The U.S. Food and Drug Administration has approved the first specific therapy for hepatitis D, a rare and aggressive liver infection that until now had no authorized treatment. The decision marks a turning point for tens of thousands of Americans living with the virus, which can rapidly cause severe liver damage when combined with hepatitis B.
On May 22, the FDA granted accelerated approval to Hepcludex (bulevirtide) for adults with chronic hepatitis D virus (HDV) who have either no cirrhosis or compensated cirrhosis. Hepatitis D is a defective virus that can only replicate in the presence of hepatitis B virus (HBV). Dual infection accelerates liver disease, leading to cirrhosis, liver cancer and liver failure much faster than hepatitis B alone. An estimated 40,000 to 80,000 people in the United States, or 2 to 4 percent of those with chronic hepatitis B, also carry HDV.
Hepcludex is a first-in-class entry inhibitor. It binds to receptors on liver cells and blocks both HBV and HDV from entering, effectively stopping the hepatitis D virus from replicating. In the pivotal Phase III MYR301 trial, 48 percent of participants who started the drug immediately achieved a combined endpoint of undetectable or substantially reduced HDV RNA and normalized liver enzyme levels at 48 weeks, compared with only 2 percent of those who delayed treatment. Some research suggests the drug may lead to a functional cure, meaning sustained viral suppression even after stopping therapy.
The FDA had previously declined to approve Hepcludex in November 2022 due to manufacturing and delivery concerns, though it did not question the drug’s safety or efficacy. Gilead Sciences, which acquired the drug’s developer, addressed those issues and resubmitted its application. The therapy is given as a once-daily subcutaneous injection that patients can self-administer in the thigh or abdomen. It can be used alongside standard hepatitis B medications such as Viread, Vemlidy or Baraclude. Treatment continues as long as it suppresses viral replication, and the drug is generally well tolerated, though stopping it can lead to worsening liver disease.
With this approval, doctors now have a tool to alter the course of a disease that has long been neglected. “All of us at the Hepatitis B Foundation are optimistic that the FDA’s approval of Hepcludex will be a game changer,” said Chari Cohen, president of the Hepatitis B Foundation, in a statement. The accelerated approval requires ongoing studies to confirm clinical benefits, but for patients who have faced decades without options, the new therapy brings a long-awaited sense of hope.