As the options for treating hepatitis C have expanded dramatically over the last 3 years for adult patients, news this week indicate that the benefits of hepatitis C therapy are now extending into pediatric populations. Sofosbuvir and the combination ledipasvir/sofosbuvir is now approved for children aged between 12 and 17 years who have chronic HCV infection.
While this population comprises only a small fraction of the number of people infected with HCV in the United States, it does represent between 25,000 and 40,000 children who will benefit from the approval of these two therapeutic options.
Sofosbuvir is approved for use in all genotypes, but it requires being paired with other drugs to have its maximum effectiveness. For genotype 2, sofosbuvir needs to be paired with ribavirin and it is typically not used for genotype 3 unless paired with velpatasvir, which is not yet approved for use in pediatric populations. For all other genotypes including 1, 4, 5, and 6 sofosbuvir/ledipasvir is now available for these younger patients and can be used to cure their infections.
In clinical trials, the activity of sofosbuvir combined with ledipasvir or other agents was able to eradicated HCV in up to 98% of children, mirroring the findings in adults. Moreover, the pharmacokinetic profiles indicated that these drugs will be safe in this younger population of patients.
While it is not a surprise that these drugs are now available for pediatric populations, it is welcome news.
Owing to the need for the regulatory agency to prove not only efficacy, but also safety in younger populations, it is very common for drug approvals to be delayed, often by a period of years relative to the adult population. Hopefully, several of the other HCV drugs will be approved for this younger population and, ultimately, for children aged younger than 12.
We eagerly await clinical studies that can document safety and efficacy in these younger populations as well.
Michael S. Saag, MD
Co-Chief Medical Editor
Disclosures: Saag reports no relevant financial disclosures.