The drug was previously approved to treat HCV in adults
The FDA has approved a supplemental application for sofosbuvir and velpatasvir (Epclusa, Gilead Sciences Inc) to treat any strain of hepatitis C (HCV) in children aged 6 years and older, or weighing at least 37 pounds.
The drug was previously approved to treat HCV in adults. In combination with ribavirin, the drug is indicated to treat pediatric patients aged 6 and older with severe cirrhosis.
HCV is a viral disease that causes liver inflammation, which can lead to diminished liver function or liver failure. The CDC estimates that there were 2.4 million people in the United States with chronic HCV in 2016. Children born to mothers with HCV are also at risk for infection.
The pharmacokinetics, safety, and efficacy for treatment of HCV genotypes 1, 2, 3, 4, and 6 were established in an open-label, multicenter clinical trial that included 173 treatment naïve and treatment-experienced pediatric patients aged 6 and older with no or mild cirrhosis. No meaningful differences in pharmacokinetics were seen in pediatric patients compared to adults, and the safety and efficacy results were comparable.
In 102 patients between ages 12 and 17 years, 93% of patients with genotype 1, and 100% of patients with genotypes 2, 3, 4, and 6 hd no detectable virus in the blood 12 weeks after finishing treatment. Among the 71 patients ages 6 to 11 years with HCV genotypes 1, 2, 3, or 4, 93% with genotype 1, 91% with genotype 3, and 100% with genotypes 2 and 4 had no virus detected 12 weeks after treatment.
The most common adverse reactions were fatigue and headache, consistent with reactions observed in adults.
The drug includes a boxed warning that hepatitis B virus (HBV) reactivation has been reported in patients infected with both HCV and HBV who were taking or had completed treatment with HCV antivirals and were not taking HBV antivirals.
FDA Approves New Treatment for Pediatric Patients with Any Strain of Hepatitis C [news release]. FDA website; March 19, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-pediatric-patients-any-strain-hepatitis-c?utm_campaign=031920_PR_FDA%20Approves%20New%20Treatment%20for%20Pediatric%20Patients%20with%20Any%20Strain%20of%20Hepatitis%20C&utm_medium=email&utm_source=Eloqua. Accessed March 19, 2020.