The US Food and Drug Administration (FDA) has approved ponesimod (Ponvory), a new oral medication given once daily to treat adults with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease.
Ponesimod is a selective sphingosine-1-phosphate receptor 1 modulator.
In the OPTIMUM phase 3 trial, use of oral ponesimod yielded significant reductions in relapse rate, new active lesions, and fatigue-related symptoms in comparison with the oral agent, teriflunomide.
Ponesimod is the first FDA-approved oral MS disease-modifying therapy studied against an established oral comparator, according to Janssen.
“In the pivotal study, ponesimod demonstrated superior clinical efficacy in reducing annual relapses and MRI activity compared against teriflunomide, another oral MS therapy. Those results, combined with a favorable side effect profile, make ponesimod a useful treatment option for people with relapsing MS,” Robert J. Fox, MD, Mellen Center for MS Treatment and Research, Cleveland Clinic, Cleveland, Ohio, said in a Janssen news release.
As previously reported by Medscape Medical News, the OPTIMUM trial enrolled 1133 patients with relapsing-remitting MS. Patients were randomly allocated to receive ponesimod 20 mg or teriflunomide 14 mg once daily for 108 weeks (2 years, 1 month).
Ponesimod was gradually up-titrated over 14 days, starting with a 2-mg once-daily dose to mitigate potential effects on heart rate associated with sphingosine-1-phosphate receptor modulators.
The annualized relapse rate was significantly reduced by 30% with ponesimod compared with teriflunomide. Over the study period, no confirmed relapses occurred among 71% of patients treated with ponesimod, compared to 61% in the teriflunomide group.
Ponesimod was also superior to teriflunomide in reducing the number of new gadolinium-enhancing T1 lesions and the number of new or enlarging T2 lesions by 59% and 56%, respectively.
Regarding disability, 12-week and 24-week confirmed disability risk estimates were 17% and 16% lower, respectively, for ponesimod compared with teriflunomide, but these differences did not reach statistical significance.
In clinical testing, ponesimod was generally well tolerated. The most common adverse events observed in the phase 3 trial were upper respiratory infection, increased levels of hepatic transaminase, and hypertension.
“MS is a complex disease, and any individual’s response to MS disease-modifying therapy can vary,” said Bruce Bebo, PhD, executive vice president of research, National MS Society, commented in the Janssen release.
“It’s so important that people living with MS have access to effective treatment options. We are pleased that there is a new therapy approved for relapsing MS,” said Bebo.
Full prescribing information for ponesimod is available online.