Pediatric Updates From the Food and Drug Administration

Marcia L. Buck, PharmD, FCCP, FPPAG, BCPPS


Pediatr Pharm. 2019;25(4) 

In This Article

New and Extended Pediatric Indications

In addition to manufacturer incentives provided through the FDA's Pediatric Research Equity Act (PREA), the National Institutes of Child Health and Human Development (NICHD) provides funding for research in off-patent drugs through the Best Pharmaceuticals for Children Act (BCPA). A total of 773 pediatric labeling changes have been made since the inception of these programs.[6] Labeling changes include not only new indications and age-based extensions, but also new pharmacokinetic, dosing, and safety information. The following examples highlight the range of new information and extended indications approved during the past 15 months.


Use of dasatinib (Sprycel®) was extended to pediatric patients one year and older with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia in combination with chemotherapy. Efficacy in newly diagnosed B-cell precursor Ph+ ALL was evaluated in a single cohort of 78 children, median age 10 years, taking part in a larger multicenter, multiple-cohort study. The 3-year event-free survival rate was 64.1% (95% CI: 52.4, 74.7). At the end of induction, 75 patients (96%) had a bone marrow biopsy with <5% lymphoblasts; 76 patients (97%) achieved this goal by the end of consolidation.


The approval for dupilumab (Dupixent®) for the treatment of uncontrolled moderate-to-severe atopic dermatitis was recently extended to patients 12 to 17 years of age. The drug targets the interleukin (IL-4/IL-13) pathway and is the first monoclonal antibody for atopic dermatitis to be approved in the pediatric population. Dupilumab can be used with corticosteroids or alone. In a phase 3 trial, dupilumab produced significantly greater improvement in the Eczema Area and Severity Index compared to placebo (66% versus 24%).


The indication for fosaprepitant injection (Emend®) has been extended to include infants and children 6 months of age and older for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy and delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic cancer chemotherapy. This approval was based on extrapolation of data from studies of fosaprepitant injection in adults, as well as additional safety, efficacy and pharmacokinetic data in pediatric patients. Efficacy was also supported by data from a controlled study of an oral 3-day aprepitant regimen in pediatric patients 6 months to 17 years.


The indication for perampanel (Fycompa®) has been extended to include treatment of partial onset seizures, with or without secondarily generalized seizures, in patients 4 years and older. Use of perampanel in this population is based on evidence from several controlled trials, including an open-label study with a 41-week extension phase, pharmacokinetic data from adult and pediatric patients, and safety data in 225 pediatric patients 4 years to less than 12 years of age.[7]


Approval for voriconazole (Vfend®) was extended to patients 2 years of age and older with invasive aspergillosis, disseminated candidiasis, and candidemia. The approval was based on the results of two multicenter open-label non-comparative trials in children 2 to 18 years of age. Thirty-one children were enrolled in the first study. Global response, defined as clinical improvement and a 50% or greater reduction in radiologic lesions, was reported in 40% of the patients 2–11 years of age and 75% of the group 12 years and older. The second study enrolled 22 children 2–18 years of age. Global response rates were 70% in the group with invasive candidiasis and 86% in those with esophageal candidiasis.