FDA Grants Gilenya^® Breakthrough Therapy Designation for Pediatric MS

On December 18, 2017, Novartis announced that Gilenya® (fingolimod) was granted breakthrough therapy designation by the United States Food and Drug Administration (FDA) for the treatment of children and adolescents ages 10 years or older with relapsing multiple sclerosis (MS). Known as “pediatric MS,” nearly all (98 percent) of those diagnosed experience the relapsing form of the disease. Currently, all 16 disease-modifying therapies (DMTs) for MS have been approved for adults; no treatment has been approved specifically for children and adolescents with MS.

The FDA’s breakthrough therapy designation is designed to expedite the development and review of a medication. Gilenya’s designation 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 or older) 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.

In an announcement released by Novartis, the principal investigator of the study, Dr. Tanuja Chitnis, explained that children experience approximately two-to-three times as many relapses as an individual with adult-onset MS. Dr. Chitnis also expressed the need for additional treatment options for pediatric patients.

Gilenya was initially approved by the FDA in September 2010 as a first-line treatment for adults with relapsing forms of MS. Taken as a once-daily pill, Gilenya was the first oral DMT approved for the long-term treatment of MS. Prior to this time, all DMTs were given either via self-injection or via intravenous (IV) infusion – typically at a hospital or infusion center.

The breakthrough therapy designation has been implemented through the FDA since July 2012. According to the FDA, a breakthrough therapy is a medication that (1) is under investigation for the treatment of a serious or life-threatening disease or condition, and (2) preliminary clinical evidence indicates that the medication may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. When a medication is designated as a breakthrough therapy, the FDA will expedite the development and review of the medication.

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 Barry Singer, MD, MS Neurologist, MSAA Board Member, and Director of The MS Center for Innovations in Care at Missouri Baptist Medical Center