The approval of Epclusa for younger hepatitis C patients is based on the data from a Phase 2 open-label study
FDA Building 51 is one of the main buildings in its White Oak campus that houses the Center for Drug Evaluation and Research. (Credit: The U.S. Food and Drug Administration/Wikipedia)
The US Food and Drug Administration (FDA) has approved the expanded indication of Gilead Sciences’ Epclusa (sofosbuvir/velpatasvir) to include children aged three and older with chronic hepatitis C (HCV).
The latest indication includes children of that age irrespective of their HCV genotype or severity of the liver disease.
It also includes a new drug application (NDA) approval for two strengths of an oral formulation, dependent on the patient weight, of sofosbuvir/velpatasvir (200 mg/50mg, 150mg/37.5mg) for administration in young children who cannot swallow tablets.
Gilead Sciences chief medical officer Merdad Parsey said: “Gilead remains steadfast in our commitment to supporting HCV elimination. Today’s decision by the FDA represents important progress toward that goal by expanding more cure options for children living with HCV.
“This approval adds to the robust clinical evidence supporting the safety and efficacy of Epclusa across a broad set of patients, including those with end-stage renal disease and all stages of fibrosis.”
The FDA approval of Epclusa for younger hepatitis C patients is based on the data from a Phase 2 open-label study.
The clinical trial enrolled 41 children aged three years to less than six years to be treated with Epclusa for 12 weeks.
Upon completion of the treatment at 12 weeks, the drug attained a sustained virologic response (SVR12) or cure rate of 83% among all patients, 88% in children with HCV genotype 1, 50% in children with HCV genotype 2, and 100% in children with HCV genotype 3 and HCV genotype 4.
In the trial, seven patients who did not achieve a cure were discontinued treatment within one to 20 days of starting treatment.
The paediatric study lead investigator Karen Murray said: “Treating paediatric HCV remains an important public health priority. The Phase 2 clinical trial results previously showed that this medication was effective in treating many HCV-infected patients, regardless of genotype.
“Now, the expanded approval and oral pellet formulation offer new treatment strategies in younger patients with HCV.”