A Biologics License Application has been submitted to the Food and Drug Administration (FDA) for bulevirtide, an investigational first-in-class antiviral, for the treatment of chronic hepatitis delta virus (HDV) infection in adults with compensated liver disease.
The application submission is supported by data from the ongoing phase 3 MYR301 study (ClinicalTrials.gov Identifier: NCT03852719), along with completed and ongoing phase 2 studies. MYR301 is a multicenter, open-label, randomized study evaluating the long-term efficacy and safety of bulevirtide in 150 adults with chronic HDV infection. Patients were randomly assigned to receive either bulevirtide 2mg (n=49) or 10mg (n=50) subcutaneously once daily, or no antiviral treatment (delayed treatment, n=51) for 24 weeks.
The primary endpoint was combined response at week 48, defined as the percentage of patients with undetectable HDV RNA (<LoD) or at least 2log10 IU/mL decline from baseline and ALT normalization.
Interim results showed that 36.7% and 28% of patients treated with bulevirtide 2mg and 10mg, respectively, achieved combined virological and biochemical response after 24 weeks compared with 0% of those who did not receive antiviral treatment. The bulevirtide 2mg treatment arm had a statistically significant combined response compared with the delayed treatment arm (P <.001). Moreover, greater than 50% of patients in the bulevirtide 2mg arm had rapid ALT reduction and normalization vs 5.9% of those in the delayed treatment group.
The safety profile of bulevirtide was consistent with that observed in previously completed clinical studies. The most common adverse events observed with bulevirtide were elevated levels of bile salts in the blood, injection site reactions, and worsening of liver disease after discontinuing bulevirtide.
Bulevirtide was previously granted Breakthrough Therapy and Orphan Drug designations. If approved, it would be the first treatment option for patients with chronic HDV infection with compensated liver disease.
Gilead submits Biologics License Application to US Food and Drug Administration for bulevirtide, an investigational treatment for people living with chronic hepatitis delta. News release. Gilead Sciences, Inc. Accessed November 19, 2021. https://www.businesswire.com/news/home/20211119005269/en/Gilead-Submits-Biologics-License-Application-to-U.S.-Food-and-Drug-Administration-for-Bulevirtide-an-Investigational-Treatment-for-People-Living-With-Chronic-Hepatitis-Delta.