FDA Okays Two Hepatitis C Drugs for Pediatric Patients

The US Food and Drug Administration (FDA) today granted approval for supplemental applications for sofosbuvir (Sovaldi) and ledipasvir and sofosbuvir (Harvoni) to treat hepatitis C virus (HCV) in children ages 12 to 17.

Most children who have HCV are infected at birth through vertical transmission of infection from mother to child. If a mother is diagnosed with HCV, there is a 1 in 20 chance of her child becoming infected at birth. According to a report by the Liver Foundation, there are an estimated 23,000 to 46,000 children in the United States with HCV — the condition occurs in approximately 0.15% of 6-11 year olds and 0.4% of 12-19 year olds.

While Harvoni and Sovaldi, both marketed by Gilead Sciences, were previously approved to treat HCV in adults, these are the first direct-acting antiviral treatments approved for children and adolescents with HCV.

According to researchers, the approvals will provide pediatric treatment options for 6 major genotypes of HCV. Sovaldi, combined with ribavirin, is indicated to treat pediatric patients 12 years older or weighing at least 77 pounds (35 kilograms) with genotype 2 or 3 HCV infection without cirrhosis or with mild cirrhosis.

Harvoni is indicated for the treatment of pediatric patients 12 years and older or weighing at least 77 pounds (35 kilograms) with HCV genotype 1, 4, 5 or 6 infection without cirrhosis (liver disease) or with mild cirrhosis.

The approval for the new indication was based on an open-label, multicenter clinical trial including 100 pediatric patients 12 years and older looking at the safety, pharmacokinetics, and efficacy of Harvoni to treat HCV genotype 1 infection. The study results mirrored that of adults and demonstrated that 98% patients had no virus detected in the blood 12 weeks after finishing treatment, suggesting the patients’ infections were cured.

Also, the safety and efficacy of Harvoni for treating HCV genotypes 4, 5, or 6 infection in pediatric patients 12 years and older were assessed by looking at data showing similar exposures 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.

The most commonly reported adverse events with Sovaldi in combination with ribavirin were fatigue and headache. Officials cautioned that health care professionals should screen all patients for evidence of current or prior HBV infection before starting treatment with Harvoni or Sovaldi.

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