US FDA has approved a supplemental New Drug Application (sNDA) 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. The recommended dosage of Epclusa in children ages 6 years and older is based on weight and liver function. Epclusa is the first pan-genotypic, protease inhibitor-free regimen approved in the United States for adults and children.
Clinical Basis of approval
- 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 per cent (71/76) in those with genotype 1 HCV infection and 100 per cent 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 per cent (50/54) in those with genotype 1 HCV infection, 91 per cent (10/11) in those with genotype 3 HCV infection, and 100 per cent 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 per cent, all grades) observed with treatment with Epclusa for 12 weeks in adults are headache and fatigue.
- For adults, Epclusa was first approved by the US 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.