FDA approves oral pellet formulation of Epclusa®

The US Food and Drug Administration has approved an extension of indication in pediatrics for Epclusa® (sofosbuvir/velpatasvir), a treatment for chronic hepatitis C virus. The approval allows the drug from Gilead Sciences, Inc. are used in children from 3 years of age without regard to hepatitis C virus genotype or severity of liver disease. 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 unable to swallow tablets.

A Phase 2, open-label clinical trial formed the basis for the approval. The study included 41 children aged 3 years to less than 6 years who were treated with sofosbuvir/velpatasvir for 12 weeks. After the course of treatment, the medication had a sustained virologic response or cure rate of 83% in all patients; 88% in children with 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. Of the children who did not achieve a cure, all discontinued treatment 1 to 20 days after initiation of treatment.

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


1. Gilead Sciences, Inc. U.S. Food and Drug Administration Approves New Formulation of Epclusa®, Expanding Pediatric Indication for the Treatment of Children 3 Years of Age and Old 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- children-age-3-and-parent-with-chronic-treating

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