Novartis announces FDA approval of Gilenya as the first disease-modifying therapy for pediatric relapsing multiple sclerosis

Novartis announced that the US Food and Drug Administration (FDA) has approved Gilenya (fingolimod) for the treatment of children and adolescents 10 to less than 18 years of age with relapsing forms of multiple sclerosis (RMS), making it the first disease-modifying therapy indicated for these patients.

This approval expands the age range for Gilenya, which was previously approved for patients aged 18 years and older with RMS. Gilenya was granted Breakthrough Therapy designation by the FDA in December of 2017 for this pediatric indication.

“We now finally have an FDA-approved treatment for children and adolescents with relapsing MS,” said Dr. Brenda Banwell, Chief of the Division of Neurology at Children’s Hospital of Philadelphia, who served as co-Principal investigator of the pivotal study that supported the pediatric approval. “Repeated relapses are more common in young people with MS than in adults, so this is heartening news for patients and their families.”

While MS is mostly diagnosed in adults, children and adolescents with the chronic disease often experience more frequent relapses and brain lesions than adults with MS.

“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,” said Paul Hudson, CEO, Novartis Pharmaceuticals. “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.”

The approval of Gilenya for the younger patient population was supported by PARADIGMS, a double-blind, randomized, multi-center Phase III safety and efficacy study of Gilenya vs. interferon beta-1a, designed specifically for children and adolescents with RMS. The primary endpoint demonstrated that Gilenya reduced the rate of relapses (annualized relapse rate) by approximately 82% (p <0.001) over a period of up to two years compared to interferon beta-1a intramuscular injections in children and adolescents (ages 10 and older) with relapsing MS. The safety profile of Gilenya in this study was overall consistent with that seen in previous clinical trials in adults.