The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), has recommended that Gilenya (fingolimod) be approved to treat children and adolescents, ages 10 to 17, with relapsing-remitting multiple sclerosis (RRMS).
If the CHMP opinion is accepted, Gilenya — marketed by Novartis — would become the first oral disease-modifying therapy for these patients in Europe. The therapy is already approved in Europe for adults with RRMS (18 and older). Gilenya became the first disease-modifying therapy approved by the U.S. Food and Drug Administration (FDA) for children and adolescents with relapsing MS, starting at age 10, in May.
The European Commission will now review CHMP’s opinion and is expected to reach a final decision within three months. Its decision will be applicable to all 28 European Union member states plus Iceland, Norway and Liechtenstein.
The positive opinion was based on results from the PARADIGMS Phase 3 trial (NCT01892722) in 215 relapsing MS patients, ages 10 to 17, treated either with Gilenya 0.25 or 0.5 mg capsules once daily (depending on the child’s weight) or intramuscular injections of Avonex (interferon beta-1a, marketed by Biogen) at a weekly dose of 30 mg.
PARADIGMS – supported by Novartis – evaluated the safety and efficacy of Gilenya for up to two years, followed by a five-year open-label extension phase. Included patients had an Expanded Disability Status Scale (EDSS) score between 0 and 5.5. Of note, EDSS ranges from 0 to 10, with higher scores representing greater disability.
Results showed that, compared to Avonex, Gilenya significantly lowered the rate of relapses by 82%, and delayed the time to first relapse in patients treated for up to 24 months. After two years of treatment, 85.7% of patients given Gilenya were relapse-free, compared to 38.8% given Avonex. Gilenya also reduced the annual rate of brain shrinkage, or atrophy, by 40%, and significantly lowered the number of new or newly enlarged lesions, as assessed magnetic resonance imaging. Full results were recently published.
“We are very excited to be a step closer to bringing this much-needed treatment to young MS patients across Europe,” Paul Hudson, Novartis’s CEO, said in a press release. He added that CHMP’s positive opinion “is a testament” to Novartis’ commitment to develop MS care across all generations.
Patients diagnosed with MS at young ages may experience two-to-three times as many relapses as adults, often associated with a more severe prognosis and earlier disability.
“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,” Hudson said. “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.”
Pedro Carrascal, president of the European Multiple Sclerosis Platform, concluded: “Today’s CHMP positive opinion is a momentous advancement for the children and adolescents impacted by MS.” He added that he hoped young European patients and their families could soon “have a new treatment option to alleviate the devastating impact of this condition.”