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FDA approves oral pellet formulation of Epclusa®

The US Food and Drug Administration (FDA) approved expanded pediatric indication for Epclusa® (sofosbuvir/velpatasvir) to treat chronic hepatitis C virus.

The US Food and Drug Administration has approved an indication expansion in pediatrics for Epclusa® (sofosbuvir/velpatasvir), which is a treatment for chronic hepatitis C virus. The approval allows the medication from Gilead Sciences, Inc. to be used in children as young as 3 years of age, with no regard for hepatitis C virus genotype or liver disease severity. The approval was for 2 strength oral pellet formulations of Epclusa (sofosbuvir 200 mg/velpatasvir 50 mg and sofosbuvir 150 mg/velpatasvir 37.5 mg) for use in children who are not able to swallow tablets.

A Phase 2, open-label clinical trial was the basis for the approval. The trial include 41 children aged 3 years to less than 6 years who were treated with sofosbuvir/velpatasvir for 12 weeks. Following the treatment course, the medication had a sustained virologic response or cure rate of 83% among all patients; 88% in children with a hepatitis C virus genotype 1; 50% in children with hepatitis C virus genotype 2; and 100% in children with hepatitis C virus genotype 3 and hepatitis C virus genotype 4. Among the children who did not achieve cure, all discontinued the treatment 1 to 20 days following the start of treatment.

Safety was overall consistent with what was seen with adults. Vomiting was reported in 15% of the subjects and 10% of the children had product use issues. They were rated as mild.


1.Gilead Sciences, Inc. U.S. Food and Drug Administration approves new formulation of Epclusa®, expanding pediatric indication to treat children ages 3 and older with chronic hepatitis C. Published June 10, 2021. Accessed June 11, 2021. https://www.gilead.com/news-and-press/press-room/press-releases/2021/6/us-food-and-drug-administration-approves-new-formulation-of-epclusa-expanding-pediatric-indication-to-treat-children-ages-3-and-older-with-chronic