FDA Approves Gilead’s Epclusa for Children with Hepatitis C

The U.S. Food and Drug Administration (FDA) approved Gilead Sciences’ Epclusa for Hepatitis C (HCV) for use in children as young as six years of age or who weigh at least 17 kilograms. The recommended dose of the drug for this age group is based on weight and liver function.

The drug has been on the market in the U.S. and Europe since 2016 for adults with hepatitis C. It is a pan-genotypic, protease inhibitor-free regimen. That basically means that the drug can be used regardless of the strain of hepatitis C. It is the first of this type of drug to be approved in the U.S. for both adults and children.

“While the treatment of HCV has been transformed in recent years, physicians caring for some children have still needed to take several factors into consideration, including genotype and liver disease severity, when selecting the appropriate treatment plan,” said Kathleen B. Schwarz, professor of Pediatrics, Johns Hopkins University School of Medicine. “The expanded approval of Epclusa can help eligible children living with HCV combat this life-threatening and debilitating disease.”

The approval was based on results from a Phase II, open-label trial of 175 children who received Epclusa for 12 weeks. Of those 175, 173 were included in the efficacy analysis. In children 12 years of age to just under 18, there was a cure rate (SVR12) of 93% in patients with genotype 1 HCV infection and 100% in genotype 2, 3, 4 and 6.

In the younger group, age six to just under 12 years, the SVR rate was 93% in genotype 1, 91% in genotype 3, 100% in genotypes 2 and 4. The safety profile in the overall group was generally consistent as that seen in adults. The most common side effects seen in adults receiving the drug for 12 weeks were headache and fatigue.

“Gilead’s continued commitment to HCV elimination includes bringing our medicines to the most difficult-to-cure populations and today’s decision by the FDA represents an important step toward that goal,” said Merdad Parsey, Gilead’s chief medical officer. “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 United States.”

There are about 23,000 to 46,000 children in the U.S. diagnosed with HCV. They are often born to mothers with HCV. HCV is commonly acquired through high-risk activities such as intravenous drug use.

In 2019, Epclusa brought in $971 million in the U.S., $553 million in Europe, and $441 million in other parts of the world, for a total sales of $1.965 billion. Another HCV drug, Harvoni (ledipasvir/sofosbuvir) that also contains one of the ingredients in Epclusa, (sofosbuvir/velpatasvir), brought in a total of $643 million in 2019. The company’s overall HCV product sales for 2019 were $2.9 billion, down from $3.7 billion in 2018. The company indicated that the drop was mostly due to lower average net selling price. It has also been noted that Gilead’s HCV products are a victim of their own success—the drugs are so successful at eradicating Hepatitis C that there is an increasingly smaller market for the products.