FDA Approves Novartis’ Mayzent for Relapsing Forms of Multiple Sclerosis, Including Active SPMS

FDA Approves Novartis’ Mayzent for Relapsing Forms of Multiple Sclerosis, Including Active SPMS

The U.S. Food and Drug Administration (FDA) has approved Novartis’ Mayzent (siponimod) oral tablets for adults with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome (CIS), relapsing-remitting disease (RRMS), and active secondary progressive disease (SPMS).

Mayzent was designed to inhibit the activity of two sphingosine-1-phosphate receptors on the surface of immune cells. It prevents immune cells from migrating to the brain and spinal cord, thereby reducing the inflammatory process that promotes MS development and progression.

FDA’s decision was based, in part, on data from the EXPAND Phase 3 clinical trial (NCT01665144), with 1,651 SPMS patients (mean age 48 years) who had evidence of disability progression in the prior two years and no relapses in the three months prior to enrollment. Participants, recruited across 31 countries, were randomized to receive 2 mg of Mayzent orally or a placebo once a day.

According to Novartis, results showed that Mayzent reduced the risk of disability progression at three months (the trial’s primary endpoint, or goal) by 21% in SPMS patients compared to placebo. Of note, in those with active SPMS (meaning with relapses) a 33% reduction was observed. The risk of disability progression at six months was of 26% compared to placebo.

The therapy also decreased the annualized relapse rate by 55% in this group, and improved cognitive processing speed both in patients who had relapses within two years of treatment (those with active SPMS) and in those who had not (non-active SPMS).

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Mayzent was also seen to significantly reduce blood levels of neurofilament light chain — a biomarker of nerve cell damage — meaning the therapy can effectively protect nerve cells.

However, according to an FDA press announcement, “in the subgroup of patients with non-active SPMS, the results were not statistically significant.” People with non-active SPMS experience disability progression but without relapses.

Previous results from a Phase 2 clinical trial (NCT00879658; BOLD) showed that Mayzent was able to reduce the number of active brain lesions in RRMS patients compared to a placebo.

Of note, it is estimated that up to 80% of RRMS patients will develop SPMS. Mayzent may address an unmet need for RRMS patients in transition, and those with active SPMS who have transitioned.

“Multiple sclerosis can have a profound impact on a person’s life,” Billy Dunn, MD, director of the division of neurology products at the FDA’s Center for Drug Evaluation and Research, said in the release. “We are committed to continuing to work with companies that are developing additional treatment options for patients with multiple sclerosis.”

Mayzent may increase the risk of infections, and its accompanying medication guide makes recommendations to possible serious side effects, including that patients undergo a complete blood count prior to starting this therapy. Treatment may also lead to impaired vision (a condition called macular edema), so patients are advised to contact a physician if they experience changes in vision.

The most common adverse reactions associated with Mayzent in clinical trials include headache, high blood pressure, and changes in liver function. Healthcare professionals should monitor the patient’s blood pressure and heart rate during treatment, and assess liver enzyme levels before the start of the treatment.

Novartis has also submitted a Marketing Authorization Application to the European Medicines Agency (EMA). The application is under review, and the EMA’s decision is expected at the end of 2019.


  1. Linda Hulne says:

    As a person with MS for 28 years, I am so thankful for us MSers to have another choice of treatment. Thank you for keeping us informed on the latest information available.

  2. Use of p-values is harmful in statistics, but FDA still requires them. The estimated of the differences of non active SPMS vs placebo were not reported here. P-values depend critically on the number of events (n), so that even “large” differences could be have p-values >5% due to low number of events. Was the study assuming higher number of progressive events in non active SPMS-group and the power was too low due to that?

  3. Ann says:

    I’ve been waiting over a year for Siponimod to be finally approved as a DMT for SPMS. Now I read it’s for active SPMS. If my husband’s ms is not currently active does that mean he’s not eligible for Siponimod? Does he have to have an MRI to prove his SPMS is active?

  4. DJ Hartt says:

    Novartis cancelled its trial for fingolimod (Gilenya) for progressive MS as its patent was running out. Here we have a drug with almost identical mode of action siponimod (Mayzent) both made by Novartis. I am sure if trials were run head to head of Gilenya vs. Mayzent, they would have identical underwhelming results.

    Mayzent will bring you to the same miserable endpoint in the horrible disease just slightly slower at a whopping 21% above taking nothing at all if you happen to be an active SPMS.

    The FDA read through this non sense (“in the subgroup of patients with non-active SPMS, the results were not statistically significant”) and is why they approved it for a small subgroup of SPMS patients, the active SPMS patient.

    This selection bias trial does not equate to a meaningful treatment of non active progressive MS, but I am sure Novartis wants neurologists to “push” their drug as a treatment for all SPMS.

    Where are the effective treatments for inhibiting neurodegeneration and enhancing remyelination and neurorestoration? Plain and simple, the immunosuppressive treatments reducing B and T-cells work very little, if at all, in progressive MS.

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