Will MSD’s rare cancer drug Welireg be reimbursed this time

Company makes second attempt at reimbursement in Korea

Expected to be submitted to the Cancer Disease Deliberation Committee... Attention to whether or not the reimbursement standard will be set this time

A treatment option for Von Hippel-Lindau syndrome... reduces HIF-2α gene transcription and expression

Attention is gathering as to whether the rare anticancer drug Welireg will succeed in its second attempt at reimbursement listing in Korea.

 

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

 

This drug was submitted for reimbursement in April last year and was put up for public petition in May, gaining the consent of more than 50,000 people, but it failed to overcome the CDDC review hurdle.

 

The key to Welireg’s second reimbursement attempt will again be finances.

 

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

 

Specifically, the drug is indicated for the treatment of adult patients with VHL who require therapy for associated renal cell carcinoma (RCC), CNS hemangioblastoma, or pancreatic neuroendocrine tumors (pNET), that do not require immediate surgery.

 

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.

 

Patients enrolled in the trial had other VHL-associated tumors including CNS hemangioblastomas and pNET.

 

The major efficacy endpoint of the clinical trial was the overall response rate (ORR) in patients with VHL-associated RCC as measured by radiology assessment using RECIST v1.1 as assessed by an independent review committee (IRC).

 

Additional efficacy endpoints included duration of response (DoR) and time to response (TTR).

 

In the study, Welireg showed an ORR of 49% in patients with VHL-associated RCC.

 

All responses were partial responses.

 

The median DoR had not yet been reached, and the DoR among responders who were still responding after at least 12 months was 56%.

 

Median TTR was 8 months.

 

Also, in patients with VHL-associated CNS hemangioblastomas, Welireg showed an ORR of 63%, with a complete response rate of 4% and a partial response rate of 58%.

 

Recently, Welireg was additionally approved for a kidney cancer indication in the US.

 

Its efficacy for the indication was confirmed in LITESPARK-005, a trial conducted on patients with unresectable locally advanced or metastatic clear cell renal cell carcinoma (RCC) that had progressed following both a PD-1 or PD-L1 checkpoint inhibitor and a VEGF-TKI.

 

Results showed that Welireg improved progression-free survival (PFS) and reduced the risk of disease progression or death by 25% compared to everolimus in patients with advanced renal cell carcinoma who received a PD-1 or PD-L1 immune checkpoint inhibitor and a VEGF receptor-targeted therapy, either sequentially or in combination.