A panel of the European Medicines Agency (EMA) has recommended marketing authorization for siponimod (Mayzent, Novartis) for the treatment of adults with secondary progressive multiple sclerosis (SPMS) with active disease, as evidenced by relapses or imaging features of inflammatory activity.
The positive opinion from the Committee for Medicinal Products for Human Use (CHMP) was based on results from the phase 3 EXPAND trial, a randomized, double-blind, placebo-controlled trial that compared the efficacy and safety of siponimod vs placebo in 1651 patients with SPMS, Novartis said in a news release.
The researchers also investigated a subgroup of 779 patients who had active disease, defined as relapse during the 2 years prior to the study and/or the presence of gadolinium-enhancing T1 lesions at baseline.
In the overall population, treatment with siponimod significantly reduced the risk for 3-month confirmed disability progression (CDP) (primary endpoint; 21% reduction vs placebo; P = .013) and delayed the risk for 6-month CDP (26% vs placebo; P = .0058).
In the subgroup of siponimod-treated patients with active disease, time to onset of 3-month and 6-month CDP was significantly delayed by 31% and 37%, respectively, compared to patients who received placebo.
The confirmed annualized relapse rate was reduced by 46% relative to placebo.
Treatment with siponimod has been shown to have favorable effects on MRI disease activity and brain volume loss and to slow disability in SPMS, as reported by Medscape Medical News.
In March, the US Food and Drug Administration approved siponimod for the treatment of adults with relapsing forms of MS, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease.
Once approved in Europe, siponimod will be available as 0.25-mg and 2-mg film-coated tablets. The drug should be prescribed by physicians experienced in the treatment of MS, the EMA said.
Detailed recommendations for the use of siponimod will be described in the summary of product characteristics, which will be published in the European public assessment report and made available in all official European Union languages after the marketing authorization has been granted by the European Commission.
Cite this: CHMP Backs Oral Siponimod (Mayzent) for Multiple Sclerosis - Medscape - Nov 15, 2019.