The U.S. Food and Drug Administration (FDA) has approved Hepcludex (bulevirtide-gmod) injection as the first treatment for chronic hepatitis delta virus (HDV) infection in adults without cirrhosis or with compensated cirrhosis. This approval marks a significant milestone for patients affected by this serious liver disease, which can rapidly progress to liver fibrosis, cancer, failure, and death.
HDV infection occurs only in individuals already infected with hepatitis B virus (HBV). Transmission risks include unprotected sex, intravenous drug use, and occupational exposure to blood. Vaccination against HBV also protects against HDV infection.
Clinical Trial Efficacy
The FDA’s decision was based on results from a multicenter, randomized, open-label phase 3 trial known as MYR301. The study compared immediate treatment with Hepcludex 8.5 mg once daily against delayed treatment after an observational period. The primary endpoint measured at 48 weeks combined virologic response, defined as undetectable HDV RNA or a significant decline from baseline, and normalization of liver enzyme ALT levels.
At 48 weeks, 48% of participants receiving immediate Hepcludex treatment achieved the combined response, compared with only 2% in the delayed treatment group. Undetectable HDV RNA was observed in 20% of treated patients versus none in those delayed. Continued treatment showed increased rates of undetectable virus: 36% at 96 weeks and 50% at 144 weeks.
Safety and Regulatory Status
Side effects reported in the trial with Hepcludex included injection site reactions, headache, abdominal pain, fatigue, itching, and hypersensitivity reactions, including rare cases of anaphylaxis. The drug’s labeling carries a boxed warning that stopping treatment may lead to severe acute flare-ups of HDV and HBV infections.
The FDA granted Hepcludex Breakthrough Therapy and Orphan Drug designations. It also received priority review and was approved through the Accelerated Approval program. These pathways aim to expedite access to treatments for serious or rare diseases with unmet medical needs.
Why it matters
Before this approval, no FDA-approved therapies existed for chronic HDV infection, leaving patients with limited options to manage a disease associated with rapid liver deterioration. Hepcludex offers a new therapeutic choice to help control viral replication and potentially reduce progression of liver damage. This approval highlights ongoing efforts to provide innovative treatments for challenging infectious diseases.
Sources
This article is based on reporting and publicly available information from the following source:
Read more Health & Public Health stories on Goka World News.