction study, 17% reached the endpoint and that number climbed to 44% in the maintenance study.

The approval for moderate to severely active ulcerative colitis marks the fourth indication the treatment has received since 2009—with ustekinumab also receiving approvals for adolescent patients with moderate to severe plaque psoriasis, adults with active psoriatic arthritis, and adults with moderately to severely active Crohn’s disease.