Safety and effectiveness of fingolimod in <scp>Japanese</scp> patients with multiple sclerosis: Results of a post‐marketing surveillance study

Abstract Objective Fingolimod is the first oral sphingosine‐1‐phosphate receptor modulator approved in Japan for multiple sclerosis (MS). A large Japanese observational study of fingolimod in patients with MS was carried out to support its safety and effectiveness in a real‐world setting. Methods This 2‐year, prospective, multicenter, single‐cohort, observational study included all Japanese patients with MS who initiated fingolimod (0.5 mg/day). Safety endpoints included adverse events and adverse drug reactions. Effectiveness endpoints included the annualized relapse rate, Kurtzke's Expanded Disability Status Scale score and physician clinical global impression. All endpoints were analyzed in fingolimod‐naïve patients. Results Of the 1792 patients who started fingolimod between 28 November 2011 and 31 May 2013, 1624 and 1623 fingolimod‐naïve patients were included in the safety and effectiveness analysis sets, respectively. The most common MS type was relapsing–remitting MS (89.47%). Adverse events, adverse events leading to discontinuation of fingolimod, adverse drug reactions and serious adverse drug reaction incidences were 64.10%, 15.33%, 57.88% and 23.46%, respectively. No new/unexpected safety signals were identified. The annualized relapse rate was 0.97 during the 1 year before baseline, and decreased to 0.22 after treatment. The mean Expanded Disability Status Scale score remained stable throughout treatment, irrespective of the baseline Expanded Disability Status Scale score (≥3 or &lt;3). Physician clinical global impression was classified as ‘effective’ in the majority of patients (70.3%–90.1%) throughout the treatment period. Conclusion Fingolimod was well tolerated and no new safety concerns were identified in this Japanese 2‐year post‐marketing study. Additionally, fingolimod was effective in preventing MS relapse and physical disability progression in this real‐world population comprising mainly relapsing–remitting MS patients.