The Food and Drug Administration (FDA) has expanded the approval of Epclusa® (sofosbuvir/velpatasvir) to include patients 3 years of age and older for the treatment of 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 treatment was only approved for patients 6 years of age and older.
The New Drug Application (NDA) for an oral pellet formulation of Epclusa 200mg/50mg and 150mg/37.5mg has also been approved by the FDA for use in younger patients who have difficulty swallowing tablets. The oral pellets can be taken directly in the mouth or sprinkled on non-acidic soft food (eg, pudding, chocolate syrup, ice cream) to increase tolerability related to palatability.
The expanded approval was based on data from an open-label phase 2 trial (ClinicalTrials.gov Identifier: NCT03022981) that evaluated the efficacy and safety of Epclusa in 41 treatment-naïve patients 3 years to less than 6 years of age with genotype 1, 2, 3, or 4 HCV infection. Patients weighing less than 17kg received Epclusa 150mg/37.5mg while those weighing at least 17kg received Epclusa 200mg/50mg; both groups received treatment once daily for 12 weeks.
The sustained virologic response (SVR12) rate was 83% (n=34/41) among all patients, 88% (n=28/32) in patients with HCV genotype 1, 50% (n=3/6) in patients with HCV genotype 2, and 100% in patients with HCV genotype 3 (n=2/2) and HCV genotype 4 (n=1/1). The 7 patients who did not achieve SVR12 discontinued Epclusa within 20 days of starting treatment.
The safety profile of Epclusa in younger patients was generally consistent with that seen in adults. The most common adverse reactions reported included vomiting and product use issue (spitting up the drug).
“The phase 2 clinical trial results previously showed that this medication was effective in treating many HCV-infected patients, regardless of genotype,” said Karen Murray, MD, Chair of Cleveland Clinic Children’s and lead investigator of the pediatric study. “Now, the expanded approval and oral pellet formulation offer new treatment strategies in younger patients with HCV.”
Epclusa is currently available as 400mg/100mg and 200mg/50mg strength tablets in 28-count bottles. The new oral pellet formulation will be supplied as 200mg/50mg and 150mg/37.5mg strength unit-dose packets in 28-count cartons.
- U.S. Food and Drug Administration approves new formulation of Epclusa®, expanding pediatric indication to treat children ages 3 and older with chronic hepatitis C. [press release]. Foster City, CA: Gilead Sciences, Inc.; June 10, 2021.
- Epclusa [package insert]. Foster City, CA: Gilead Sciences, Inc.; 2021.