FDA Expands Tenofovir Alafenamide Indication to Treat HBV in Patients as Young as 6


Tenofovir alafenamide was originally approved by the FDA in 2016 to treat adults with chronic HBV infection with compensated liver disease and in 2022 to treat pediatric patients aged 12 years and older.

The FDA has approved a supplemental new drug application (sNDA) from Gilead Sciences for tenofovir alafenamide (Vemlidy) to treat chronic hepatitis B virus (HBV) infection in pediatric patients 6 years of age and older with compensated liver disease who weigh at least 25 kg, the company announced in a release.1

FDA White Oak Campus headquarters / Tada Images - stock.adobe.com

FDA White Oak Campus headquarters / Tada Images - stock.adobe.com

The expanded indication for tenofovir alafenamide was based on data from the phase 2 Trial 1092 (NCT02932150) study, which demonstrated that the drug induced progressive increases in the rates of virological suppression in children and adolescents.

About Trial 1092

Trial Name: Study of Tenofovir Alafenamide (TAF) in Children and Teen Participants With Chronic Hepatitis B Virus Infection

ClinicalTrials.gov Identifier: NCT02932150

Sponsor: Gilead Sciences

Summary: The goals of this clinical study are to compare the effectiveness, safety and tolerability of study drug, tenofovir alafenamide (TAF), versus placebo in teens and children with CHB and to learn more about the dosing levels in children.

“The expanded indication for Vemlidy for the treatment of children as young as six years old is a testament to the safety, tolerability and efficacy profile of this therapy,” Frank Duff, MD, senior vice president and virology therapeutic area head at Gilead, said in a release.1 “Effective and tolerable options for children require our best science and a dedicated focus."

Trial 1092 is an ongoing phase 2 clinical trial comparing the efficacy, safety and tolerability of tenofovir alafenamide versus placebo. The study cohort included 88 treatment-naïve and treatment-experienced patients with chronic HBV infection. Cohort 1 included patients aged between 12 to less than 18 years weighing at least 35 kg who received either 25 mg of tenofovir alafenamide or placebo once daily. Cohort 2 included patients aged 6 to less than 12 years weighing at least 25 kg.

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At 42 weeks, the study met its primary endpoint of percentage of patients with HBV DNA levels below 20 IU/mL at 24 weeks of therapy. In total, 19% of patients who received the treatment achieved the reduction in HBV DNA levels, compared to 0% in placebo. The percentage of patients in the treatment group who achieved HBV DNA below 20 IU/mL continued to increase throughout the study, with 37% reaching the outcome at 48 and 61% at week 96. Alanine transaminase (ALT) level normalization was achieved in 54% of treated patients and 57% of placebo patients who switched to treatment.

Additionally, the most common adverse events at week 96 were nasopharyngitis, headache, COVID-19, pyrexia, diarrhea, upper respiratory tract infection, cough, viral respiratory tract infection, and upper abdominal pain.

“Chronic hepatitis B can have a significant and lasting impact on the health of children,” Chaun-Hao Lin, MD, associate professor of clinical pediatrics at the Krek School of Medicine of USC, said in a release.1 “If left untreated, hepatitis B can lead to liver cirrhosis and liver cancer. As a clinician, I am well aware of the critical importance of promptly treating this disease to avoid possible complications and liver damage. The clinical trial demonstrated that tenofovir alafenamide may represent an effective treatment option for children as young as six years old affected by this chronic disease.”

Tenofovir alafenamide, which is a targeted prodrug of tenofovir, was originally approved by the FDA in 2016 to treat adults with chronic HBV infection with compensated liver disease. It was also approved by the FDA in 2022 to treat chronic HBV infection in pediatric patients aged 12 years and older with compensated liver disease.

In the release, Gilead noted that tenofovir alafenamide includes a boxed warning regarding “post-treatment severe acute exacerbation of hepatitis B.” Patients who discontinue the drug should be closely monitored with clinical and laboratory follow-up for at least several months.

“The work of our Gilead Pediatric Center of Excellence is responsible for coordinating pediatric clinical trials for treatments for cancer, HIV, hepatitis B, and COVID-19 and we will continue our research to help address unmet treatment needs for children,” Duff said.1

READ MORE: Infectious Disease Resource Center

1. FDA Expands Indication for Gilead's Vemlidy (Tenofovir Alafenamide) to Treat Chronic HBV Infection in Pediatric Patients as Young as Six. News Release. Gilead Sciences. March 28, 2024. Accessed March 28, 2024. https://www.gilead.com/news-and-press/press-room/press-releases/2024/3/fda-expands-indication-for-gileads-vemlidy-tenofovir-alafenamide-to-treat-chronic-hbv-infection-in-pediatric-patients-as-young-as-six
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