Janssen Announces FDA Approval of STELARA (ustekinumab) for the Treatment of Adults with Moderately to Severely Active Ulcerative Colitis

The Janssen Pharmaceutical Companies of Johnson & Johnson announced the U.S. Food and Drug Administration’s (FDA) approval of STELARA® (ustekinumab) for the treatment of adult patients with moderately to severely active ulcerative colitis. The approval for this new indication is based on the pivotal Phase 3 UNIFI clinical trial which achieved its primary endpoint of clinical remission. Results from UNIFI demonstrate that treatment with STELARA both induced and maintained clinical remission in a significantly greater proportion of adult patients with moderately to severely active ulcerative colitis (UC) compared to placebo.

UC is a serious, chronic and progressive immune-mediated inflammatory disease of the large intestine, affecting approximately 910,000 people in the United States. STELARA is the first and only approved biologic therapy for UC that targets the interleukin (IL)-12 and IL-23 cytokines. The IL-12 and IL-23 cytokines have been shown to play an important role in inflammatory and immune responses.

The pivotal trial included an initial Induction study (UNIFI-I) where patients received a single dose of STELARA 6 mg/kg intravenous (IV) infusion. It was followed 8 weeks later by a Maintenance study (UNIFI-M) where patients received STELARA 90 mg subcutaneous (SC) injections every 8 weeks for 44 weeks. Both studies demonstrated the safety and efficacy of STELARA as a treatment option for patients with moderately to severely active UC, and the design and complete results were recently published in the New England Journal of Medicine.

In the Induction study, 19 percent of patients receiving STELARA achieved clinical remission in just 8 weeks. In addition, STELARA provided patients with rapid relief of their symptoms as 58 percent of patients receiving STELARA experienced a clinical response at Week 8.

In the Maintenance study, 45 percent of patients receiving STELARA were in remission at one year. STELARA also helped patients achieve clinical remission without the use of corticosteroids. At 1 year, 43 percent of patients treated with STELARA were in clinical remission and not receiving steroids.

STELARA is the first and only approved UC treatment to provide improvement of the intestinal lining as assessed by a novel histologic-endoscopic mucosal improvement endpoint. In the Induction study, 17 percent of patients receiving STELARA achieved histologic-endoscopic mucosal improvement at Week 8. In the Maintenance study, 44 percent of patients receiving STELARA achieved histologic-endoscopic mucosal improvement at 1 year. Histologic-endoscopic mucosal improvement is a combined measure that assesses the improvement of the colon at the cellular level through histologic examination and through images observed during colonoscopy. The relationship of histologic-endoscopic mucosal improvement to long-term outcomes was not studied in the clinical trial.

“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, M.D., Chief, Division of Gastroenterology, and Professor of Medicine, UC San Diego School of Medicine, and study investigator.^* “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.”

Since receiving approval in September 2009 for the treatment of adults living with moderate to severe plaque psoriasis, STELARA has received approval for four additional indications: adolescent patients with moderate to severe plaque psoriasis, adults with active psoriatic arthritis, adults with moderately to severely active Crohn’s disease (CD), and now adults with moderately to severely active ulcerative colitis. More than 45,000 patients have been treated with STELARA for CD to-date. Consistent with the approved dosing in CD, STELARA for UC starts with a weight-based, one-time IV infusion induction dose followed by a convenient maintenance dosing schedule of a 90 mg SC maintenance injection every 8 weeks.

“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,” said Caren Heller, M.D., MBA, Chief Scientific Officer at the Crohn’s & Colitis Foundation.^† “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.”

The overall safety profile of STELARA in UC was consistent with what has been observed across all approved indications of STELARA.

“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, M.D., Ph.D., Therapeutic Area Head, Immunology, Janssen Research & Development, LLC. “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.”

Janssen will work closely with payers, providers and pharmacy benefit managers to ensure STELARA is broadly accessible and affordable for patients living with UC. Janssen CarePath offers a comprehensive support program that helps patients get started on STELARA and stay on track. Janssen CarePath provides information on insurance coverage, potential out-of-pocket costs, and treatment support, and identifies options that may help make treatment more affordable, including the Janssen CarePath Savings Program for commercially insured patients who are eligible.