Combination Hepatitis C Therapy Wins FDA Approval for Pediatric Patients

The FDA today approved supplemental applications for sofosbuvir (Sovaldi) and ledipasvir/sofosbuvir (Harvoni) to treat pediatric patients with hepatitis C virus (HCV).

This is the first direct-acting antiviral treatments approved for children and adolescents aged 12 to 17.

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

Harvoni is indicated for the treatment of pediatric patients 12 years and older or weighing at least 77 pounds with HCV genotype 1, 4, 5, or 6 infection without cirrhosis or mild cirrhosis. Sovaldi plus ribavirin is indicated for the treatment of pediatric patients 12 years and older or weighing at least 77 pounds with genotype 2 or 3 HCV infection without cirrhosis or with mild cirrhosis.

The results were based on an open-label, multicenter clinical trial that included 100 pediatric patients 12 years and older to examine the safety, efficacy, and pharmacokinetics of Harvoni for the treatment of HCV genotype 1.

The findings were comparable to those observed in adults and showed that in 98% of patients, the virus was undetectable 12 weeks after treatment.

The safety and efficacy of Harvoni for treatment of HCV genotypes 4, 5, or 6 infection in pediatric patients 12 years and older is based on data that showed similar exposures to Harvoni among adults and adolescents with HCV genotype 1 infection. It also showed similar efficacy and exposures to Harvoni across HCV genotypes 1, 4, 5, and 6 in adults.

Common adverse events (AEs) with Harvoni were fatigue and headache.

The combination of sofosbuvir and Harvoni was evaluated in an open-label clinical trial that included 50 pediatric patients 12 years and older. The results were comparable to those in adults and 100% of patients with HCV genotype 2. Ninety-seven percent of patients with HCV genotype 3 had no detectable virus in the blood 12 weeks after finishing treatment.

Common AEs for the combination treatment were fatigue and headache. All contraindications to ribavirin also apply to sofosbuvir combination therapy.