The Swiss company will present the trial’s results at the 7th Joint ECTRIMS-ACTRIMS meeting, set for Oct. 25-28 in Paris.
The study addressed the safety and efficacy of an oral, once-daily dose of Gilenya in 215 MS patients ages 10 to 17. Participants received 0.5 mg or 0.25 mg of Gilenya, according to their body weight, and results were compared with those of intramuscular Avonex (interferon beta-1a given once weekly).
The trial — conducted at 87 sites in 25 countries — was designed in partnership with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the International Pediatric Multiple Sclerosis Study Group.
Gilenya led to a “clinically meaningful decrease in the number of relapses” over a period of up to two years, compared to Avonex, according to the trial. The safety results of Gilenya matched those observed in previous trials, with adverse events more likely among the Avonex group.
Importantly, the PARADIGMS trial is the first-ever randomized, controlled Phase 3 study of a disease-modifying therapy in pediatric MS. No treatment is currently available for children and adolescents with MS.
Novartis will now complete a thorough evaluation of the results and later submit Gilenya for approval by regulatory agencies. It will also extend the study to a five-year period.
“Living with MS is a tremendous challenge at any age,” Vas Narasimhan, global head of drug development and chief medical officer at Novartis, said in a press release. “However, its appearance in children and adolescents — when these young individuals should be developing and focusing on their future — can be devastating.”
Narasimhan explained that without any specifically approved treatment options based on thorough clinical trials, the risk of long-term disease progression in such patients is much higher.
“The outcome of this study is very exciting news for the MS patient community, all of whom benefit from potential advances in high-quality, evidence-based care such as this,” he said. “I would like to thank the young people with MS and their families, physicians and nurses who participated and made this landmark study possible.”
Between 3 to 5 percent of the roughly 2.3 million people worldwide with MS are children. Pediatric MS causes physical disability at an early age, and relapses are more frequent and often more severe than those seen in adults with MS. About 98 percent of children with MS have relapsing-remitting multiple sclerosis.
The FDA has approved Gilenya as a first-line therapy for RRMS in adults. Likewise, the EMA has approved it for adults with highly active RRMS. In adults, Gilenya has been shown to improve four key measures of RRMS: relapses, MRI lesions, brain shrinkage and disability progression.