Regulatory Affairs

FDA expands approval of Novartis’ Gilenya to treat MS in pediatric patients

PBR Staff Writer Published 14 May 2018

Novartis has secured an expanded approval of Gilenya (fingolimod) in the US for the treatment of children and adolescents, in the age group of 10-18 years, with relapsing forms of multiple sclerosis (RMS).

In 2010, the oral disease-modifying therapy (DMT) was approved by the US Food and Drug Administration (FDA) for the treatment of RMS for patients aged 18 years and older.

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.