- The pivotal Phase 3 OPTIMUM trial showed treatment with ponesimod led to a 30.5 percent reduction in annual relapse rate (p<0.001) vs. treatment with teriflunomide, an active comparator and widely-used first-line oral treatment, in adult patients with relapsing multiple sclerosis, (RMS)
- The OPTIMUM trial is the first of its kind to compare head-to-head two oral disease modifying treatments (DMTs) in RMS
- Approval follows more than 10 years of cumulative data from Phase 2 and Phase 3 studies demonstrating ponesimod’s efficacy and safety
- Approval builds on Janssen’s deep-rooted history in neuroscience and reinforces Company commitment to addressing unmet needs for neurological conditions like MS
Janssen, the Pharmaceutical Companies of Johnson & Johnson, announced today that the European Commission (EC) has approved PONVORY (ponesimod) for the treatment of adult patients with relapsing multiple sclerosis (RMS) with active disease defined by clinical or imaging features.
“Relapsing multiple sclerosis is an unpredictable and complex disease that can present very differently from individual to individual, placing a heavy burden on the patient and their loved ones,” said Professor Gavin Giovannoni, MBBCh, PhD, FCP (Neurol., SA), FRCP, FRCPath, Professor of Neurology at Queen Mary University of London. “I welcome the European Commission’s approval of ponesimod as an additional treatment option for those living with relapsing multiple sclerosis – it will provide patients with additional choice when making decisions about their treatment.”
The EC approval of ponesimod is based on data from the Phase 3 OPTIMUM trial, a multicentre, randomised, double-blind, parallel-group, active-controlled superiority study of 1,133 adult patients (aged 18-55 years) in 28 countries. The trial was designed to evaluate the efficacy and safety of once daily oral ponesimod (20mg) vs. once-daily teriflunomide (14mg), an approved and widely-used first-line oral treatment, in adult patients with RMS. The large, Phase 3 study showed superior efficacy of ponesimod 20mg on the primary endpoint, annualised relapse rate (ARR), with a rate reduction of 30.5 percent (p<0.001) compared with teriflunomide.1 Ponesimod also showed statistically significant superiority on one of the secondary endpoints, combined unique active lesions (CUALs). Ponesimod significantly reduced the number of new inflammatory lesions on brain MRI by 56 percent (p<0.0001) at week 108 when compared to teriflunomide.
“The OPTIMUM study is the first Phase 3 study establishing superiority versus another disease modifying treatment for relapsing multiple sclerosis, with ponesimod showing significant reductions in annual relapse rates versus teriflunomide, an active comparator and widely-used first-line oral treatment,” said Catherine Taylor, M.D., Vice President, Medical Affairs, Therapeutic Area Strategy, Europe, Middle East and Africa (EMEA), Johnson & Johnson Middle East FZ-LLC. “The approval of ponesimod by the European Commission is a positive step for people living with relapsing multiple sclerosis, as we work to provide them with an additional treatment option to manage and control their condition.”
Within the OPTIMUM study, overall, the number of treatment-emergent adverse events reported was similar between the ponesimod and teriflunomide treated groups, and the majority were mild/moderate and did not warrant treatment discontinuation. The most commonly reported adverse events in either the ponesimod 20mg group versus the teriflunomide 14mg group were Alanine Aminotransferase (ALT) enzyme elevations (19.5% vs. 9.4%), nasopharyngitis (19.3% vs. 16.8%), headache (11.5% vs. 12.7%), upper respiratory tract infection (10.6% vs. 10.4%) and alopecia (3.2% vs. 12.7%). The safety profile of ponesimod is consistent with the known safety profile of other S1P receptor modulators, although a head-to-head comparison, other than with teriflunomide, is not available.
“At Janssen, our mission is to reduce the burden, disability and devastation caused by diverse diseases of the central nervous system, including multiple sclerosis, for which there remains significant unmet patient need,” said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, Johnson & Johnson. “The approval of ponesimod by the European Commission is a significant milestone as it showcases our continued commitment in neurology, reflecting our deepened focus and commitment to this space.”
The EC approval follows the positive CHMP opinion for ponesimod in March 2021 and the announcement of the U.S. Food and Drug Administration (FDA) approval of ponesimod for use in adults with relapsing forms of MS. EC approval is valid in all 27 member states of the European Union, and the European Economic Area countries (Norway, Iceland and Liechtenstein).