Novartis multiple sclerosis therapy fingolimod granted FDA Breakthrough Therapy designation for pediatric MS.
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 U.S., is approved to treat relapsing forms of MS in adults. It is not currently approved for children and adolescents with relapsing MS.
Gilenya was the first once-a-day pill approved to treat relapsing multiple sclerosis (MS). Approved for first-line use, Gilenya is a disease-modifying therapy that offers freedom from injections, which may fit many patients’ lifestyles.
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 percent 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.
Currently, no disease-modifying therapies are approved for pediatric patients with MS, who often have more frequent relapses than adults with early MS3.
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.
1 US Food & Drug Administration. Breakthrough Therapy. https://www.fda.gov/forpatients/approvals/fast/ucm405397.htm. Visited November 20, 2017.
2 Chitnis T, Arnold DL, Banwell B, et al. PARADIGMS: A Randomised Double-blind Study of Fingolimod Versus Interferon β-1a in Paediatric Multiple Sclerosis. Abstract no. 276. Oral presentation at 7th Joint ECTRIMS-ACTRIMS Meeting, Paris, France, October 25-28, 2017.
3 National Multiple Sclerosis Society. Pediatric MS. https://www.nationalmssociety.org/What-is-MS/Who-Gets-MS/Pediatric-MS. Accessed November 20, 2017.
4 ClinicalTrials.gov. Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis. NCT01892722. https://clinicaltrials.gov/ct2/show/NCT01892722. Accessed November 20, 2017.
5 Novartis data on file.
(Source: Novartis Pharmaceuticals Corporation)
Filed Under: Drug Discovery