The US Food and Drug Administration (FDA) has approved the use of fingolimod (Gilenya®, Novartis, Basel, Switzerland) for the treatment of paediatric patients aged 10 to 18 years with relapsing forms of multiple sclerosis (RMS). This is the first time a disease-modifying therapy (DMT) has been approved for RMS in this age range.
A sphingosine 1-phosphate receptor modulator, Gilenya, was previously approved for adult RMS patients in 2010. It is the most prescribed oral once-daily RMS DMT, with a global exposure of more than 231,000 patients since its initial approval.1
Its new indication in children and adolescents comes following the Phase III PARADIGMS study2, a double-blind, randomised, multi-centre safety and efficacy study of Gilenya versus interferon beta-1a, designed specifically for children and adolescents with RMS. The primary endpoint demonstrated that Gilenya 0.5 mg or 0.25 mg (depending on body weight) reduced the frequency of relapses out to two years (annualised relapse rate) by around 82% (p < 0.001) when compared with interferon beta-1a intramuscular injection in 215 RMS patients aged 10 to 18 years.
“Repeated relapses are more common in young people with MS than in adults, so this is heartening news for patients and their families,” commented Brenda Banwell, Chief of the Division of Neurology at Children’s Hospital of Philadelphia, USA, who served as co-Principal investigator of PARADIGMS.1
Secondary endpoints included the number of new or newly enlarged T2 lesions, gadolinium-enhancing T1 lesions, and the safety and pharmacokinetic profiles of the drug. The safety profile of Gilenya was consistent with other previous clinical trials in adults.
- Novartis. Novartis announces FDA approval of Gilenya® as the first disease-modifying therapy for pediatric relapsing multiple sclerosis. Press release, available at: novartis.gcs-web.com.
- Chitnis T et al. PARADIGMS: A Randomised Double-blind Study of Fingolimod Versus Interferon β-1a in Paediatric Multiple Sclerosis. Abstract no. 276. Oral presentation at the 7th Joint ECTRIMS-ACTRIMS Meeting, Paris, France, October 25-28, 2017.