Novartis on Tuesday announced positive results from the Phase III PARADIGMS study, investigating the safety and efficacy of oral once-daily fingolimod in children and adolescents with multiple sclerosis (MS).
Novartis said that the data show that oral fingolimod resulted in a significant and clinically meaningful reduction in the number of relapses (annualized relapse rate) in this patient population over a period of up to two years, compared to interferon beta-1a intramuscular injections. The safety profile of fingolimod was consistent with that seen in other clinical trials, with overall more adverse events reported in the interferon group. The PARADIGMS study is the first ever randomized, controlled Phase III study of a disease-modifying therapy (DMT) in pediatric MS.
“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, Novartis. “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. 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.”
Commonly diagnosed during adolescence, pediatric MS is associated with relapses that are more frequent and often more severe than those seen in adults with MS. Relapses negatively affect mobility, balance and cognitive function, and patients with pediatric MS are more likely to accumulate physical disability at an earlier age than those diagnosed as adults. There is currently no treatment indicated for children and adolescents living with MS, based on randomized, controlled, clinical study data.
Fingolimod is not currently approved for the treatment of pediatric MS. Novartis plans to complete a thorough analysis of these important data to speak to health authorities to agree on next steps for submission.
(Image: The logo of Swiss drugmaker Novartis is seen at its headquarters in Basel, Switzerland October 22, 2013. REUTERS/Arnd Wiegmann/File Photo)