Novartis multiple sclerosis therapy Fingolimod granted FDA Breakthrough Therapy designation for pediatric MS

Breakthrough Therapy designation can expedite the development and review of therapies for serious conditions(1)– In a pivotal Phase III study, oral fingolimod significantly reduced relapses by 82% in a pediatric patient population vs. interferon beta-1a intramuscular injection(2)– Currently, no disease-modifying therapies are approved for pediatric patients with MS, who often have more frequent relapses than adults with early MS(3)

Novartis 

EAST HANOVER, N.J., Dec. 18, 2017 /PRNewswire/ — Novartis today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for fingolimod for the treatment of children and adolescents 10 years of age or older with relapsing multiple sclerosis (MS). Fingolimod, also known as Gilenya® in the US, is approved to treat relapsing forms of MS in adults. Gilenya is not currently approved for children and adolescents with relapsing MS.


The Breakthrough Therapy designation is based on data from the Phase III PARADIGMS study, which evaluated the safety and efficacy of fingolimod vs. interferon beta-1a in children and adolescents (ages 10 or older) with relapsing MS4. PARADIGMS, the first completed randomized, controlled clinical trial specifically designed for pediatric relapsing MS, found that treatment with fingolimod resulted in an 82% reduction in the rate of relapses (annualized relapse rate) in this patient population over a period of up to two years, compared to interferon beta-1a intramuscular injection (p <0.001)2. The safety profile of fingolimod in this study was overall consistent with that seen in previous clinical trials in adults5.


“Despite the fact that children experience approximately two to three times as many relapses as a typical adult onset MS patient, there are currently no disease-modifying therapies approved for the pediatric population,” said Dr. Tanuja Chitnis, Director of the Partners Pediatric Multiple Sclerosis Center, Massachusetts General Hospital, Boston, US, and Scientist, Ann Romney Center, Brigham and Women’s Hospital, Boston, US. “Children with MS differ from adults in important ways and additional treatment options for pediatric patients are needed,” added Dr. Chitnis, who also served as principal investigator for the PARADIGMS study.


The FDA grants Breakthrough Therapy designation for therapies that are intended to treat a serious condition and that have preliminary clinical evidence indicating that the treatment may demonstrate substantial improvement over available therapy on one or more clinically significant endpoints. This designation is a process designed to expedite the development and review of such therapies1.



“We’re proud of this regulatory milestone, which represents part of our commitment to advance treatment options for young people with MS,” said Fabrice Chouraqui, President of Novartis Pharmaceuticals Corporation. “Novartis is looking forward to working with the FDA to bring a therapy with a long track record in adults with relapsing MS to this younger patient population as soon as possible.”


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