Korea to cover Bylvay, first oral therapy for rare liver disease PFIC

Ipsen Korea announced on Thursday that Bylvay (odevixibat), a treatment for progressive familial intrahepatic cholestasis (PFIC), will be covered by national health insurance starting in October.

This opens the door for PFIC patients in Korea to manage their disease with an oral medication, avoiding the need for liver transplantation.

PFIC is a rare disease in which bile fails to be properly excreted from the liver due to a genetic defect, leading to its accumulation and liver damage. It typically manifests in infancy or early childhood, accompanied by severe pruritus (itching), growth delay, and impaired liver function. Ultimately, liver transplantation has been the only alternative. The number of patients with PFIC in Korea is estimated to be only a few dozen.

PFIC experts unanimously welcomed the inclusion of Bylvay in the national health insurance coverage.

“Children with PFIC suffer from relentless itching day and night, making normal life difficult. Bylvay is not just a medication; it is a treatment option that restores the lives of children and their families, making it highly significant,” said Professor Koh Hong of the Department of Pediatrics at Severance Hospital. “It represents a paradigm shift in treatment, as it allows disease management without the need for liver transplantation.”

Professor Ko Jae-sung of the Department of Pediatrics at Seoul National University Hospital also said, “Bylvay, a treatment for PFIC symptoms, has been proven to significantly lower bile acid levels and improve pruritus in PFIC patients. Particularly, long-term use holds the potential to preserve liver function, which can positively impact patient prognosis.”

Kim Jae-hak, president of the Korean Organization for Rare Diseases, remarked, “Until now, PFIC patients and their families faced a bleak situation with no options beyond liver transplantation. This health insurance coverage, enabling patient access, carries a social message beyond simply introducing a treatment: it affirms that even patients with ultra-rare diseases have the right to receive treatment.”

Bylvay is the first drug to be approved under the Ministry of Health and Welfare's “approval-evaluation-parallel pilot program,” which was introduced in 2023 to shorten the timeline by concurrently processing new drug approval and reimbursement listing procedures.

“We deeply appreciate the government's proactive efforts in introducing this pilot program to enhance access to treatments for rare diseases,” Ipsen Korea General Manager Yang Mi-sun said. “Ipsen will continue to do its utmost to provide new hope to patients in the field of rare diseases where treatment gaps exist.”