Gilenya, which was given the FDA breakthrough therapy designation in December 2017 for the pediatric indication, is now the first disease-modifying therapy indicated for RMS patients, aged 10-18 years. It is also the first approved MS drug by the FDA for pediatric patients.
FDA center for drug evaluation and research neurology products division director Billy Dunn said: “For the first time, we have an FDA-approved treatment specifically for children and adolescents with multiple sclerosis.
“Multiple sclerosis can have a profound impact on a child’s life. This approval represents an important and needed advance in the care of pediatric patients with multiple sclerosis.”
Gilenya’s approval was based on the findings of the global phase 3 trial PARADIGMS held in over 200 children and adolescents with RMS where Gilenya was evaluated against interferon beta-1a.
The trial saw an a decrease of around 82% in the rate of relapses over a period of up to two years in the patient group treated with oral Gilenya. This was in comparison to patients, who were treated with another MS drug, interferon beta-1a, which was given in the form of an intramuscular injection weekly once.
According to Novartis, Gilenya has a reversible lymphocyte redistribution effect targeting focal and diffuse central nervous system (CNS) damage resulting from multiple sclerosis.
Novartis Pharmaceuticals CEO Paul Hudson said: "Since revolutionizing the treatment of relapsing MS as the first oral disease-modifying therapy, Gilenya has become an important mainstay of treatment for adult patients.
"Today's announcement is a result of our pioneering approach and ongoing commitment to advancing care for all individuals living with MS, and we are delighted this has led to a long-awaited, specifically-approved treatment option for young patients."
Image: Novartis headquarters in Basel, Switzerland. Photo: courtesy of Novartis AG.