Rare Liver Disease Treatment "Bylvay" to Be Covered by Health Insurance Starting Next Month

Oral Treatment Now Possible Instead of Liver Transplantation
Improved Quality of Life Expected for PFIC Patients

The treatment for PFIC (Progressive Familial Intrahepatic Cholestasis), a rare cholestatic liver disease, called Bylvay (active ingredient: odevixibat), will be included in the national health insurance coverage starting next month.

Ipsen Korea announced on the 25th that, with Bylvay now covered by health insurance, PFIC patients in Korea will, for the first time, have access to an oral medication to manage their condition without the need for liver transplantation.

PFIC is an ultra-rare disease caused by genetic defects that prevent bile from being properly excreted from the liver, leading to its accumulation and subsequent liver damage. The disease typically manifests in infancy or early childhood and is accompanied by severe pruritus (itching), growth retardation, and impaired liver function. Without treatment, liver transplantation has been the only viable option, causing significant psychological and financial distress for both patients and their families. The number of PFIC patients in Korea is estimated to be in the dozens.

Bylvay is the first drug designated under the Ministry of Health and Welfare’s “Simultaneous Authorization, Evaluation, and Negotiation Pilot Project.” This initiative, established in 2023, allows the government to streamline the approval and reimbursement process for new drugs, thereby shortening the time it takes for innovative treatments to become available. The close cooperation among the Ministry of Health and Welfare, the Ministry of Food and Drug Safety, the Health Insurance Review and Assessment Service, and the National Health Insurance Service has led to this achievement, which is cited as a reflection of the government’s strong policy commitment to ensuring that even a small number of patients receive timely treatment.

Professor Koh Hong of the Department of Pediatrics at Severance Hospital stated, “Children with PFIC suffer from relentless itching day and night, making normal life impossible. Bylvay is not just a medication, but a treatment option that restores quality of life for both the children and their families. The fact that the disease can now be managed without liver transplantation represents a paradigm shift in treatment.”

Professor Koh Jaesung of the Department of Pediatrics at Seoul National University Hospital also commented, “Bylvay has been proven to significantly reduce bile acid levels and improve pruritus in PFIC patients. With long-term use, it may even preserve liver function, which could have a positive impact on patient outcomes.”

This content was produced with the assistance of AI translation services.