Amyloid cardiomyopathy drug Vyndamax to get health insurance from March
Vyndamax (tafamidis), a transthyretin amyloid cardiomyopathy (ATTR-CM) drug that has experienced difficulties getting on the health insurance list due to its high cost will receive insurance benefits from March 1, significantly easing the burden of treatment costs for Korean patients.
The Ministry of Health and Welfare held the Health Insurance Policy Review Committee on Thursday and approved the proposal to reimburse Vyndamax.
Vyndamax is a treatment proven to increase survival rates and reduce cardiovascular hospitalizations in ATTR-CM patients. It was approved by the Ministry of Food and Drug Safety in August 2020.
ATTR-CM is a disease characterized by progressive and irreversible cardiac involvement that leads to impaired cardiac function, resulting in fatal heart failure, kidney disease, liver disease, and a variety of other serious symptoms that eventually lead to death.
Life expectancy varies depending on the type of gene involved and the stage of the disease, but if left untreated, the disease is fatal. The median survival is only about 2.5 to 3.5 years after diagnosis, with some subtypes reporting a median survival of up to six years.
As a result of the decision, the insurance-applied cost of Vyndamax will be capped at 100,000 won ($68.6) per capsule. However, a risk-sharing system is applied to reduce the financial burden on health insurance, in which the pharmaceutical company reimburses a percentage of the initial treatment costs and claims.
The annual treatment cost per person is expected to be about 36.5 million won (100,000 won × 365 days), but the actual cost to the patient will be about 3.65 million won when applying the special exception for rare diseases (with a 10 percent co-payment rate).
Vyndamax won approval in 2020, but the reimbursement decision has been delayed due to low cost-effectiveness in the economic evaluation. After two failed applications in 2022 and 2023, the reimbursement process accelerated in 2024 when the drugmaker and the government agreed on a financial sharing plan.
With this decision, about 368 domestic ATTR-CM patients using Vyndamax are expected to receive health insurance benefits, with an estimated annual bill of about 13.4 billion won.
The decision to cover Vyndamax was based on its proven clinical utility. In a phase 3 clinical study of 441 patients with ATTR-CM (ATTR-ACT), Vyndamax demonstrated a 30 percent reduction in the risk of all-cause mortality compared to the control group (29.5 percent in the Vyndamax arm vs. 42.9 percent in the placebo arm) and a reduction in cardiovascular hospitalizations.
In a long-term extension study, patients who continued to receive Vyndamax showed a five-year survival rate of 53.2 percent, significantly higher than the placebo-Vyndamax switched arm (32.4 percent).
Related academic societies, including the Korean Society of Cardiology, have presented their opinions, saying, “Vyndamax is recommended for treating ATTR-CM in multiple guidelines and should be reimbursed as the only agent that has demonstrated therapeutic benefit in this indication, including a reduction in mortality in a phase 3 study and long-term effectiveness in an extension study.”
Vyndamax's detailed reimbursement criteria are “genetically diagnosed with ATTR-CM by genetic testing” or “normotyped and NYHA class I to III for the last six months continuously, diagnosed with heart failure and at least one hospitalization for heart failure within the last six months, NT-proBNP level ≥ 600 pg/mL, and end-diastolic left ventricular wall thickness ≥ 12 mm” in adult patients 18 years of age or older with confirmed ATTR-CM.