FDA Approves New Indications for Two HCV Drugs

Today, the US Food and Drug Administration (FDA) approved two hepatitis C (HCV) drugs for use in pediatric patients. According to the FDA press release, prior to this designation, there were no direct-acting antiviral HCV drugs approved for use in children or adolescents.

The Centers for Disease Control and Prevention (CDC) and the World Health Organization (WHO) recommend testing children born to mothers with HCV for infection. There are approximately 150 million people living with HCV infection around the world, and about 700,000 people die annually due to HCV-associated liver disease. The CDC estimates that there are between 23,000 and 46,000 children with HCV infection living in the United States alone. Unfortunately, there is no vaccine for HCV.

Gilead Sciences’ Sovaldi (sofosbuvir) and Harvoni (ledipasvir and sofosbuvir) just received FDA approval for designated use in child patients between the ages of 12 and 17. These two drugs were initially FDA-approved to treat HCV in adults.

Sovaldi and Harvoni are now approved to treat six HCV genotypes in these populations. Harvoni has been FDA approved for genotypes 1, 4, 5, or 6 for patients without cirrhosis or with mild cirrhosis, ages 12 and older, and who weigh at least 35 kilograms (77 pounds). Sovaldi has been approved for use in combination with ribavirin for HCV genotypes 2 or 3 in the same population.

An open-label, multicenter clinical trial studied the safety, pharmacokinetics, and efficacy of Harvoni on 100 patients aged 12 years and older who were infected with HCV genotype 1. Researchers found that 98% of the study patients showed no viremia 12 weeks after receiving final treatment dose, “suggesting the patients’ infections were cured.” These results are similar to those of adult patients receiving Harvoni. Harvoni’s safety and efficacy for treating HCV genotypes 4, 5, or 6 in this pediatric population, “is based on data showing similar exposures (amount of drug in the body) to Harvoni in adults and adolescents with HCV genotype 1 infection, as well as similar efficacy and exposures to Harvoni across HCV genotypes 1, 4, 5 and 6 in adults.”

In addition, an open-label clinical trial examined the safety and efficacy of Sovaldi in combination with ribavirin in 50 patients aged 12 years and older. The combination therapy was 100% successful in treating HCV genotype 2, and 97% successful in treating HCV genotype 3 in this population after 12 weeks of final treatment dose. According to the press release, “These results are comparable to those observed in adults.”

Common reactions to Harvoni and the combination therapy of Sovaldi + ribavirin in this population were fatigue and headache. The press release noted, “Hepatitis B virus (HBV) reactivation has been reported in HCV/HBV coinfected adult patients who were undergoing or had completed treatment with HCV direct-acting antivirals, and who were not receiving HBV antiviral therapy. HBV reactivation in patients treated with direct-acting antiviral medicines resulted in serious liver problems or death. Health care professionals should screen all patients for evidence of current or prior HBV infection before starting treatment with Harvoni or Sovaldi.”

According to the Director of the Office of Antimicrobial Products at the FDA Center for Drug Evaluation and Research, Edward Cox, MD, “These approvals will help change the landscape for HCV treatment by addressing an unmet need in children and adolescents.”