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.
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The approval of Gilenya for the younger patient population was supported by PARADIGMS, a double-blind, randomised, 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 (annualised 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.
The phase III PARADIGMS study (NCT01892722) is a flexible duration (up to two years), double-blind, randomised, multi-center study to evaluate the safety and efficacy of oral Gilenya compared to interferon beta-1a in children and adolescents with a confirmed diagnosis of multiple sclerosis (MS), followed by a five-year open label extension phase. The study enrolled 215 children and adolescents with MS, 10 to less than 18 years of age with an Expanded Disability Status Scale (EDSS) score between 0 and 5.5. Patients were randomised to receive once-daily oral Gilenya (0.5 mg or 0.25 mg, dependent on patients’ body weight) or intramuscular interferon beta-1a once weekly.