Epclusa (sofosbuvir and velpatasvir) received approval for a supplemental application to treat hepatitis C virus (HCV) in children without cirrhosis or with mild cirrhosis, the U.S. Food and Drug Administration announced Thursday.

The supplemental application of Epclusa is indicated for children ages 6 years and older or weighing at least 37 lb who have HCV genotypes 1, 2, 3, 4, or 6. Epclusa combined with ribavirin is indicated for treating children 6 years and older or at least 37 lb with severe cirrhosis, and the FDA previously approved the drug to treat HCV in adults.

Approval was based on pharmacokinetic, safety, and efficacy data from an open-label, multicenter trial of 173 treatment-naive and treatment-experienced pediatric patients 6 years and older who did not have cirrhosis or had mild cirrhosis. Children received an oral dose of Epclusa for 12 weeks. The researchers found comparable results for pharmacokinetics, safety, and efficacy to those seen in adults. Among 102 patients aged 12 to 17 years, 93 percent of those with genotype 1 and 100 percent of those with genotypes 2, 3, 4, and 6 had no detectable virus 12 weeks posttreatment. Among 71 patients aged 6 to 11 years, 93 percent with genotype 1, 91 percent with genotype 3, and 100 percent with genotypes 2 and 4 had no detectable virus 12 weeks posttreatment. For patients aged 6 years and older with genotype 5, the FDA noted that safety and efficacy for the supplemental application are supported by exposure to sofosbuvir and velpatasvir in adults with HCV genotypes 1, 2, 3, 4, or 6.

The most commonly reported adverse reactions to Epclusa included fatigue and headache, which are consistent with the adverse events reported in adult clinical trials. A boxed warning included with Epclusa cautions that patients with both HCV and hepatitis B virus (HBV) who were taking or had completed HCV antivirals but were not taking HBV antivirals reported reactivation of HBV. All patients should be tested for current or prior HBV infection before starting Epclusa.

Approval was granted to Gilead Sciences.

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