The US Food and Drug Administration (FDA) has approved Janssen Biotech, Inc.'s Simponi (golimumab) for the treatment of moderately to severely active ulcerative colitis (UC) in adult patients who have demonstrated corticosteroid dependence or who have had an inadequate response to or failed to tolerate oral aminosalicylates , oral corticosteroids, azathioprine, or 6 - mercaptopurine.

Simponi is the first and only subcutaneously administered anti - tumour necrosis factor (TNF) - alpha therapy approved to induce and maintain clinical response and improve endoscopic appearance of the mucosa during induction.

In addition, Simponi is indicated to induce clinical remission and achieve and sustain clinical remission in induction responders. As many as 700,000 people in the United States are affected by UC 1 , a chronic inflammatory bowel disease (IBD) marked by inflammation and ulceration of the innermost lining of the colon.

“The FDA approval of Simponi brings an important, new subcutaneous therapeutic option to adults living with moderate to severe ulcerative colitis, a disease where treatment options have been limited,” said William Sandborn, MD, professor and chief of the Division of Gastroenterology at the University of California, San Diego (UCSD) School of Medicine, director of the UCSD Inflammatory Bowel Disease Center, and lead study investigator. “Simponi has demonstrated significant benefits in the treatment of ulcerative colitis, a chronic inflammatory bowel disease, and represents a meaningful addition to the treatment armamentaria for gastroenterologists.”

For the treatment of UC, the Simponi dose regimen consists of 200 mg subcutaneously injected at week 0, followed by 100 mg at week 2 and then 100 mg every 4 weeks, thereafter.

The approval is supported by data from the Programme of Ulcerative Colitis Research Studies Utilizing an Investigational Treatment (PURSUIT) clinical trials, evaluating patients with moderately to severely active UC who had previously failed or were intolerant to conventional treatments . Significantly greater proportions of patients who received Simponi  200 mg/100 mg achieved clinical response, clinical remission and improvement of the endoscopic appearance of the mucosa at week 6 compared with patients receiving placebo . Through week 54, significantly greater proportions of patients in the Simponi 100 mg group maintained clinical response compared with the placebo group. The proportion of patients in clinical response following Simponi induction treatment who went on to demonstrate clinical remission at both weeks 30 and 54, without demonstrating a loss of response at any time point through week 54, were significantly greater in the Simponi  100 mg group compared with the placebo group. “The approval of Simponi for the treatment of UC is a notable milestone for adults living with this chronic, devastating disease for which there is no cure,” said Cindy Guzzo, MD, vice president, Medical Affairs, Janssen Biotech, Inc. “As leaders in the treatment of IBD for more than a decade , we are proud to offer a new sub cutaneous treatment option to patients and healthcare providers where unmet need continue s to exist.”

Simponi is also approved by the FDA for the treatment of moderately to severely active rheumatoid arthritis (RA) with the medicine methotrexate, active p soriatic arthritis alone or with the medicine methotrexate and active ankylosing spondylitis.

The Program of Ulcerative Colitis Research Studies Utilizing an Investigational Treatment (PURSUIT) included phase III multi-centre, randomized, double - blind, placebo - controlled studies designed to evaluate the safety and efficacy of subcutaneous induction and every - four - week maintenance regimens of SIMPONI in adults with moderately to severely active UC. All trial patients had failed to respond to or tolerate treatment with 6 - mercaptopurine (6 - MP), azathioprine (AZA), corticosteroids and/or 5 - aminosalicylate (5 - ASA), or were corticosteroid dependent. Study participants were naïve to treatment with TNF inhibitors and had a baseline Mayo score between 6 and 12 and an endoscopic subscore of 2 or more . The Mayo score is a 12 - point clinical assessment and colonoscopy - based measure of disease activity , which assesses improvement in symptoms based on rectal bleeding, endoscopic findings, stool frequency and a physician’s global assessment.

The induction trial (PURSUIT - SC) had an adaptive design with a phase II dose - finding portion followed by a phase III dose - confirming component. The primary endpoint was clinical response at week 6. Secondary endpoints at week 6 included clinical remission and mucosal healing ( improvement of endoscopic appearance of mucosa) - Mayo endoscopy score of 0 or 1 . Overall , 1,065 patients were treated in the study; 774 of these patients were randomized into the phase III component of the study.

Janssen Biotech is one of the Janssen Pharmaceutical Companies of Johnson & Johnson which are dedicated to addressing and solving some of the most important unmet medical needs in oncology,  mmunology, neuroscience, infectious diseases and vaccines, and cardiovascular and metabolic diseases.