By VETTAPHARMA reporter – Derek Roche: GSK plc announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has accepted for regulatory review a new drug application (NDA) for bepirovirsen, an investigational antisense oligonucleotide (ASO), as a potential first-in-class treatment for chronic hepatitis B (CHB). This submission is supported by positive results from the Phase III B-Well 1 and B-Well 2 trials, where bepirovirsen demonstrated statistically significant and clinically meaningful functional cure rates when combined with standard of care versus standard of care alone.
The press release states that more than 250 million people worldwide are affected by chronic hepatitis B and approximately nearly 1 million people in Japan live with the disease. Chronic hepatitis B often requires lifelong antiviral therapy, with existing functional cure rates remaining low, typically around 1 %. Functional cure is defined as hepatitis B virus DNA and hepatitis B surface antigen (HBsAg) levels being undetectable in the blood for at least 24 weeks after stopping all treatment and controlled by the immune system without medication.
Bepirovirsen works by inhibiting viral genome replication, suppressing HBsAg levels in the blood, and stimulating the immune system to increase the likelihood of a durable and sustained response. The drug has received SENKU designation in Japan (granted August 2024) to facilitate expedited regulatory review, reflecting its innovation and potential to address a disease with high unmet medical need.
The regulatory submission is considered a major milestone toward expanding treatment options for adults with chronic hepatitis B. Bepirovirsen is currently not approved in any market.
