Gilead’s Vemlidy gets FDA approval for paediatric patients with chronic hepatitis B infection

Gilead Sciences (Gilead) supplemental new drug application (sNDA) for Vemlidy (tenofovir alafenamide) 25mg tablets to treat chronic hepatitis B virus (HBV) infection in paediatric patients 12 years and older with compensated liver disease has been approved by the US Food and Drug Administration (FDA).

The application was supported by positive 24-week data from a phase 2 trial comparing treatment with Vemlidy 25mg to placebo in 70 treatment-naïve and treatment-experienced patients aged 12 to less than 18 years.

The study met its primary endpoint, with 21% of Vemlidy-treated patients achieving HBV DNA levels below 20 IU/mL at 24 weeks of therapy, compared to 0% of subjects treated with placebo.

Higher rates of serum alanine aminotransferase (ALT) normalisation were also observed based on criteria set by the American Association for the Study of Liver Diseases (AASLD) at 24 weeks, with 44% of Vemlidy-treated patients and 0% of placebo-treated patients achieving ALT normalisation.

Importantly, the company outlined that the mean percent changes in bone mineral density from baseline to week 24 were numerically similar for Vemlidy-treated patients and placebo-treated patients.

HBV is a serious disease that attacks the liver and can cause chronic infection, cirrhosis of the liver, liver cancer and death in up to a third of patients. Early symptoms may include loss of appetite, fever, generalised aches and pains, fatigue, itching, urticaria (hives) and joint pain, but the disease is often asymptomatic which may lead to undiagnosed individuals.

“Chronic hepatitis B can have a significant long-term health impact on children, including the development of liver cancer later in life if the disease is left untreated, which is compounded by treatment challenges in this population,” said Kathleen Schwarz, paediatric gastroenterologist, Rady Children’s Hospital-San Diego, and an investigator in the Vemlidy clinical trial.

“As a clinician, I recognise the critical importance of treating this disease as quickly as possible to help avoid complications and potential damage to the liver. In the clinical trial, we saw that [Vemlidy] may represent an effective treatment option for people as young as 12 years of age living with this chronic disease,” she added.

Vemlidy was approved by the FDA in 2016 as a once-daily treatment for adults with chronic HBV infection with compensated liver disease. It is also recommended as a preferred or first-line treatment for adults with chronic HBV with compensated liver disease in guidelines from the AASLD and European Association for the Study of the Liver.