The therapy is already approved in Europe to treat RRMS patients 18 and older. With this newest decision, Gilenya has become the first and only oral disease-modifying treatment available for this young MS population in all 28 countries in the EU plus Iceland, Norway, and Liechtenstein.
The European Commission’s Gilenya label extension approval was based on results from the Phase 3 PARADIGMS trial, and supported by a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use, released in September.
“We are excited by the news that there is now a new approved treatment indicated for young people with RRMS in Europe — hopefully this will be the first step to having more therapy options for children and adolescents with MS,” Pedro Carrascal, president of the European Multiple Sclerosis Platform, said in a press release.
“Young patients affected by MS often experience a more severe impact during their overall lifetime. Today’s approval provides an opportunity for a brighter future for them and their families,” Carrascal added.
The Phase 3 PARADIGMS trial (NCT01892722) enrolled 215 relapsing MS patients, ages 10 to 17, who randomly received 0.25 or 0.5 mg Gilenya capsules once daily (depending on the child’s weight), or weekly intramuscular injections of Avonex (interferon beta-1a, marketed by Biogen).
Researchers evaluated the safety and efficacy of Gilenya for up to two years, followed by a five-year open-label extension phase.
After two years of treatment, 85.7% of patients given Gilenya were relapse-free, whereas a similar treatment response was only achieved by 38.8% of those treated with Avonex. Treatment with Gilenya was found to reduce by 77% the risk of MS disability progression compared with Avonex.
The therapy also reduced the annual rate of brain shrinkage, or atrophy, by 40%, and significantly reduced by 53% the number of new or newly enlarged lesions, as assessed by magnetic resonance imaging (MRI).
“Our mission is to change the course of MS as we’ve been doing since the treatment’s initial approval in 2011, and we won’t stop until we stop MS,” said Paul Hudson, CEO of Novartis Pharmaceuticals. “We are delighted that today’s decision has brought us one step closer to reimagining the treatment of MS, across all ages.”
In May, Gilenya was approved by the U.S. Food and Drug Administration as the first disease-modifying therapy for children and adolescents, ages 10 and older, with relapsing MS.