Safety and Efficacy of Delayed-Release Dimethyl Fumarate in Pediatric Patients with Relapsing Multiple Sclerosis (FOCUS)

Publication date: Available online 22 March 2018
Source:Pediatric Neurology
Author(s): Raed Alroughani, Rajiv Das, Natasha Penner, Joe Pultz, Catherine Taylor, Satish Eraly
BACKGROUNDNo therapies have been formally approved by the FDA for use in pediatric multiple sclerosis (MS), a rare disease.OBJECTIVETo evaluate the safety, efficacy, and pharmacokinetics of dimethyl fumarate (DMF) in pediatric patients with MS.METHODSFOCUS, a phase 2, multicenter study of patients aged 10 to 17 years with relapsing-remitting MS (RRMS), comprised an 8-week baseline and 24-week treatment period; during treatment, patients received DMF (120 mg twice a day [BID] days 1–7; 240 mg BID thereafter). Magnetic resonance imaging (MRI) scans were obtained at week –8, day 0, week 16, and week 24. The primary endpoint was the change in T2 hyperintense lesion incidence from the baseline period to the final 8 weeks of treatment. Secondary endpoints were pharmacokinetic parameters and adverse event (AE) incidence.RESULTSTwenty of 22 enrolled patients completed the study. There was a significant reduction in T2 hyperintense lesion incidence from baseline to the final 8 weeks of treatment (P = 0.009). Adverse events (most commonly gastrointestinal events and flushing) and pharmacokinetic parameters were consistent with adult findings. No serious AEs were considered DMF related.CONCLUSIONDMF treatment was associated with a reduction in MRI activity in pediatric patients; pharmacokinetic and safety profiles were consistent with those in adults. DMF is a potential treatment for pediatric MS.



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