Fingolimod may reduce relapses more effectively than interferon beta-1a in pediatric multiple sclerosis.
Although there is no cure for multiple sclerosis (MS), several treatments are effective reducing symptoms and slowing disease progression.
Today, Novartis announced positive results from the PARADIGMS study, which explored the safety and efficacy of fingolimod in children and adolescents aged 10 to 17 years with MS, according to a press release.
The phase 3 findings show that treatment with fingolimod significantly reduced the number of relapses experienced by this population over 2 years compared with interferon beta-1a intramuscular injections, according to the release.
Novartis reports that the drug’s safety profile was similar to what was seen in other clinical trials, including more adverse events experienced with interferon.
"Living with MS is a tremendous challenge at any age. However, its appearance in children and adolescents, when these young individuals should be developing and focusing on their future, can be devastating," said Vas Narasimhan, global head of Drug Development and chief medical officer at Novartis.
PARADIGMS is the first phase 3 study of a disease-modifying therapy in pediatric MS, according to the release.
Included in the study were 215 children and adolescents with MS who were randomized to receive once-daily oral fingolimod or intramuscular interferon once per week.
The primary endpoint was the number of relapses in patients treated up to 2 years. The secondary endpoints were the number of T2 lesions, Gadolinium enhancing T1 lesions, and the safety and pharmacokinetics of fingolimod.
Pediatric MS is commonly diagnosed during adolescence and is associated with frequent and severe relapses, which can affect mobility, balance, and cognitive function, according to the release.
Patients with pediatric MS are more likely to experience physical disability earlier compared with patients diagnosed with MS as adults, Novartis reported.
While fingolimod is not approved for pediatric MS, Novartis plans to analyze the findings and speak to regulatory agencies for next steps.
Fingolimod works by deploying a reversible lymphocyte redistribution effect targeting both focal and diffuse central nervous system damage. It has been shown to reduce relapses, MRI lesions, brain shrinkage, and disability progression in adults, according to the release.
"With no specifically approved treatment options based on a thorough study such as PARADIGMS, the risk of long-term disease progression in these patients is much greater,” Narasimhan said. “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. I would like to thank the young people with MS and their families, physicians and nurses who participated and made this landmark study possible."