GC Biopharma's Livmarli (maralixibat chloride) and Recordati Korea's Isturisa (osilodrostat phosphate) have crossed the threshold for health insurance coverage in Korea. 

On Thursday, the Health Insurance Review and Assessment Service (HIRA) held the eighth Pharmaceutical Reimbursement Evaluation Committee (PREC) meeting for 2025 to review the appropriateness of reimbursement for two rare disease treatments that had applied for medical benefits, as well as eight components of drugs subject to reevaluation.

As a result, GC Biopharma Livmarli, which used to treat cholestatic pruritus in patients with Alagille syndrome, and Recordati Korea's Isturisa, approved for adult Cushing's disease where pituitary surgery is not possible or insufficient effects are achieved post-surgery, were recognized as appropriate for reimbursement.

Alagille syndrome is a rare disease caused by mutations in the JAG1 gene and other genes, such as the NOTCH2 gene, leading to bile accumulation in the liver due to impaired bile flow from the liver to the bile ducts. This results in liver damage and bile acid buildup in the bloodstream, causing itching. It is estimated that approximately 50 patients with Alagille syndrome reside in Korea.

Livmarli is the first treatment for cholestatic itching in Alagille syndrome patients, developed by U.S.-based Mirum Pharmaceuticals, and received approval from the U.S. Food and Drug Administration in 2021. GC Biopharma holds the sales rights for the drug in Korea.

Cushing's disease is a rare condition characterized by chronic elevation of cortisol levels in the blood due to conditions, including pituitary adenoma, leading to severe endocrine metabolic disorders, cardiovascular complications, and increased risk of death.

Recordati Korea's Cushing's disease treatment, Isturisa, which received approval from the Ministry of Food and Drug Safety (MFDS) in November last year, inhibits 11-beta-hydroxylase (CYP11B1), an enzyme responsible for cortisol synthesis in the adrenal glands, reducing cortisol production and demonstrating therapeutic effects. It is effective for patients with Cushing's disease who cannot undergo pituitary surgery or for whom surgery is ineffective. In October 2023, it was designated as the 18th product under the Global Innovative Product Fast-Track review support system (GIFT).

After passing the PREC, Livmarli and Isturisa will undergo price negotiations with the National Health Insurance Service (NHIS) and review by the Health Insurance Policy Review Committee. If the price negotiations with the NHIS proceed smoothly, reimbursement may be possible within this year.

Meanwhile, the results of the re-evaluation review of the eight drug components, as reviewed by the PREC, showed that only the following drug components were deemed appropriate for reimbursement: olopatadine hydrochloride, the three herbal components of uiryeongseon, gualugeun, and hagocho, and bepotastine-based drugs. For L-aspartic acid-L-ornithine, reimbursement appropriateness was recognized only for oral formulations and 0.5 g/mL injections as an adjunctive therapy for severe liver disease detoxification. For the 0.1 g/mL injection currently under clinical review by the MFDS, the appropriateness of reimbursement was not recognized.

In addition, spherical activated carbon, artemisia extract, sulglycoside, and kenodeoxycholic acid-ursodeoxycholic acid trihydrate magnesium salt for the treatment of chronic kidney disease (progressive) uremia, acute gastritis, chronic gastritis, gastroduodenitis, and gallstone- or bile-related dyspepsia have also been evaluated as having no clinical efficacy, unclear efficacy, or lacking cost-effectiveness.

For drugs that fail to pass the drug price reimbursement reevaluation, an appeal can be submitted within 30 days for reconsideration; however, overturning the reevaluation results is unlikely.

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The Health Insurance Review and Assessment Service in Wonju, Gangwon Province