Efficacy and Safety of Ozanimod Hydrochloride for the Treatment of Moderately to Severely Active Ulcerative Colitis

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Investigators assessed the safety and efficacy of ozanimod as maintenance therapy for patients with moderately to severely active ulcerative colitis over a 52-week period.

For patients with ulcerative colitis (UC), ozanimod was found to be a more effective maintenance therapy than placebo. These findings, from a phase 3, multicenter, randomized, double-blind, placebo-controlled trial, were published in The New England Journal of Medicine.

Patients with moderately to severely active UC were recruited at 285 sites in 30 countries between 2015 and 2020; the study was funded by Bristol Myers Squibb. Patients in cohort 1 were randomly assigned in a 2:1 ratio to receive a 10-week induction regimen of 1 mg/day ozanimod hydrochloride (n=429) or placebo (n=216). Those in cohort 2 (n=367) received 10-weeks open-label ozanimod. All treatment responders (n=457) were randomly assigned in a 1:1 ratio to receive maintenance therapy with ozanimod (n=230) or placebo (n=227) through week 52. Efficacy and safety were assessed.

Recipients of the cohort 1 ozanimod, cohort 2 ozanimod, and placebo treatments were aged mean 41.4, 42.1, and 41.9 years; 57.1%, 58.3%, and 66.2% were men; total Mayo scores were 8.9, 9.1, and 8.9; and UC diagnosis occurred 6.9, 7.9, and 6.8 years prior, respectively.

At week 10, 18.4% of ozanimod and 6.0% of placebo recipients were in clinical remission (P <.001). Similar group differences were reported for clinical response (47.8% vs 25.9%; P <.001), endoscopic improvement (27.3% vs 11.6%; P <.001), and mucosal healing (12.6% vs 3.7%; P <.001), respectively.

At week 52, 37.0% of ozanimod and 18.5% of placebo recipients were in clinical remission (P <.001). Similar group differences were observed for clinical response (60.0% vs 41.0%; P <.001), endoscopic improvement (45.7% vs 26.4%; P <.001), glucocorticoid-free remission (31.7% vs 16.7%; P <.001), mucosal healing (29.6% vs 14.1%; P <.001), maintenance of remission (52% vs 29%; P =.002), and durable remission (17.8% vs 9.7%; P =.003), respectively.

During the maintenance period, 49.1% of the ozanimod and 36.6% of the placebo cohorts had an adverse event and 5.2% and 7.9%, a serious adverse event, respectively. A total of 3 participants discontinued ozanimod and 6 discontinued placebo due to adverse events. Ozanimod was associated with increased infection rates (23.0% vs 11.9%, respectively).

This study was limited by its relatively short maintenance period; however, an open-label extension study is ongoing.

The study authors concluded that ozanimod was more effective at treating moderate to severe UC than placebo.

Disclosure: Multiple authors declared affiliations with industry. Please refer to the original article for a full list of disclosures.


Sandborn WJ, Feagan BG, D’Haens G, et al. Ozanimod as induction and maintenance therapy for ulcerative colitis. N Engl J Med. 2021;385(14):1280-1291. doi:10.1056/NEJMoa2033617