FDA Approves Gilenya^® for Pediatric MS

On May 11, 2018, Novartis announced that Gilenya^® (fingolimod) was approved by the United States Food and Drug Administration (FDA) for the treatment of children and adolescents, ages 10 through 17, with relapsing multiple sclerosis (MS). This is the first disease-modifying therapy (DMT) to be approved for this form of the disease in this age group.

Known as “pediatric MS,” nearly all (98 percent) of those diagnosed have the relapsing form of the disease. Children experience approximately two-to-three times as many relapses as an individual with adult-onset MS. Prior to this approval, all 15 disease-modifying therapies (DMTs) for MS have only been approved for adults; no treatment had been approved specifically for children and adolescents with MS.

The FDA’s approval of Gilenya was based on the results from the Phase III PARADIGMS study. This clinical trial evaluated the safety and effectiveness of Gilenya versus Avonex^® (interferon beta-1a) in children and adolescents (ages 10 through 17) with relapsing MS. PARADIGMS is the first study to compare two MS therapies, approved for adults, in children and adolescents with MS. Enrolling 215 young individuals with MS, this study was conducted at 87 sites in more than 25 countries. PARADIGMS was a double-blind, randomized, multi-center Phase III study with a duration of up to two years, followed by a five-year, open-label extension phase.

Study participants were either given Avonex, a once-weekly intramuscular injection, or Gilenya, an oral pill taken once daily. Compared to the Avonex group, those taking Gilenya experienced an 82-percent reduction in their annualized relapse rate over a period of up to two years. Side effects and adverse events were similar to those seen in other clinical trials with adults.

For more information on Gilenya for treating pediatric MS, please refer to MSAA’s online news article, “FDA Grants Gilenya® Breakthrough Therapy Designation for Pediatric MS,” posted in December 2017. Individuals may also visit www.gilenya.com/c/ms-pill/go-program for more information, or call 800-GILENYA (800-445-3692) to speak with a member of their support team.

For general information or to speak with a trained Client Services Specialist, please call MSAA’s Helpline at (800) 532-7667, extension 154. Questions to MSAA’s Client Services department may also be emailed to MSquestions@mymsaa.org.

Written by Susan Wells Courtney, MSAA Senior Writer

Reviewed by Dr. Jack Burks, MSAA Chief Medical Consultant