Novartis today announced that the US Food and Drug Administration (FDA) has approved Mayzent® (siponimod) for the treatment of adults with relapsing forms of multiple sclerosis, including secondary progressive multiple sclerosis (SPMS) with active disease, relapsing remitting multiple sclerosis (RRMS) and clinically isolated syndrome (CIS). SPMS is a debilitating form of multiple sclerosis (MS) characterized by progressive and irreversible neurological disability. Mayzent is expected to be available in the US in approximately one week. Patients will not require a first dose observation (FDO, cardiac monitoring upon initiation) unless they have certain pre-existing cardiac conditions.
One of the most important aims of MS treatment is delaying disability progression and preserving cognition. 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. 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,
said Paul Hudson,
Chief Executive Officer,
Most patients transition from RRMS to SPMS over time. Therefore, starting therapy early is critical for patients to help slow the rate of disability progression. Disability progression most frequently includes – but is not limited to – an impact on ambulation, which could lead to patients needing a walking aid or a wheelchair, bladder dysfunction and cognitive decline.
We are grateful that there is a new treatment option for adults with active secondary progressive MS. We are hopeful this approval will stimulate a conversation between patients and healthcare professionals about disability progression after relapsing remitting MS and its early management,
said Bruce Bebo, PhD,
Executive Vice President,
Research, US National MS Society.
The approval of Mayzent is based on groundbreaking data from the Phase III EXPAND study, a randomized, double-blind, placebo-controlled study, comparing the efficacy and safety of Mayzent versus placebo in people living with SPMS. Patients enrolled in EXPAND were representative of a typical SPMS population: at study initiation, patients had a mean age of 48 years, had been living with MS for approximately 16 years and more than 50% had a median Expanded Disability Status Scale (EDSS) score of 6.0 and relied on a walking aid. Mayzent significantly reduced the risk of three-month confirmed disability progression (CDP) (primary endpoint; 21% reduction versus placebo, p=0.013; 33% reduction versus placebo in patients with relapse activity in the two years prior to screening, p=0.0100). Additionally, Mayzent meaningfully delayed the risk of six-month CDP (26% versus placebo, p=0.0058) and reduced the annualized relapse rate (ARR) by 55%. Furthermore, EXPAND showed significant favorable outcomes in other relevant measures of MS disease activity, including cognition, MRI disease activity and brain volume loss (brain shrinkage).
Most common adverse reactions (incidence greater than 10%) were headache, hypertension, and transaminase increase.
With the approval of Mayzent, we now have a much-needed therapeutic option to address SPMS with active disease. Importantly, healthcare professionals now have even more reason to help patients identify changing symptoms and uncover early signs of progression.
said EXPAND Steering Committee member Bruce Cree,
MD, PhD, MAS, Clinical Research Director and George A. Zimmermann Endowed
Professor in Multiple Sclerosis, University of California, San Francisco, School of Medicine.
Novartis is committed to bringing Mayzent to patients worldwide, and additional regulatory filings are currently underway with other health authorities outside the US. Regulatory action for Mayzent in the European Union is anticipated in late 2019, with additional regulatory action anticipated in Switzerland, Japan, Australia and Canada this year.