FDA Approves Tenofovir Alafenamide Treatment for Chronic Hepatitis B Virus in Pediatric Patients 12 and Older

The US Food and Drug Administration (FDA) has approved 25 mg tablets of tenofovir alafenamide (Vemlidy) as a once-per-day treatment for pediatric patients with chronic hepatitis B virus (HBV) infection who are 12 years and older and have compensated liver disease.

The drug treatment’s approval by the FDA was announced by Gilead Sciences, Inc. They note that the drug is a novel, targeted prodrug of tenofovir previously approved by the FDA in 2016 as a once-daily treatment for adults with these conditions.

Now, it has been recommended as a first-line treatment for adults with chronic HBV and compensated liver disease.

The investigators noted that chronic HBV can lead to significant long-term health impacts for younger patients, including such illnesses as liver cancer if the disease remains untreated throughout a patient’s life.

“As a clinician, I recognize the critical importance of treating this disease as quickly as possible to help avoid complications and potential damage to the liver,” said Kathleen Schwarz, MD, Pediatric Gastroenterologist for Rady Children’s Hospital-San Diego, and an investigator in the Vemlidy clinical trial. “In the clinical trial, we saw that tenofovir alafenamide may represent an effective treatment option for people as young as 12 years of age living with this chronic disease.”

The approval of tenofovir alafenamide for these conditions was supported by data gathered from Phase 2 clinical trials which compared the treatment with 25 mg of a placebo in a group of 70 treatment-naïve and experienced patients ages 12 to just under 18 years.

The trial was able to meet its primary endpoint of percentage of patients with HBV DNA levels below 20 IU/mL by 24 weeks of therapy, with researchers finding that 21% of patients treated achieved HBV DNA <20 IU/mL by 24 weeks in comparison to 0% with placebo.

“While pediatric hepatitis B prevalence has dropped significantly in the U.S., children who develop chronic hepatitis B following an acute infection can experience lifelong health impact,” Merdad Parsey, MD, PhD, the Chief Medical Officer of Gilead Sciences, explained. “Gilead is focused on meeting the biggest challenges in liver disease and impacting the course of disease. With an established safety profile and once-daily dosing, Vemlidy provides physicians a new option to address the treatment needs of pediatric patients living with hepatitis B.”