Health & Medical Children & Kid Health

New Drugs for Children in the FDA Pipeline

´╗┐New Drugs for Children in the FDA Pipeline


On January 21, 2015, UCB announced regulatory filings at the FDA and the European Medicines Agency (EMA) for brivaracetam. This drug, structurally similar to levetiracetam, has a more than 30-fold higher affinity for synaptic vesicle protein 2A (SV2A) ligand in the human cerebral cortex. Both oral and intravenous formulations are under investigation. Three phase 3 studies, enrolling over 3,000 adults and children, have been conducted to establish the safety and efficacy of brivaracetam. A recently published phase 3 randomized, double-blind, placebo-controlled trial conducted in 398 adults found that a dose of 100 mg/kg/day reduced baseline-adjusted focal seizures by 11.7% over placebo (p = 0.004). Thirty-six percent of patients receiving brivaracetam achieved a reduction in seizure frequency of 50% or greater.

An open-label pharmacokinetic, safety, and efficacy study of brivaracetam as adjunctive therapy in 101 children (1 month-16 years of age) with epilepsy was completed in 2014. In patients younger than 8 years of age, brivaracetam was initiated at a dose of 0.5 mg/kg given twice daily for the first week. The dose was increased to 1 mg/kg twice daily for week 2 and 2 mg/kg twice daily for week 3. Patients were evaluated for the presence of treatment-emergent adverse effects, change in seizure frequency, and treatment adherence at the end of week 3. After assessment, the dose was reduced in the same stepwise manner on weeks 4 and 5. For patients 8 years of age and older, the dose was initiated at 0.4 mg/kg given twice daily for the first week, followed by 0.8 mg/kg twice daily for week 2 and 1.6 mg/kg twice daily for week 3. Down-titration was done in the same manner for weeks 4 and 5. The results of the study have not yet been published. Several other studies of brivaracetam are currently enrolling patients, including a pediatric follow-up study.

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