The U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application, while the European Medicines Agency (EMA) has accepted for review its Marketing Authorization Application.
The FDA is expected to make a final decision by March 2019, and the EMA sometime in late 2019. To expedite the FDA review process, Novartis used a review voucher.
“We are excited to see a potential new treatment on the horizon,” Bruce Bebo, executive vice-president of research at the National MS Society, said in a press release. “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.”
Siponimod, formerly known as BAF312, was designed to inhibit the activity of two sphingosine-1-phosphate receptors on the surface of immune cells. It prevents immune cells from migrating to the brain and spinal cord, thereby reducing the inflammatory process that promotes SPMS development and progression.
Novartis supported its submissions with clinical data from the EXPAND Phase 3 clinical trial (NCT01665144), which included1,651 SPMS patients with moderate to severe disability across 31 countries. Participants were randomized to receive orally 2 mg of siponimod or a placebo once a day.
Clinical trial data showed that siponimod could lower disability progression in non-relapsing patients by 14 to 20 percent at three months, and by 29 to 33 percent at six months compared to placebo. It also improved cognitive processing speed in patients who had relapses within two years and in those who did not.
Siponimod could also significantly reduce blood levels of neurofilament light chain — a biomarker of nerve cell damage — meaning the therapy can effectively protect nerve cells.
Results from the Phase 2 BOLD trial (NCT00879658) have also demonstrated that siponimod can significantly reduce the annualized relapse rate of patients with relapsing-remitting MS over six months, compared to placebo, as well as the number of brain lesions.
“Siponimod is the first investigational medicine to show a significant delay in disability progression in typical SPMS patients,” said Paul Hudson, CEO of Novartis. “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.”
He added: “We are closely working with the FDA and EMA to ensure siponimod is available for patients as soon as possible.”
Swissmedic, Switzerland’s medicines regulatory agency, granted fast-track authorization for siponimod in SPMS. Novartis is currently undergoing additional discussions with other health authorities regarding siponimod use.