FDA Approves Combination Drug for Pediatric HCV Infection

FDA Approves Combination Drug for Pediatric HCV Infection

August 24, 2011 — The US Food and Drug Administration (FDA) has approved an expanded indication for peginterferon alfa-2a [40 KD] and ribavirin (Pegasys and Copegus, both made by Hoffman-LaRoche, Inc), allowing use of the combination treatment in pediatric patients aged 5 through 17 years with chronic hepatitis C (HCV) who have compensated liver disease and no prior history of interferon therapy.

The approval was based on the results of Study NV17424, which was conducted by the company in accordance with FDA postmarketing requirements for pediatric evaluation when approving the drug combination for adult use.

For the study, investigators randomly assigned 114 children to receive 48 weeks of treatment either with peginterferon alfa-2a (body surface area × 180 μg/1.73 m2 once weekly) plus ribavirin (~15 mg/kg/day in 2 divided doses) or with peginterferon alfa-2a plus placebo.

As previously demonstrated in adults, the addition of ribavirin to peginterferon alfa-2a therapy significantly improved the rate of sustained virologic response (HCV RNA < 50 IU/mL) at 24 weeks posttherapy (53% vs 20%). Treatment was particularly effective for eliciting a sustained response in patients with non–genotype 1 HCV compared with the difficult-to-treat genotype 1 (80% vs 47%).

The most important adverse event related to pediatric peginterferon alfa-2a/ribavirin therapy was growth delay relative to baseline: Weight and height for age z-scores and percentiles decreased during treatment but returned to normal for most children by 2 years posttherapy.

Other treatment-emergent effects were similar to those observed in adults and included influenza-like illness (91%), headache (62%), gastrointestinal disorders (56%), injection site reactions (45%), irritability (31%), fatigue (27%), rash (20%), pruritis (15%), and insomnia and decreased appetite (13% each). Dose modifications were required for about 35% of patients in each treatment group, most commonly because of neutropenia (peginterferon alfa-2a/ribavirin) and anemia (peginterferon alfa-2a monotherapy).

Peginterferon alfa-2a and ribavirin combination therapy is also approved by the FDA for the treatment of HCV in patients coinfected with HIV.