Novartis’ Mayzent receives FDA approval for treating multiple sclerosis

Swiss pharmaceutical company Novartis has received a significant milestone approval from the US Food and Drug Administration (FDA) for its MS drug, Mayzent (siponimod), now authorised for the treatment of relapsing forms of multiple sclerosis (MS) in adults. This approval expands the range of treatment options for patients suffering from secondary progressive multiple sclerosis (SPMS) with active disease, relapsing-remitting multiple sclerosis (RRMS), and clinically isolated syndrome (CIS).

Multiple sclerosis, a chronic inflammatory autoimmune disease of the central nervous system (CNS), remains the leading cause of neurological disability in young adults. The disease affects the nervous system by attacking the protective sheath around nerve fibres, leading to progressive disability. Despite current treatment options, MS has no cure, and there remains an urgent need for effective therapies that can manage disease progression, particularly in its more debilitating forms.

Mayzent is a novel sphingosine 1-phosphate receptor modulator, acting on the S1P1 and S1P5 receptors. Through its selective binding, Mayzent helps block the egress of lymphocytes from the lymph nodes, thereby preventing their entry into the CNS. This action leads to anti-inflammatory effects, essential for reducing the progression of MS in patients.

Driving the approval: Clinical trial success

The FDA’s approval of Mayzent is grounded in the positive results of the phase 3 EXPAND trial. This large-scale clinical trial assessed the efficacy and safety of Mayzent in patients with SPMS, compared to a placebo. The trial included 1,651 participants, all of whom had evidence of disability progression over the past two years but had not experienced a relapse within the three months leading up to the study.

The primary endpoint of the EXPAND trial was the time taken to reach confirmed disability progression over a three-month period. The results demonstrated a significant reduction in the rate of disability progression in patients treated with Mayzent compared to those given a placebo. Additionally, patients in the Mayzent group experienced fewer relapses. However, statistical significance was not observed in those with non-active SPMS.

A game-changer for MS patients

Commenting on the approval, Paul Hudson, CEO of Novartis Pharmaceuticals, expressed his pride in the company’s achievements. “One of the most important aims of MS treatment is delaying disability progression and preserving cognition,” he said. “With Mayzent, SPMS patients with active disease will have access to the first effective oral therapy directed towards disease progression, even when MS transitions to a stage where deterioration is less dependent on the usual relapse activity.”

Hudson further highlighted the importance of Mayzent in fulfilling Novartis’ mission to “reimagine medicine” and its commitment to addressing the urgent needs of MS patients. He added, “Mayzent is a testament to the Novartis mission to reimagine medicine. We are delighted that our ongoing commitment to stop MS has led to a much-awaited treatment for these patients in need.”

The future of MS treatment

With the approval of Mayzent, Novartis has bolstered its position in the competitive MS treatment market. This is a significant win for both the pharmaceutical company and patients suffering from progressive forms of MS. Mayzent represents a promising new option for managing a disease that significantly impacts the quality of life for millions of people globally.

The drug’s approval by the FDA opens up new avenues for clinicians treating SPMS, a population that has historically had limited therapeutic options. As the MS treatment landscape continues to evolve, Mayzent offers hope for improved patient outcomes, reduced disability progression, and better quality of life for those living with this debilitating disease.