Mayzent Approved in Europe as First Oral Treatment for Active Secondary Progressive MS

The European Commission has approved Novartis‘s Mayzent (siponimod) as the first oral treatment for adults with active secondary progressive multiple sclerosis (SPMS).

Active SPMS is defined by the presence of evident relapses or the detection of inflammatory activity in brain lesions on imaging scans.

“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,” Max Bricchi, Novartis Pharmaceuticals’s global head of neuroscience franchise, said in a press release.

Novartis is now working closely with all European stakeholders to accelerate patients’ access to Mayzent.

The decision was supported by the recent approval recommendation from the Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency.

It follows similar Mayzent approvals in Australia and in the United States — where the therapy also was approved for the treatment of adults with clinically isolated syndrome (CIS) and relapsing-remitting disease (RRMS). Novartis also is seeking Mayzent’s approval for similar indications in Switzerland, Canada, Japan, and China.

“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.”

Mayzent belongs to the same therapy class as Novartis’s Gilenya (fingolimod), which is approved in Europe for the treatment of RRMS in patients age 10 and older.

The therapy works by binding to two sphingosine-1-phosphate (S1P) receptors on the cell surface of immune cells, leading to the internalization and degradation of the receptors. This prevents immune cells in the lymph nodes from reaching the brain and spinal cord, thus reducing the inflammatory processes that promote MS development and progression.

Mayzent’s approval was based on data from the Phase 3 EXPAND clinical trial (NCT01665144), which evaluated the safety and efficacy of Mayzent, compared with a placebo, in 1,651 patients with active or non-active SPMS.

Participants, recruited across 31 countries, were randomly assigned to receive 2 mg of Mayzent or placebo orally, once a day, and were followed for up to three years. An extension study is ongoing, in which all participants are given Mayzent for up to 10 years.

The total 779 patients, mean age of 46.6 years, had active disease, determined through the presence of relapses in the two years prior to the study and/or active inflammatory areas in the brain at enrollment.

The results from this subgroup of active SPMS patients showed that Mayzent significantly reduced the risk of disability progression by 31% at three months — the trial’s primary goal — and by 37% at six months, compared with placebo.

Mayzent treatment in these participants also led to a significant reduction in annualized relapse rates — by 46% — in inflammatory disease activity, in the number of new brain lesions, and in brain shrinkage.

In the overall patient population, Mayzent treatment also significantly delayed cognitive decline and reduced the risk of becoming unable to walk and restricted to a wheelchair by 31%, compared with placebo.

Mayzent’s safety profile — which included reduced numbers of white blood cells and cardiac and liver problems as side effects — was consistent with those observed in trials of similar MS therapies.

“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,” Bricchi concluded.