On Mar. 19, 2020, Gilead Sciences announced the U.S. Food and Drug Administration (FDA) had approved a supplemental New Drug Application for Epclusa (sofosbuvir 400mg/velpatasvir 100mg; sofosbuvir 200mg/velpatasvir 50 mg) for the treatment of people with chronic hepatitis C infection (HCV) as young as 6 years of age or weighing at least 17 kg, regardless of HCV genotype or liver disease severity. Epclusa was the first pan-genotypic, protease inhibitor-free regimen approved in the U.S. for adults and children.

In the United States, there are approximately 23,000-46,000 children living with HCV. Children born to mothers with HCV are a growing concern, increasing in prevalence by 60 percent from 2011 to 2014. Additionally, engagement in high‐risk practices, such as intravenous drug use, is an increasingly common route of HCV transmission in adolescents and young adults.

The approval of Epclusa is based on data from a Phase 2, open-label clinical trial (Study 1143) that enrolled 175 children who were treated with Epclusa for 12 weeks, of which 173 were included in the efficacy analysis. In children 12 to <18 years old, treatment with Epclusa resulted in a cure rate (SVR12) of 93 percent (71/76) in those with genotype 1 HCV infection and 100 percent in those with genotype 2 (6/6), genotype 3 (12/12), genotype 4 (2/2) and genotype 6 (6/6) HCV infection.

In children 6 to <12 years old, the SVR rate was 93 percent (50/54) in those with genotype 1 HCV infection, 91 percent (10/11) in those with genotype 3 HCV infection, and 100 percent in those with genotype 2 (2/2) and genotype 4 (4/4) HCV infection. The safety profile of Epclusa in children 6 years of age and older treated was generally consistent with that observed in clinical trials in adults. The most common adverse reactions (incidence greater than or equal to 10 percent, all grades) observed with treatment with Epclusa for 12 weeks in adults are headache and fatigue.

For adults, Epclusa was first approved by the FDA and European Medicines Agency (EMA) in 2016. A line extension application for the use of Epclusa in children aged 6 to <18 years of age is currently under review with the EMA.