New Drug Application for Ponesimod Was Submitted to FDA

A new drug application (NDA) for ponesimod (Janssen Pharmaceutical, Titusville, NJ) for the treatment of adults with relapsing multiple sclerosis (MS), was submitted to the Food and Drug Administration (FDA).

At week 108, a statistically significant reduction in annualized relapse rate (ARR) of 30.5% was observed with ponesimod when compared with teriflunomide. Additionally, a statistically significant reduction in fatigue and a 56% reduction on combined unique active lesions (CUALs) in the brain were observed with ponesimod compared with teriflunomide. The safety profile observed for ponesimod was consistent with previous studies of ponesimod and the known safety profile for other drugs of this class  

"Nearly 1 million people over the age of 18 in the US live with MS, and approximately 85% of people with the condition are initially diagnosed with relapsing MS. Despite new advancements and treatments coming to market, a number of unmet needs still remain–leaving patients struggling to manage often-debilitating symptoms," said Mathai Mammen, MD, PhD, global head of Janssen Research & Development. "In the coming months, we'll work closely with the FDA to bring ponesimod 1 step closer to the MS patient community and remain encouraged by its superior efficacy profile–specifically in reducing new inflammatory lesions and disability accumulation—in comparison to a leading therapy on the market."

The NDA is based on the head-to-head optimum phase 3 study (NCT03232073) which showed superior efficacy of ponesimod 20 mg on the primary endpoint of reduced ARR, as well as most secondary endpoints, compared with teriflunomide 14 mg in adults with relapsing MS. 

"What's interesting about MS is that symptoms are not always visible. Fatigue is one of the most common and debilitating symptoms of MS and yet, it's one of the most challenging to manage and treat," said Husseini Manji, MD, FRCPC, global therapeutic area head for Neuroscience at Janssen Research & Development, LLC. "We were thrilled to see improvement in fatigue-related symptoms as part of the phase 3 optimum trial as we know the profound impact it may have on a person's daily life. The improvement in fatigue, coupled with reduction in ARR, demonstrate great promise for ponesimod with individuals seeking a more targeted treatment option."  

Ponesimod is an investigational selective sphingosine-1-phosphate receptor 1 (S1P1) modulator that inhibits S1P protein activity. Ponesimod is believed to reduce the number of circulating lymphocytes that can cross the blood-brain barrier, and thus reduce inflammation, demyelination, and neurodegeneration. 

The clinical study data that supports this filing was presented in September 2019 at the 35th Congress of The European Committee for Treatment and Research in Multiple Sclerosis Conference (ECTRIMS) in Stockholm, Sweden.