The FDA on Thursday granted expanded approval for Gilead Sciences' Epclusa (sofosbuvir/velpatasvir), in combination with ribavirin, to include treating hepatitis C virus (HCV) in children as young as six with any of the HCV genotypes, who either have no or mild cirrhosis. According to the company, the decision makes Epclusa the first pan-genotypic, protease inhibitor-free regimen approved in the US for adults and children.
Approval was based on data from the Phase II Study 1143 that enrolled 175 children who were treated with Epclusa for 12 weeks. Results showed that in participants ages six through 11 years, sustained virologic response after 12 weeks of treatment (SVR12) with Epclusa was 93% among those with genotype 1 HCV infection, 91% in those with genotype 3, and 100% in those with genotypes 2 and 4. In children aged 12 to 17 years, the SVR12 rate was 93% for those with genotype 1 infection and 100% in those with genotype 2, 3, 4 and 6.
The FDA noted that the safety and efficacy results were comparable to those observed in adults, for whom the drug has been cleared in the US since 2016 to treat all six subtypes of HCV. Debra Birnkrant, director of the Division of Antivirals in the FDA's Center for Drug Evaluation and Research, suggested the approval "will also be important in settings where there is limited ability for healthcare professionals to conduct HCV genotype testing."
Meanwhile, chief medical officer Merdad Parsey remarked that "with consistently high cure rates in clinical trials and in the real world, Epclusa has the potential to help many of the children living with HCV in the US." Gilead estimates that there are approximately 23,000 to 46,000 children in the country who have the disease.
The company indicated that a line extension application for the use of Epclusa in children as young as six years of age is currently under review with the European Medicines Agency. It reported last month that the drug and its authorised generic version generated sales of $465 million in the fourth quarter.
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