Novartis chases untapped MS market with siponimod

US and European regulators have accepted filings for Novartis’s siponimod for secondary progressive multiple sclerosis (SPMS).

The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) accepted the filings for investigational oral, once-daily siponimod (BAF312) for the treatment of SPMS in adults.

If approved, the drug will become the first and only drug available, to date, to significantly delay typical SPMS.

Paul Hudson, CEO, Novartis Pharmaceuticals, said: “Siponimod is the first investigational medicine to show a significant delay in disability progression in typical SPMS patients.

“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.”

Primary progressive MS (PPMS) is the first — and only — phase of the illness for a minority of people with MS. Most sufferers will be diagnosed with relapsing-remitting MS (RRMS) and 80% of these people will develop SPMS.

The MS Society explained that usually, with SPMS, a patient’s disability or other symptoms gradually worsen. A pattern of relapses followed by the person getting better usually comes to an end. Some people may still experience relapses but they tend not to make a full recovery from these episodes.

SPMS can lead to progressive, irreversible disability, such as the need for enhanced walking aids and wheelchairs, bladder dysfunction and cognitive decline, largely independent of relapses.

The filing was based on the results of Novartis’s EXPAND clinical phase 3 study. At the beginning of the study, more than 50% of patients relied on a walking aid.

Trial results showed siponimod significantly reduced the risk of three-month confirmed disability progression versus a placebo.

The drug also meaningfully delayed the risk of six-month confirmed disability progression and demonstrated favourable outcomes in other relevant measures of MS disease activity and progression, giving hope to sufferers.

Bruce Bebo, executive vice president research, National MS Society, US, welcomed the news: “We are excited to see a potential new treatment on the horizon. 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.”

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