The Food and Drug Administration has approved diroximel fumarate (Vumerity; Biogen, Cambridge, MA and Alkermes, Dublin, Ireland) for the treatment of multiple sclerosis (MS) including clinically isolated syndrome (CIS), relapsing remitting MS (RRMS), and active secondary progressive MS (SPMS). Diroximel fumarate is a novel second-generation oral fumarate with a distinct chemical structure that in clinical trials was found to have low rates of gastrointestinal side effects, that have made previous fumarates less tolerable.
In interim exposure and safety findings from EVOLVE-MS-1 (NCT02634307) an ongoing, phase 3 single-arm open-label safety study of diroximel fumarate for RRMS. Those results include a low overall rate of discontinuation due to adverse events (6.3%) for participants treated with diroximel fumarate. Of note, less than 1% of participants discontinued diroximel fumarate because of gastrointestinal adverse events. This ongoing study is also exploring efficacy endpoints of change in clinical and radiologic measures of MS activity compared to participants baseline measures.
Approval was based on a new drug application (NDA) submitted under the 505(b)(2) filing pathway, including pharmacokinetic bridging studies comparing diroximel fumarate to dimethyl fumarate (Tecfidera; Biogen, Cambridge, MA) to establish bioequivalence. This application relied, in part, on safety and efficacy findings previously filed for dimethyl fumarate with the FDA.
“The FDA’s approval of Vumerity delivers on Biogen’s commitment to pursue new therapies that may provide meaningful impact for people living with relapsing MS, and we look forward to bringing it to the MS community as an additional treatment option,” said Alfred Sandrock, Jr, MD, PhD, executive vice president, research and development, and chief medical officer at Biogen.
“Vumerity is a novel fumarate that offers the well-characterized efficacy of Tecfidera (dimethyl fumarate) and has been studied for improved patient-reported gastrointestinal tolerability.”
“MS is a heterogeneous disease, and real-world patient circumstances can vary, reinforcing the benefits of having therapeutic choices to support the diverse range of treatment considerations,” said Robert Naismith, MD, professor of neurology, Washington University School of Medicine in St. Louis. “Throughout its clinical development program, Vumerity has demonstrated a desirable therapeutic profile, making it a compelling new option for patients.”
“MS is a lifelong disease that has a significant impact on the people affected and their caregivers. We are encouraged by the progress being made in the treatment of MS and pleased that another treatment option will soon be available,” said Bruce Bebo, PhD, executive vice president, research, National MS Society. “It’s important for people with MS to have treatments that are both efficacious and tolerable to help manage their disease.”