Gilead Sciences, Inc. (Nasdaq:
GILD) announced the U.S. Food and Drug Administration (FDA) granting accelerated approval for Hepcludex (bulevirtide-gmod)
8.5 mg for the treatment of adults living with chronic hepatitis delta virus
(HDV) infection, making it the first and only approved treatment for HDV in the
United States.
- Chronic HDV is considered the most severe form of
viral hepatitis due to rapid disease progression towards liver failure and
liver-related death
- Approval introduces the first FDA-approved therapy
for this disease with limited treatment options and high unmet need
The FDA granted accelerated
approval to Hepcludex based on reductions in HDV RNA and normalization of
alanine aminotransferase (ALT), supported primarily by data from the pivotal,
controlled Phase 3 MYR301 study. At Week 48, the study demonstrated a statistically
significant improvement versus the control (delayed treatment) group in a
combined virologic and biochemical response. Improvement in disease-related
clinical outcomes has not been established. Continued approval for the approved
indication may be contingent on verification and description of clinical
benefit in a confirmatory trial.
Chronic HDV is considered the
most severe form of viral hepatitis and is associated with a markedly higher
risk of rapid disease progression, liver failure, and mortality compared with
HBV alone. In the United States, studies in general populations have estimated
that HDV affects between 2% and 4% of individuals who have chronic hepatitis B
virus (HBV), representing ~40,000-80,000 people.
“Hepatitis delta virus is
associated with rapid progression of liver disease and a high risk of serious
or even life-threatening liver-related complications,” said Dr. Ira Jacobson,
MD, Department of Medicine at NYU Grossman School of Medicine. “For patients,
an HDV diagnosis means managing two distinct viral liver diseases—hepatitis B
and hepatitis D—each contributing to disease progression, monitoring demands,
and treatment complexities. The approval of Hepcludex for chronic HDV
represents a critical advancement, introducing a long-awaited option that
begins to address a significant unmet medical need and has the potential to
meaningfully alter the course of this devastating disease for people living
with HDV in the United States.”
MYR301 (NCT03852719) evaluated
the efficacy and safety of Hepcludex in adults with chronic HDV, with treatment
administered for up to 144 weeks followed by 96 weeks of off-treatment
follow-up. Hepcludex met its primary endpoint at Week 48, with continued
treatment, demonstrated sustained efficacy and was generally well tolerated
through up to 144 weeks of on-treatment exposure.
“The approval of Hepcludex
represents a historic milestone for people living with HDV in the United
States, marking the first FDA-approved treatment for HDV,” said Dietmar Berger,
MD, PhD, Chief Medical Officer at Gilead Sciences. “This reflects years of
close engagement with the FDA and the application of rigorous science to
address a serious disease with long-standing unmet need. With Hepcludex, we now
have the opportunity to deliver a meaningful clinical advancement that has the
potential to change the trajectory of HDV for patients in the U.S.”
U.S. Access and Hepcludex
Approval Across Markets
The Gilead Support Path® Program
offers information and resources to help patients diagnosed with chronic HBV,
HDV and hepatitis C virus (HCV) and primary biliary cholangitis (PBC), as well
as healthcare professionals, understand coverage and financial options for
prescribed Gilead treatments.
Bulevirtide 2 mg is also approved
for use in the European Economic Area (EEA) and other countries globally to
treat people living with chronic HDV.
Please see below for U.S.
Indication and Important Safety Information for Hepcludex.
U.S. Indication for Hepcludex
Hepcludex (bulevirtide-gmod) 8.5
mg for injection is indicated for the treatment of chronic hepatitis delta
virus infection in adults without cirrhosis or with compensated cirrhosis.
This indication is approved under
accelerated approval based on a decrease in HDV RNA and alanine
aminotransferase (ALT) normalization. An improvement in disease-related
clinical outcomes has not been established. Continued approval for this
indication may be contingent upon verification and description of clinical
benefit in a confirmatory trial(s).