CHMP recommends approval of Mayzent in secondary progressive multiple sclerosis.- Novartis

Novartis announced the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion for Mayzent (siponimod) for the treatment of adult patients with secondary progressive multiple sclerosis (SPMS) with active disease evidenced by relapses or imaging features of inflammatory activity (i.e. Gd-enhancing T1 lesions or active, new or enlarging, T2 lesions). While MS progression is different for each patient and influenced by multiple factors, including use of MS disease-modifying treatments, it is estimated that up to 80% of patients will eventually transition from RRMS to SPMS. If approved, Mayzent is expected to be the first and only oral treatment specifically indicated for patients with active SPMS based on a randomized clinical trial of a broad SPMS patient population.

The positive CHMP opinion for Mayzent is based on groundbreaking data from the Phase III EXPAND study, a randomized, double-blind, placebo-controlled trial, comparing the efficacy and safety of Mayzent versus placebo in people with SPMS. EXPAND also investigated a subgroup of patients with active disease (n=779), defined as patients with relapses in the two years prior to the study and/or presence of Gd-enhancing T1 lesions at baseline. The baseline characteristics were similar except for signs of activity compared to the overall population. Results from EXPAND in the overall population showed that Mayzent significantly reduced the risk of three-month confirmed disability progression (CDP) (primary endpoint; 21% reduction versus placebo, p=0.013) and meaningfully delayed the risk of six-month CDP (26% versus placebo, p=0.0058).

In the subgroup of Mayzent-treated patients with active disease, results showed that the time to onset of three month and six month CDP was significantly delayed by 31% compared to placebo and by 37% compared to placebo, respectively. In addition, the annualized relapse rate (ARR � confirmed relapses) was reduced by 46% compared to placebo. Significant favorable outcomes were seen in other relevant measures of MS disease activity, including MRI disease activity and brain volume loss (brain shrinkage). Additional analyses of the EXPAND study were presented this year at the 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), demonstrating that Mayzent can help patients keep their mobility for over four years longer on average, and reduced grey matter volume loss at one and two years, a key driver of disability progression and cognitive decline in patients with SPMS.