Therapeutic efficacy of Epclusa

Multiple phase 3 clinical trials evaluated the efficacy and safety of Epclusa (Sofosbuvir/Velpatasvir) in patients with different HCV genotypes, different disease stages (without cirrhosis/compensated cirrhosis/decompensated cirrhosis), and special populations (HCV/HIV-1 coinfection, post-liver transplantation, severe renal impairment requiring dialysis, injection drug users, and pediatric patients).

Primary Efficacy Endpoint

At 12 weeks after the end of treatment, HCV RNA was below the lower limit of quantification (SVR12, Sustained Virologic Response at Week 12).

Summary of Key Results

HCV Genotypes 1, 2, 4, 5, 6 (without cirrhosis/compensated cirrhosis): The SVR12 rate of 12-week Epclusa treatment was as high as 98%–100%.

HCV Genotype 2 (without cirrhosis/compensated cirrhosis): The SVR12 rate of 12-week Epclusa treatment (99%) was superior to that of 12-week sofosbuvir + ribavirin treatment (94%).

HCV Genotype 3 (without cirrhosis/compensated cirrhosis): The SVR12 rate of 12-week Epclusa treatment (95%) was superior to that of 24-week sofosbuvir + ribavirin treatment (80%). Patients with compensated cirrhosis and Y93H substitution had a higher failure rate.

HCV/HIV-1 Coinfection (without cirrhosis/compensated cirrhosis): The SVR12 rate of 12-week Epclusa treatment was 95%.

Decompensated Cirrhosis (Child-Pugh Class B): The SVR12 rate of 12-week Epclusa treatment combined with ribavirin (94%) was superior to that of 12-week or 24-week Epclusa monotherapy.

Liver Transplant Recipients (without cirrhosis/compensated cirrhosis): The SVR12 rate of 12-week Epclusa treatment was 96%.

Severe Renal Impairment Requiring Dialysis: The SVR12 rate of 12-week Epclusa treatment was 95%.

Injection Drug Users (including MAT, Medication-Assisted Treatment): The SVR12 rate of 12-week Epclusa treatment was 94%.

Pediatric Patients (aged 3 years and older, without cirrhosis/compensated cirrhosis): The overall SVR12 rate of 12-week Epclusa treatment was favorable across different age groups and genotypes. However, it was relatively lower in the 3 to <6 years age group (83%) and in children with genotype 2 within this group (50%), which was partially associated with treatment discontinuation due to vomiting/drug regurgitation.

FDA，2022.04