FDA Accepts Siponimod NDA for Secondary Progressive MS


The application was based on data from the EXPAND phase 3 trial of siponimod in adults with secondary progressive multiple sclerosis.


The US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for Novartis’ siponimod (BAF312) for secondary progressive multiple sclerosis. The investigational drug is an oral, once-daily medication for the treatment of the condition in adults.

In addition to the NDA acceptance, a Marketing Authorization Application was accepted by the European Medicines Agency (EMA). Novartis anticipates regulatory action on siponimod in the US in March of 2019 and in Europe in late 2019.

"We are excited to see a potential new treatment on the horizon," said Bruce Bebo, PhD, Executive Vice President Research, National MS Society, United States, in a statement. "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 New Drug Application was based on data from the EXPAND study of the safety and efficacy of siponimod versus placebo in adults with typical secondary progressive multiple sclerosis (SPMS). The randomized, double-blind phase 3 trial included 1651 patients in 31 countries who received either siponimod 2 mg (n = 1105) or placebo (n = 546).

In the first 12 months, there was a 75% decrease in T2 lesion volume for the siponimod treatment group from a baseline of 10286 mm3 (205 mm3 for the siponimod group compared to 818 mm3 with placebo). By month 24, T2 lesion volume for the siponimod group had decreased 83% from baseline (163 mm3 with siponimod, 940 mm3 with placebo; P <.001).

The number of new or enlarging T2 lesions was 1.00 in the siponimod treatment arm compared to 3.78 in the placebo arm (P <.0001) at month 12. At month 24, this dropped to an average of .49 new lesions in the siponimod group compared to 3.44 in placebo.

From a baseline of 1421 cm3, the brain volume change was -.28 cm3 with siponimod while the placebo group changed -.46 cm3 from a baseline of 1425 cm3, at month 12. At 24 months, the siponimod group had an average reduction of -.63 cm3 compared to -.91 cm3 in the placebo group.

Additionally, according to the Novartis statement, siponimod significantly reduced the risk of three-month disability progression compared to placebo (21% versus placebo, P = .013).

"Siponimod is the first investigational medicine to show a significant delay in disability progression in typical SPMS patients," said Paul Hudson, Chief Executive Officer, Novartis Pharmaceuticals. "With siponimod, we underpin our strong commitment to the MS community by reimagining care for people whose lives have been considerably disrupted by this devastating illness. We are closely working with the FDA and EMA to ensure siponimod is available for patients as soon as possible."

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