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Fingolimod’s (Gilenya®) expanding role in the treatment of pediatric multiple sclerosis

By: Michael Lim, PharmD Candidate c/o 2020

In 2010, the Food and Drug Administration (FDA) approved fingolimod (Gilenya®) for the treatment of adults with relapsing multiple sclerosis (MS).1 Historically, fingolimod has been used as a convenient second line oral treatment upon failure of initial disease-modifying therapy for relapsing-remitting MS in adult populations.2 Therefore, when a patient is refractory to treatment with first line disease-modifying agents such as glatiramer acetate (Copaxone®) or one of the beta interferons such as interferon beta-1b (Betaseron®), he or she may be placed on fingolimod. In May of 2018, the FDA announced the approval of fingolimod for the treatment of relapsing MS in children that are ten years of age or older.1 The approval marked a milestone in MS treatment with fingolimod becoming the first drug approved to treat multiple sclerosis in pediatric populations.1

Evidence of fingolimod’s efficacy in pediatric patients was established in a clinical trial conducted by fingolimod’s manufacturer, Novartis.3 Comparing fingolimod against interferon beta-1a intramuscular injections, the Novartis PARADIGMS study – a phase three clinical trial – explored both the safety and efficacy of the agents in children and adolescents with MS.4 The PARADIGMS study was also the first ever randomized controlled clinical trial specifically designed to study pediatric multiple sclerosis.3 In the trial, treatment with fingolimod over a period of up to two years resulted in a statistically significant eighty two percent reduction in the annualized relapse rate compared to a forty six percent reduction found with interferon beta-1a intramuscular injections.1,3 In addition, data measured via magnetic resonance imaging demonstrated a significant reduction in newly enlarging T2 and Gd-T1 lesions in the brains of patients treated with fingolimod compared to the interferon beta-1a treatment arm.3 According to one meta-analysis of randomized trials in relapsing-remitting MS, the volume and number of such lesions were associated with increased relapses and disability progression.3,5 Thus, the MRI data further suggests fingolimod’s efficacy in pediatric patient populations.

Prior to initiation of fingolimod in pediatric patients, it is recommended that all appropriate immunizations are completed in accordance with current immunization guidelines.6 The recommended dosage of fingolimod in adults and children ten years of age and older weighing more than forty kilograms is 0.5 mg orally once daily.6 For the same group of patients weighing less than or equal to forty kilograms, the recommended dosage of fingolimod is 0.25 mg orally once daily.6 When fingolimod is initiated in pediatric patients, monitoring for signs and symptoms of bradycardia is recommended for six hours after the first dose, as initiation of this therapy results in a decreased heart rate.6 Monitoring during the six-hour period should be continued until resolution of the abnormality if heart rate is less than 45 bpm in adults, less than 55 bpm in pediatric patients twelve years of age or older, or less than 60 bpm in pediatric patients aged ten or eleven.6 In the PARADIGMS study, the side effects observed in pediatric patients were similar to those seen in adults and fingolimod’s package insert notes no difference between patient populations in terms of the drug causing increased risk of infection and progressive multifocal leukoencephalopathy.1,6

While most patients with multiple sclerosis experience their first symptoms between the ages of twenty and forty, two to five percent of people with MS experience symptom onset before the age of eighteen with estimates suggesting that 8,000 to 10,000 children and adolescents in the United States have multiple sclerosis.1 In light of fingolimod’s new role, Billy Dunn, M.D. and director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, comments, “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.”1


  1. Food and Drug Administration. FDA expands approval of Gilenya to treat multiple sclerosis in pediatric patients. Published 05/11/18. Accessed 05/15/18. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm607501.htm
  2. Michael J Olek, DO. Disease-modifying treatment of relapsing-remitting multiple sclerosis in adults. UpToDate. https://www-uptodate-com.jerome.stjohns.edu/contents/disease-modifying-treatment-of-relapsing-remitting-multiple-sclerosis-in-adults?sectionName=Fingolimod&topicRef=1686&anchor=H26726955&source=see_link#references. Updated 04/12/18. Accessed 05/16/18.
  3. Novartis PARADIGMS data show children and adolescents with MS had an 82% lower relapse rate with Gilenya® vs. interferon beta-1a. Novartis. https://www.novartis.com/news/media-releases/novartis-paradigms-data-show-children-and-adolescents-ms-had-82-lower-relapse. Published 10/28/17. Accessed 05/18/18.
  4. Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis. Full Text View – ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01892722. Published 07/04/13. Accessed 05/18/18.
  5. Sormani MP, Bruzzi P. MRI lesions as a surrogate for relapses in multiple sclerosis: a meta-analysis of randomised trials. Lancet Neurol. 2013;12(7):669-76. doi: 10.1016/S1474-4422(13)70103-0
  6. Gilenya (Fingolimod) [package insert]. East Hanover, NJ; Novartis AG; Revised 05/11/2018.
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