The FDA has approved sofosbuvir and velpatasvir (Epclusa) to treat hepatitis C virus (HCV) in children ages 6 years of age and older or weighing at least 37 pounds, and with any of the 6 HCV strains without cirrhosis or with mild cirrhosis.
Sofosbuvir and velpatasvir in combination with ribavirin is indicated for the treatment of pediatric patients 6 years of age and older or weighing at least 37 pounds with severe cirrhosis. The FDA previously approved sofosbuvir and velpatasvir to treat HCV in adults.
HCV is a viral disease that causes liver inflammation, and subsequently diminished liver function or liver failure. The CDC reported that in 2016 there were approximately 2.4 million individuals in the United States with chronic HCV; children born to mothers with HCV are also at risk for HCV infection.
The FDA’s approval of the pharmacokinetics, safety, and efficacy of sofosbuvir and velpatasvir for this indication was based on an open-label, multicenter clinical trial of 173 pediatric patients, ages 6 years or older. Some participants had had previous treatment, whereas others had no treatment experience. The patients also either did not have cirrhosis or experienced mild cirrhosis. Sofosbuvir and velpatasvir safety and efficacy has not been previously tested in patients younger than 6 years of age.
The results of the clinical trial were comparable to those observed in adult trials. In 102 patients ages 12 through 17, 93% of patients with genotype 1 and 100% of patients with genotypes 2, 3, 4, and 6 had no detectable virus in the blood 12 weeks after finishing treatment, suggesting the patients’ infection was cured. Among the 71 patients ages 6 to 11 years of age with HCV genotypes 1, 2, 3, or 4, 93% with genotype 1, 91% with genotype 3, and 100% with genotypes 2 and genotype 4 had no virus detected in the blood 12 weeks after finishing treatment.
Adverse events (AEs) of sofosbuvir and velpatasvir observed during the clinical trial included fatigue and headache, which is similar to AEs demonstrated in adults.
“This approval will provide additional treatment options for children and adolescents with HCV,” said Debra Birnkrant, MD, director of the Division of Antivirals in the FDA’s Center for Drug Evaluation and Research. “This approval will also be important in settings where there is limited ability for health care professionals to conduct HCV genotype testing.”
FDA Approves New Treatment for Pediatric Patients with Any Strain of Hepatitis C [news release]. FDA’s website. https://www.fda.gov/news-events/press-announcements/fda-approves-new-tre.... Accessed March 20, 2020.