Mayzent Approved for Active SPMS Patients in England and Wales

Mayzent (siponimod) has become the first oral medication to be approved for people with active secondary progressive multiple sclerosis (SPMS) in England and Wales.

Following this decision by England’s National Institute for Health and Care Excellence (NICE) — which reversed its opinion announced in June — the National Health Service (NHS) will have two months in Wales and three months in England to prepare for providing the medication.

The NHS is then expected to make Mayzent available at discounted prices when prescribed to patients.

Most people with relapsing-remitting multiple sclerosis (RRMS) eventually progress to SPMS. Whereas RRMS is characterized by relapses — where symptoms get worse, then better — in SPMS, symptoms tend to get steadily worse. Active SPMS refers specifically to a disease course in which a person continues to experience relapses and/or has evidence of new and active brain lesions.

“At the moment there are few options for someone whose MS is changing from relapsing remitting to secondary progressive, so this approval is very welcome and a big step forward,” David Martin, CEO of the MS Trust, said in the U.K. association’s press release.

The MS Trust played a central role in NICE’s reappraisal of the treatment, including in its recent submission arguing for approval views that the group gathered from nearly 400 patients. These views gave “a compelling account of the challenges of living with secondary progressive MS and [made] a strong case for this new treatment” the MS Trust stated in its release.

Mayzent, marketed by Novartis, is a once-daily tablet. The medication works by binding to the sphingosine-1-phosphate (S1P) receptor, a protein on immune cells. By targeting the S1P receptor, Mayzent is designed to prevent immune cells from leaving lymph nodes and entering the nervous system via circulating blood, causing disease-driving inflammation. 

The therapy’s safety and efficacy in these patients were demonstrated in the Phase 3 clinical trial EXPAND (NCT01665144), which enrolled 1,651 people with active and non-active SPMS.

Results showed that six months of treatment with Mayzent lowered the risk of confirmed disability progression by 37% in the active SPMS group (779 patients) compared with a placebo. Treatment also reduced cognitive decline. Long-term follow-up data from the trial further supported the medication’s effectiveness.

“By slowing down disability progression and improving cognition, siponimod has the potential to allow people to carry on working, remain independent and stay connected with family and friends,” Martin said.

Reported side effects associated with Mayzent’s use include low white blood cell count, increased levels of liver enzymes, slower heart rate when starting treatment, macular edema (swelling in the back of the eye that affects vision), high blood pressure, shingles, and convulsions.

Mayzent was previously approved for active SPMS in the U.S., EU, Australia, and Canada.

NICE’s approval reverses the agency’s earlier decision to reject Mayzent. At that time, the agency had requested more detailed evidence and analysis of data demonstrating the therapy is cost-effective.

It also noted that EXPAND did not directly compare Mayzent with interferon beta-1b (sold in Europe as Betaferon and Extavia), which prior to this week had been the only available treatment for active SPMS patients in the U.K.

The Scottish Medicines Consortium also approved Mayzent for active SPMS this week. Northern Ireland’s health agency typically reviews NICE positions when deciding on medications to be offered patients there.

“We hope that the availability of this new treatment will lead to a greater focus on services for progressive MS which would benefit a much wider group of people,” Martin said.

“This is fabulous news,” Caroline, a person with SPMS quoted on the MS Trust webpage, added. “While many of us won’t meet the criteria, it is a game-changer for some and hopefully signals the start of more treatments for all with SPMS.”