Bylvay may be prescribed at Big 5 Hospitals in Korea

Listed for reimbursement in October last year… prescribing environment continues to expand

Efficacy demonstrated in Phase III ASSERT trial… an alternative to liver transplantation

Bylvay, the first drug approved under Korea’s parallel approval–assessment–negotiation linkage pathway, has successfully secured prescribing access at major tertiary hospitals.

According to industry sources, Ipsen Korea’s Bylvay (odevixibat), a treatment for pruritus in patients aged three months and older with progressive familial intrahepatic cholestasis (PFIC), has been approved by the Drug Committees (DCs) of Korea’s “Big 5” hospitals, including Samsung Medical Center, Seoul National University Hospital, Asan Medical Center, and Severance Hospital.

Since its inclusion in the national health insurance reimbursement list in October last year, Bylvay has rapidly expanded its prescribing environment.

Bylvay is the world’s first oral therapy indicated for the treatment of PFIC-related symptoms. It offers a non-invasive and sustainable treatment option for patients who previously had few alternatives other than high-risk procedures such as liver transplantation.

Following its initial approval in the US and Europe in 2021, it has received authorization in major countries. In Korea, it was selected as the first drug under the Ministry of Health and Welfare's ‘Parallel Approval-Evaluation-Negotiation Pilot Program’ in 2023.

PFIC is a rare, inherited disease that typically manifests in childhood and causes severe pruritus, growth impairment, and liver failure. Patients and their families endure suffering across all aspects of daily life, including sleep deprivation, interrupted education, and social isolation. Bylvay is a treatment that alleviates this diminished quality of life and opens the possibility for patients to return to their daily routines.

Meanwhile, Bylvay demonstrated its efficacy through the Phase III ASSERT study conducted in pediatric and adolescent patients aged 17 years and younger.

Study results showed that Bylvay met its primary endpoint by significantly reducing pruritus compared with placebo. It also statistically significantly improved the key secondary endpoint, mean serum bile acid levels at weeks 20 and 24 of treatment, compared to placebo. These therapeutic effects were sustained through 24 weeks of treatment.

Professor Seak-hee Oh of the Department of Pediatrics at Asan Medical Center said, “Liver transplantation was a treatment option that had to be chosen as a last resort, despite an average failure rate exceeding 10% and the risk of serious complications. In a situation where there were virtually no alternatives other than transplantation, Bylvay could establish itself as an important alternative that can protect patients without the need for liver transplantation.”