Today, the U.S. Food and Drug Administration approved Hepcludex (bulevirtide-gmod) injection to treat chronic hepatitis delta virus (HDV) infection in adults without cirrhosis (advanced liver scarring) or with compensated cirrhosis. Bulevirtide is the first FDA-approved treatment for chronic HDV infection, a serious and life-threatening condition that can cause rapid development of liver fibrosis (scarring), liver cancer, liver failure, and even death.
"Today's approval fills a critical gap in care for patients with chronic HDV infection, who until now have had no FDA-approved therapies available," said Wendy Carter, D.O., Acting Director of the Office of Infectious Diseases in FDA’s Center for Drug Evaluation and Research. "For individuals living with this chronic viral infection, this new treatment option offers hope in managing a disease that can rapidly progress to serious liver complications."
HDV infection only occurs in individuals who have hepatitis B virus (HBV) infection. Some risk factors for contracting HDV include unprotected sex, injecting drugs, and occupational exposure to blood. HBV vaccination protects against HBV and HDV as well.
The efficacy of Hepcludex was demonstrated in a multi-center, randomized, open-label, parallel-arm phase 3 trial. In Trial MYR301, participants were randomly assigned to immediate treatment with Hepcludex 8.5 mg once daily for 144 weeks or to delayed treatment with an observational period of 48 weeks followed by Hepcludex 8.5 mg once daily for 96 weeks.
The primary efficacy endpoint was combined response, defined as undetectable HDV RNA (defined as less than the lower limit of quantification [LLOQ] [50 IU/mL] with target not detected) or ≥ 2 log10 IU/mL decline from baseline and aminotransferase (ALT) normalization, at week 48. At this time, the combined response was 48% in the Hepcludex group compared with 2% in the delayed treatment group.
At week 48, the rate of undetectable HDV RNA was 20% in the Hepcludex group compared with 0% in the delayed treatment group. At weeks 96 and 144, the rate of undetectable HDV RNA increased to 36% and 50%, respectively, in the Hepcludex group.
Possible side effects associated with Hepcludex include hypersensitivity reactions, including anaphylaxis (severe allergic reactions), injection site reactions, headache, abdominal pain, fatigue, and pruritus (itching). The labeling includes a boxed warning that discontinuation of Hepcludex may result in severe acute exacerbations of HDV and HBV infection.
The FDA granted Hepcludex Breakthrough Therapy Designation and Orphan-Drug Designation. Hepcludex received priority review and was approved under the Accelerated Approval pathway. This approval underscores the FDA's ongoing efforts to expedite access to innovative therapies for patients with diseases that have limited or no treatment options.
Today's approval addresses a critical need for people with chronic HDV infection and builds on the FDA's commitment to advancing the health of all Americans.
The FDA granted the approval to Gilead Sciences, Inc.
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