Novartis (NYSE: NVS) today announced the European Commission (EC) has approved 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. Although every patient's MS journey is unique, up to 80% of relapsing remitting MS (RRMS) patients will eventually transition to SPMS4. Mayzent addresses an unmet need for SPMS patients with active disease who, until now, did not have an oral treatment that has been shown to be effective in delaying progression in this patient population. The European marketing authorization makes Mayzent the first and only indicated oral treatment proven in SPMS patients with active disease based on a randomized clinical trial of a broad range of SPMS patients.
"We are delighted by the news that there is now a treatment available for people in Europe living with active SPMS to potentially delay the progression of this debilitating disease," said Pedro Carrascal, President of the European MS Platform. "This treatment brings hope for improved care and quality of life to patients who have long been underserved."
The EC's approval is based on data from the EXPAND study, a randomized, double-blind, placebo-controlled trial, comparing the efficacy and safety of Mayzent versus placebo in a broad range of SPMS patients (EDSS score 3·0–6·5 at baseline). EXPAND included 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.
In the subgroup of Mayzent-treated patients with active disease, results showed:
The risk of three‑month and six‑month confirmed disability progression (CDP) was significantly reduced by 31% compared to placebo and by 37% compared to placebo, respectively5.
Significant favorable outcomes in other relevant measures of MS disease activity, including annualized relapse rate (ARR – confirmed relapses), MRI disease activity and brain volume loss (brain shrinkage)5.
Results in the overall population showed that Mayzent significantly reduced the risk of three-month 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)2. Mayzent also has a meaningful benefit on cognition and demonstrated clinically relevant effects on cognitive processing speed5.
"As the only indicated oral therapy proven for people living with SPMS with active disease, we are pleased that the European approval of Mayzent will help change the conversation about progressing MS and expand possibilities for patients and their caregivers," said Max Bricchi, Global Head, Neuroscience Franchise, Novartis Pharmaceuticals. "Delaying progression is hugely important for people living with MS who want to maintain independence longer and today's decision gives them a chance to achieve this goal. We are dedicated in our mission to reimagine medicine and enable brighter futures for people with severe progressive diseases like MS."
Novartis is working closely with all stakeholders to ensure that eligible European patients can start benefitting from this treatment as quickly as possible. In March 2019, Novartis received approval from the US Food and Drug Administration (FDA) for Mayzent for the treatment of relapsing forms of multiple sclerosis (RMS), to include clinically isolated syndrome (CIS*), relapsing remitting disease, and active secondary progressive disease, in adults. In November 2019, Novartis received approval from the Australian Therapeutic Goods Administration (TGA) for Mayzent for adult patients with SPMS. Novartis is committed to bringing Mayzent to patients worldwide, and additional regulatory filings are currently underway in Switzerland, Japan, Canada and China.