The Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for Epclusa® (sofosbuvir/velpatasvir; Gilead Sciences) for the treatment of pediatric patients aged ≥6 years weighing at least 17kg with chronic hepatitis C virus (HCV) genotype 1, 2, 3, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis; or in combination with ribavirin in those with decompensated cirrhosis. Previously, the combination therapy had only been approved for adults.
Epclusa is a fixed-dose combination of sofosbuvir, a hepatitis C virus (HCV) nucleotide analog NS5B polymerase inhibitor, and velpatasvir, an HCV NS5A inhibitor. The approval was based on data from a 12-week, open-label, phase 2 trial (Study 1143) that evaluated the efficacy and safety of Epclusa in 173 patients aged ≥6 years with chronic HCV infection without cirrhosis or with compensated cirrhosis.
Results demonstrated the following sustained virologic responses at 12 weeks (SVR12) by age group and genotype:
- Patients aged 12 to <18 years: SVR12 of 93% in genotype 1 HCV infection (n=71/76); and SVR12 of 100% in genotype 2 (n=6/6), genotype 3 (n=12/12), genotype 4 (n=2/2), genotype 6 (n=6/6).
- Patients aged 6 to <12 years: SVR12 of 93% in genotype 1 HCV infection (n=50/54); 91% in genotype 3 (n=10/11); and 100% in genotype 2 (n=2/2) and genotype 4 (n=4/4).
With regard to safety, adverse reactions in pediatric patients were consistent with those observed in adult patients treated with Epclusa.
The safety and effectiveness of Epclusa for treatment of HCV genotype 5 in pediatric patients ≥6 years weighing at least 17kg without cirrhosis or with compensated cirrhosis are supported by sofosbuvir and velpatasvir exposures in adults and pediatric patients. Similar rationale was used to support dosing recommendations for pediatric patients with HCV genotype 1, 2, 3, 4, 5, or 6 infection who have decompensated cirrhosis (Child-Pugh B or C).
The product is available as 400mg/100mg and 200mg/50mg strength tablets in 28-count bottles. The recommended dosage for pediatric patients is based on weight.
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