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. However, it 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 MS.
PARADIGMS, the first completed randomised, controlled clinical trial specifically designed for paediatric 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. The safety profile of fingolimod in this study was overall consistent with that seen in previous clinical trials in adults.
“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 paediatric population,” said Dr. Tanuja Chitnis, Director of the Partners Paediatric Multiple Sclerosis Centre, Massachusetts General Hospital, Boston, US, and Scientist, Ann Romney Centre, Brigham and Women's Hospital, Boston, US. “Children with MS differ from adults in important ways and additional treatment options for paediatric 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 therapies.