Ponesimod NDA Submitted for Relapsing Multiple Sclerosis


The oral selective S1P1 receptor modulator functionally inhibits S1P activity and reduces circulating lymphocytes.

Husseini Manji, MD, FRCPC

Husseini Manji, MD, FRCPC

Janssen Pharmaceutical Companies of Johnson & Johnson has submitted its new drug application for ponesimod for the treatment of adults with relapsing multiple sclerosis (MS).

The investigational oral drug is a selective sphingosine-1-phosphate receptor 1 (S1P1) modulator that functionally inhibits S1P activity and reduces circulating lymphocytes. The drug was previously submitted to the European Medicines Agency for approval, as well.

The NDA is based off of data from the phase 3 OPTIMUM study, a head to head trial that examined the safety and efficacy of 20 mg ponesimod against 14 mg teriflunomide (Aubagio; Sanofi) in adults with relapsing MS.

The data, which were presented at the 2019 ECTRIMS annual meeting, showed a 30.5% greater reduction in annualized relapse rate (ARR) with treatment with ponesimod compared with teriflunomide at week 108. ARRs for ponesimod and teriflunomide were 0.202 and 0.290, respectively (P = .003).2

The investigational treatment also showed positive impacts on secondary end points, including fatigue-related symptoms as assessed with the Fatigue Symptom and Impact Questionnaire-Relapsing Multiple Sclerosis (FSIQ-RMS), an MS-specific, 20-item patient-reported outcome measure. At week 108, scores suggested statistically significant effects on symptoms of fatigue with ponesimod treatment compared with teriflunomide, with a mean difference of —3.57 (P = .0019).2

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“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, in a statement.1 "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 patients seeking a more targeted treatment option."

In addition, treatment with ponesimod was associated with a 56% reduction in cumulative number of combined unique active lesions (P <.001). The most common adverse events recorded were nasopharyngitis, headache, upper respiratory tract infections, and an increase in alanine aminotransferase.


1. Janssen Submits Ponesimod New Drug Application to the U.S. FDA for Treatment of Adults with Relapsing Multiple Sclerosis [news release]. Titusville, NJ: The Janssen Pharmaceutical Companies of Johnson & Johnson. March 18, 2020. prnewswire.com/news-releases/janssen-submits-ponesimod-new-drug-application-to-the-us-fda-for-treatment-of-adults-with-relapsing-multiple-sclerosis-301026319.html

2. Kappos L. Efficacy and safety of ponesimod compared to teriflunomide in patients with relapsing multiple sclerosis: results of the randomized, active-controlled, double-blind, parallel-group phase 3 OPTIMUM study. Presented at: ECTRIMS 2019. September 11-13, 2019; Stockholm, Sweden. Abstract 93.

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