Double regulatory approval for Novartis’ SPMS treatment siponimod

Ozanimod under review in US & EU for multiple sclerosis
Credit: Sebastian Kaulitzki

Novartis has revealed that its New Drug Application and Marketing Authorisation Application for siponimod has been accepted by the US FDA and the European Medicines Agency (EMA) respectively.

Siponimod is the first and only drug shown to meaningfully delay disability progression in typical secondary multiple sclerosis (SPMS). It is now approved as an oral, once-daily treatment for adults.

SPMS can substantially impact lives, due to physical and cognitive impairments. To bring this treatment to the MS community as quickly as possible, Novartis used a review voucher to expedite the review of siponimod in the US.

Regulatory action for siponimod is anticipated in the US in March of 2019 and in Europe in late 2019.

More than 80% of people with relapsing-remitting MS (RRMS) – the most common form of the condition at diagnosis – go on to develop SPMS, with or without relapses.

SPMS is a form of MS that leads to progressive, irreversible disability, such as the need for enhanced walking aids and wheelchairs, bladder dysfunction and cognitive decline, largely independent of relapses.

Following the initial RRMS course, there is a gradual increase in the number of patients transitioning to SPMS, with around 25% progressing by 10 years post-onset, 50% by 20 years and more than 75% by 30 years.

“We are excited to see a potential new treatment on the horizon,” said Bruce Bebo, Executive Vice President Research, National MS Society, United States. “It is a significant milestone in our unrelenting search for treatments that can benefit adults living with secondary progressive MS who currently have few options.”

The regulatory application is based on data from the EXPAND study, a randomised, double-blind, placebo-controlled Phase III study, comparing the efficacy and safety of siponimod versus placebo in people living with typical SPMS

In addition, Novartis conducted the BOLD study, a randomised, double-blind, placebo-controlled, adaptive dose-ranging, Phase II study in patients with RRMS. The study showed that siponimod significantly reduced the annualized rate of relapses over six months compared to placebo.

In Switzerland, Swissmedic granted fast track authorisation procedure for siponimod in SPMS. Discussions with additional health authorities regarding siponimod are ongoing.