Bylvay (odeviksibab), a treatment for Progressive Familial Intrahepatic Cholestasis (PFIC), will be included in the National Health Insurance reimbursement program starting next month.

As a high-cost medication for a severe and rare disease, Bylvay's reimbursement and monitoring have been categorized under government management.

According to industry sources on September 22, the Ministry of Health and Welfare recently issued an administrative pre-announcement for a revision to the 'Detailed Standards for the Application and Methods of Health Reimbursement Benefits,' which includes reimbursement for Ipsen Korea's Bylvay. If there are no major objections, this drug can be reimbursed from October.

PFIC is a rare genetic disorder where the accumulation of bile acids in the liver causes damage and can lead to liver failure. It is a serious condition that may require a liver transplant if not diagnosed and managed effectively early on.

A key symptom of the disease, pruritus, significantly impacts sleep and daily activities, leading to skin damage, sleep loss, irritability, and reduced attention.

Ipsen Korea has estimated that the treatment applies to approximately 50 patients, highlighting the rarity of the disease.

Due to the severity of PFIC and the innovative nature of the drug with few alternatives, Bylvay was selected as the first drug in the Concurrent Approval-Evaluation-Negotiation Pilot Program initiated by the Ministry of Health and Welfare in 2023 to accelerate the introduction of innovative new drugs.

However, the reimbursement process was not easy. Bylvay went through a 're-evaluation' at the Health Insurance Review & Assessment Service's (HIRA) Drug Reimbursement Evaluation Committee (DREC). Due to the drug's status as a treatment for a rare disease and Ipsen Korea's firm resolve, Bylvay has been included in the reimbursement list in October.

The Ministry of Health and Welfare selected Bylvay as a target for managing reimbursement of high-cost drugs. High-cost drugs are defined as 'medications that require price management and long-term efficacy verification due to their high cost and uncertain efficacy, or medications that require usage management due to their significant financial impact.' With the inclusion of Bylvay, the total number of managed treatments has increased to seven.

Accordingly, the Ministry of Health and Welfare's policy is to approve an additional six months of administration if the treatment response criteria are met at the six-month evaluation after the first dose of Bylvay. After that, the patient will be re-evaluated every six months, and continued administration will be approved if the treatment response is maintained.

The ministry also plans to approve reimbursement for patients who have been receiving Bylvay on a non-reimbursed basis, including through the pharmaceutical company's free supply program, after an evaluation. Specifically, if the reimbursement application is made within six months of the first dose, reimbursement will be approved if the patient's eligibility is confirmed in their medical records.

If the reimbursement is applied for more than six months after the first dose, reimbursement will be approved if the patient's eligibility is confirmed in their medical records and their serum bile acid (sBA) concentration has decreased by 30% or more from baseline in the most recent response evaluation. If the sBA concentration has not reduced by 30% or more, reimbursement may still be approved if there is an improvement in pruritus (a CGIS score of 1 or less, or a decrease of 1 point or more from baseline).

However, the most recent response evaluation must have been conducted within six months of the date the reimbursement application was submitted.

The Ministry of Health and Welfare stated, "If a patient is eligible for treatment, administration for six months will be approved based on the most recent response evaluation. After that, the patient will be evaluated every six months, and continued administration will be approved if the treatment response meets the evaluation criteria."

Meanwhile, Ipsen Korea is also expanding its influence in the clinical setting in Korea. In addition to securing reimbursement for Bylvay, the company recently obtained domestic approval for its primary biliary cholangitis (PBC) treatment, 'Iqirvo (elafibranor).'

Ipsen Korea CEO Yang Mi-sun explained, "Bylvay is a treatment for PFIC, an ultra-rare disease that is very rarely caused by a failure of bile secretion," and added, "In the past, there were no suitable treatments, and in most cases, a liver transplant was necessary."

Yang also said, "The fact that a patient must give a liver transplant to their child is something that I cannot take lightly. In this situation, Ipsen providing the first effective treatment option is very significant," and added, "PBC is also a disease that has been known for a long time but has lacked a suitable treatment. Therefore, we plan to redefine the approach to treating this disease together with experts in academia."