MSD Korea is re-applying for reimbursement of the rare anticancer drug ‘Welireg,’ following the re-application for reimbursement of its Keytruda

Results of the first hurdle, whether the Cancer Disease Review Committee sets a reimbursement standard, are expected to determine the fate of the drug’s quick reimbursment.

# According to industry sources, MSD Korea’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor Welireg (belzutifan) will be once again presented to the Health Insurance Review and Assessment Service’s Cancer Disease Deliberation Committee on March 19th.

Von Hippel-Lindau disease is a hereditary disease associated with tumors in various organs and is caused by genetic abnormalities in the tumor suppressor gene VHL. It is characterized by causing various malignant and benign tumors, including central nervous system hemangioblastoma, retinal hemangioblastoma, lymphatic cysts of the inner ear, cysts of the kidneys and pancreas, pheochromocytoma and paraganglioma of the adrenal gland, and adenoma of the gonads, which occur in the brain and spinal cord.

Its incidence rate is about 1 in 36,000 people, and it is estimated that about 200,000 people worldwide and about 10,000 people in the United States are suffering from it.

In Korea, the number of adult patients with VHL disease is estimated to be in the low to mid-200s, based on data from the Health Insurance Review and Assessment Service.

Based on this, Welireg was designated an orphan drug in Korea for the treatment of Von Hippel-Lindau disease in 2023, then formally approved in May of the same year.

The company then submitted a reimbursement review application to the Health Insurance Review and Assessment Service (HIRA) in April last year and embarked on a full-fledged reimbursement journey.

However, in August of the same year, the application was submitted to the CDDC, but the committee concluded that the reimbursement criteria should not been set, resulting in the first setback.

The company then again submitted an application and the application is likely to be resubmitted at the 2025 3rd CDDC meeting scheduled for the 18th of this month.

Meanwhile, the voices of patient groups are growing along with the national petition urging for Welireg’s reimbursement.

In fact, the petitioner of the petition for national consent urged the government to reimburse the drug, imploring, “The price of one month (90 tablets) of Welireg, the only treatment for VHL, is KRW 22.61 million, aggravating the economic burden of the patients.”

As a HIF-2α inhibitor, Welireg reduces transcription and expression of HIF-2α target genes associated with cellular proliferation, angiogenesis, and tumor growth.

The drug’s efficacy was demonstrated in the open-label Study 004 trial, which investigated 61 patients with VHL-associated RCC who were diagnosed with at least one measurable solid tumor localized to the kidney.