FDA OKs Multiple Sclerosis Therapy for Pediatric Patients


Fingolimod (Gilenya, Novartis) is the first FDA-approved treatment to treat multiple sclerosis in pediatric patients.

Officials with the FDA have approved fingolimod (Gilenya, Novartis) for the treatment of relapsing multiple sclerosis (MS) in pediatric patients aged 10 years and older, making it the first FDA-approved drug to treat MS in this population, according to a press release.

Fingolimod was initially approved by the FDA in 2010 to treat adults with relapsing MS. It has previously been granted priority review and breakthrough therapy designation for its newest indication.

According to the FDA, 2% to 5% of patients with MS experience symptoms before the age of 18 and estimates suggest that 8000 to 10,000 children and adolescents in the United States have MS. For most patients with MS, episodes of symptoms including worsening function are initially followed up with periods of remission, but over time patients may experience progressive decline in function and increased disability.

The approval is based on a clinical trial evaluating fingolimod’s efficacy in treating pediatric patients with MS. In the trial, 214 patients aged 10 to 17 years old were evaluated, comparing fingolimod to another MS drug, interferon beta-1a.

According to the results, 86% of patients who received fingolimod remained relapse-free after 24 months of treatment, compared to 46% of those receiving interferon beta-1a.

Adverse effects associated with fingolimod in the trial were similar to those seen in adults, including headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain, and pain in extremities.

According to the press release, fingolimod must be dispensed with a patient Medication Guide describing important information about the drug’s use and risks, especially serious risks such as slowing of the heart rate, especially after the first dose. Because fingolimod may increase risk of infection, patients should be monitored for infection during treatment and for 2 months after discontinuation of treatment. A rare brain infection called progressive multifocal leukoencephalopathy has been reported in patients treated with fingolimod.

“For the first time, we have an FDA-approved treatment specifically for children and adolescents with multiple sclerosis,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in the press release. “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.”


FDA expands approval of Gilenya to treat multiple sclerosis in pediatric patients [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm607501.htm?utm_campaign=05112018_FDA%20approves%20Galenya&utm_medium=email&utm_source=Eloqua. Accessed May 11, 2018.

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