CHMP recommends approval of Gilenya for treatment of multiple sclerosis in children, adolescents

Novartis today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Gilenya^® (fingolimod) for the treatment of children and adolescents 10 to 17 years of age with relapsing remitting forms of multiple sclerosis (RRMS). If approved, Gilenya^® is expected to be the first oral disease-modifying therapy indicated for these patients based on a randomized controlled clinical study[1]. The younger patient population experiences two-to-three times as many relapses as adults, often leading to a more severe prognosis and earlier disability compared to adult-onset MS[2].  If approved, Gilenya would address the urgent need faced by these young people. This market authorization would expand the age range of Gilenya, one of the most prescribed MS treatments worldwide. Gilenya was previously approved for adults with RRMS aged 18 years and older in Europe.

"The lives of kids are immensely impacted by the early onset of MS, from playing sports, going to school or enjoying time with friends and family. We need to address the urgent need for new and effective treatments, and with Gilenya, we may now have an option that can make a substantial difference for young patients", said Paul Hudson, Chief Executive Officer, Novartis Pharmaceuticals. "This CHMP positive opinion is a testament to our relentless dedication to reimagining MS care across all generations. We are very excited to be a step closer to bringing this much-needed treatment to young MS patients across Europe."

"Today's CHMP positive opinion is a momentous advancement for the children and adolescents impacted by MS," said Pedro Carrascal, President, European Multiple Sclerosis Platform. "Young European patients and their families, who have long been hoping for an effective disease modifying therapy, could soon have a new treatment option to alleviate the devastating impact of this condition."

The CHMP positive opinion is based on the PARADIGMS trial, a first-of-its-kind clinical study in MS specifically designed for children and adolescents aged 10 to 17 years[3]. Results from the double-blind, randomized, multi-center Phase III study of Gilenya vs. interferon beta-1a show that compared to interferon beta-1a, Gilenya significantly reduced the annualized relapse rates by 82% (relative difference to interferon beta-1a, p<0.001) and delayed the time to first relapse[4]. Furthermore, it also significantly reduced the number of new or newly enlarged T2 lesions up to 24 months by 53% (p<0.001) and the annualized rate of brain volume loss (brain shrinkage) by 40%[4]. The full PARADIGMS data was recently published in The New England Journal of Medicine.

The European Commission will review the CHMP opinion and is expected to deliver its final decision within three months. The decision will be applicable to all 28 European Union member states plus Iceland, Norway and Liechtenstein. Gilenya received FDA approval for the treatment of children and adolescents 10 years of age and older with MS on May 11, 2018.