Novartis multiple sclerosis drug Gilenya shows siginificant effect in phase III trial

The Swiss pharma major has reported positive topline results from the study, which assessed the safety and efficacy of oral once-daily fingolimod in children and adolescents (ages 10 to 17) with MS.

The trial is claimed to be the first randomized and controlled phase III study of a disease-modifying therapy (DMT) in pediatric MS.

Gilenya is an oral DMT, which can effectively control disease activity in relapsing multiple sclerosis. It holds a reversible lymphocyte redistribution effect targeting both focal and diffuse central nervous system damage caused by MS.

The trial demonstrated that oral fingolimod has significantly reduced multiple relapses (annualized relapse rate) in the patient population within two years compared to interferon beta-1a intramuscular injections.

Fingolimod has not yet secured any approval for the treatment of pediatric MS.

Paradigms study is a flexible duration, double-blind, randomized and phase III study designed to assess the safety and efficacy of oral fingolimod compared to interferon beta-1a in children and adolescents with a confirmed diagnosis of MS followed by a five-year open label extension phase.

The study recruited 215 children and adolescents with MS aged between 10 and 17 with an expanded disability status scale score between 0 and 5.5.

The study’s primary endpoint was the frequency of relapses in patients treated up to 24 months and secondary endpoints include the number of new or newly enlarged T2 lesions, Gadolinium enhancing T1 lesions, safety and the pharmacokinetic properties of fingolimod.

Novartis chief medical officer and drug development global head Vas Narasimhan said: "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.”

Image: Novartis headquarters in Basel, Switzerland. Photo: courtesy of Novartis AG.