The U.S. Food and Drug Administration today approved a supplemental application for Epclusa (sofosbuvir and velpatasvir) to treat hepatitis C virus (HCV) in children ages 6 years and older or weighing at least 37 pounds (17 kilograms) with any of the six HCV genotypes—or strains—without cirrhosis (liver disease) or with mild cirrhosis. Epclusa in combination with ribavirin is indicated for the treatment of pediatric patients 6 years and older or weighing at least 37 pounds with severe cirrhosis. The FDA previously approved Epclusa to treat HCV in adults.
“This approval will provide additional treatment options for children and adolescents with HCV,” said Debra Birnkrant, M.D., 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.”
HCV is a viral disease that causes inflammation of the liver that can lead to diminished liver function or liver failure. According to the Centers for Disease Control and Prevention, in 2016 there were an estimated 2.4 million people in the United States with chronic HCV, and children born to HCV-positive mothers are at risk for HCV infection.
The pharmacokinetics (how the body absorbs, distributes and rids itself of a drug), safety and efficacy of Epclusa, taken orally for 12 weeks, for the treatment of HCV genotypes 1, 2, 3, 4 or 6 infection was established in an open-label, multicenter clinical trial that included a total of 173 treatment-naïve and treatment-experienced pediatric patients ages 6 years and older without cirrhosis or with mild cirrhosis. No meaningful differences in pharmacokinetics were seen in pediatric patients compared to adults. The safety and efficacy results were comparable to those observed in adults. In 102 patients ages 12 through17, 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 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.
The safety and efficacy of Epclusa for treatment of HCV genotype 5 in pediatric patients 6 years and older or weighing at least 37 pounds without cirrhosis or with mild cirrhosis are supported by sofosbuvir and velpatasvir exposures in adults and pediatric patients with HCV genotype 1, 2, 3, 4 or 6 infection. Similar data were used to support dosing recommendations for pediatric patients with HCV genotype 1, 2, 3, 4, 5 or 6 infection who have severe cirrhosis.
The safety and effectiveness of Epclusa have not been established in pediatric patients less than 6 years of age.
The most common adverse reactions observed with treatment with Epclusa were fatigue and headache. The adverse reactions observed were consistent with those observed in clinical trials of Epclusa in adults.
Epclusa 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. HBV reactivation has resulted in hepatitis that occurs suddenly and escalates quickly (fulminant), kidney failure and death. Health care providers should test all patients for evidence of current or prior HBV infection before initiation of Epclusa and continue to monitor patients throughout the treatment.
The FDA granted Priority Review of Epclusa to Gilead Sciences Inc.