FDA Hands Expanded Indication to Gilead’s Vemlidy for Chronic HBV Infection in Pediatric Patients

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The FDA has approved an expanded indication for Gilead’s Vemlidy (tenofovir alafenamide), to treat chronic hepatitis B virus (HBV) infection in pediatric patients aged six years and older who weigh at least 25 kg and have compensated liver disease. In 2016, the medication was initially approved for adults with chronic HBV and compensated liver disease, with an extended use approved in 2022 for children aged 12 years and above.

Approval for the expanded indication was based on positive results stemming from Gilead’s Phase II clinical trial (Trial 1092) after 96 weeks, showing safety and efficacy in younger children. According to the study data, participants experienced progressive increases in the rates of virological suppression compared to those administered a placebo.¹

“Chronic hepatitis B can have a significant and lasting impact on the health of children. If left untreated, hepatitis B can lead to liver cirrhosis and liver cancer,” said Chaun-Hao Lin, MD, associate professor, clinical pediatrics Krek School of Medicine of USC, in a press release. “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.”

Trial 1092, which began in 2016, is an ongoing interventional study that enrolled an estimated 150 children and adolescents aged 2 to 17 years with four different weight cohorts. All participants have struggled with HBV for a minimum of six months, have a normal ECG, and an estimated creatinine clearance (CLCr) ≥ 80 mL/min/1.73m^2. Exclusion criteria includes, but isn’t limited to, pregnant or breastfeeding females, abnormal hematological and biochemical parameters, patients who have received organ or bone marrow transplants, renal, cardiovascular, pulmonary, or neurological disease found to be substantial to the investigator.

The primary completion of the study is expected in August of next year with an overall expected endpoint of October 2029.²

“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,” said Frank Duff, MD, SVP, virology therapeutic area head, Gilead Sciences, in the press release. “Effective and tolerable options for children require our best science and a dedicated focus. 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.”

Vemlidy comes with a boxed warning in its product label regarding post-treatment severe acute exacerbation of HBV.¹

References

1. FDA Expands Indication for Gilead's Vemlidy (Tenofovir Alafenamide) to Treat Chronic HBV Infection in Pediatric Patients as Young as Six. Gilead. March 28, 2024. Accessed March 29, 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

2. Study of Tenofovir Alafenamide (TAF) in Children and Teen Participants With Chronic Hepatitis B Virus Infection. Clinicaltrials.gov. Accessed March 29, 2024. https://clinicaltrials.gov/study/NCT02932150