The U.S. Food and Drug Administration (FDA) has approved Stelara (ustekinumab) for the treatment of adults with ulcerative colitis (UC) that is moderately to severely active, Janssen Pharmaceuticals, Stelara’s developer and part of Johnson & Johnson, announced.

The FDA’s decision follows the approval by the European Commission in September for the same condition.

“Because of the individual nature of ulcerative colitis, what works for one patient may not work for another. That is why it is so critical that our ulcerative colitis patients have many different treatment options available to them,” Caren Heller, MD, MBA, chief scientific officer at the Crohn’s & Colitis Foundation, said in a press release.

“The approval of STELARA is extremely important for patients living with moderate to severe ulcerative colitis. STELARA gives patients another option to, hopefully, induce remission and help manage their disease,” Heller added.

Stelara is a biologic agent that works by inhibiting the activity of two pro-inflammatory proteins, interleukin (IL)-12 and IL-23. The levels of these proteins are abnormally high in patients with inflammatory bowel disease (IBD).

The FDA’s decision was based on the results from the multicenter, randomized, double-blind and placebo-controlled Phase 3 UNIFI trial (NCT02407236). These results were published in the New England Journal of Medicine, in a study titled “Ustekinumab as Induction and Maintenance Therapy for Ulcerative Colitis.”

The study, which enrolled a total of 961 patients, was divided into two phases — an induction part followed by a maintenance phase. In the induction phase, participants were given a single dose of Stelara — delivered as an intravenous or into the vein (IV) infusion at a dose of 130 mg (320 patients) or 6 mg per kilogram (322 patients) — to assess their response to treatment for at least eight weeks. A total of 319 patients received placebo.

In the maintenance phase, those who had a response in the eight-week induction phase were then treated for a total of 44 weeks. These participants were randomly assigned to receive 90 mg doses of Stelara, delivered subcutaneuously (under the skin), every 12 weeks (172 patients), or every 8 weeks (176 patients), or a placebo (175 patients).

The trial aimed to assess the safety and efficacy of Stelara, compared with a placebo, in people with moderate-to-severe UC who weren’t responding to other therapies — including corticosteroids and Entyvio — or who were intolerant to those therapies.

The primary endpoint in the induction phase and the maintenance phase was clinical remission, as defined by the participants’ scores on the Mayo scale. Scores on the Mayo scale range from 0 to 12, with higher scores indicating more severe disease. For this trial, the endpoint was a total score of equal to or less than 2 on the Mayo scale, and no subscore higher than 1 — from a range of 0 to 3 — on any of the four Mayo scale components.

The results showed that patients given Stelara responded favorably to treatment — with the positive responses already detected in the induction phase. In that first phase, a significantly higher proportion of patients given Stelara attained clinical remission — 15.6% for the 130 mg, and 15.5% for the 6mg per kg — compared with those given placebo (5.3%). Moreover, during this phase, 17% of the participants treated with Stelara showed mucosal healing during endoscopy.

In the maintenance phase, depending on how often they received the medication, 38.4% to 43.8% of patients treated with Stelara achieved remission, compared with 24% in the placebo group. At one year, 43% of the Stelara-treated patients were in clinical remission without any steroids, and 44% showed mucosal healing.

Stelara is the first and only approved UC treatment to cause improvements to the intestinal mucosa as shown by endoscopy analysis.

Stelara was generally safe and well-tolerated by patients, with the incidence of serious adverse events similar to the placebo group. After one year, there were two deaths among the Stelara group — one due to acute respiratory distress syndrome and esophageal varices bleeding, or rupture of large veins in the esophagus — and seven cases of cancer. The placebo group had no deaths and one case of cancer.

“Ulcerative colitis is a chronic and progressive disease that can have a significant impact on patients, often disrupting their day-to-day lives with frequent and urgent needs for bowel movements that can be accompanied by pain and cramping,” said William J. Sandborn, MD, chief of the division of gastroenterology and professor of medicine at UC San Diego School of Medicine.

“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,” added Sandborn, one of the study investigators.

Stelara had been approved for four conditions, including moderate-to-severe Crohn’s disease, psoriasis, and psoriatic arthritis, in both Europe and the U.S.

“At Janssen, we have a longstanding commitment to developing innovative new options that can help address the unmet treatment needs for those living with immune-mediated diseases,” said David M. Lee, MD, PhD, therapeutic area head for immunology at Janssen Research & Development.

“With today’s milestone, Stelara has received its fifth FDA approval since 2009, a testament to our unwavering focus on delivering treatments for patients who have limited therapeutic options,” he added.

The FDA-approved regime follows a weight-based, one-time IV infusion induction dose schedule. This is followed by a maintenance dosing schedule of a 90 mg, given through a subcutaneous (under the skin) injection every eight weeks.

Janssen says it will work with payers, providers, and pharmacies to ensure that Stelara is accessible and affordable for people with ulcerative colitis.