Vyndamax Nears Insurance Coverage After Four-Year Wait

The only treatment option for patients with transthyretin amyloid cardiomyopathy (ATTR-CM), Vyndamax (tafamidis), is set to receive insurance coverage nearly four years after its approval.

Vyndamax passed the first Drug Reimbursement Evaluation Committee meeting held by the Health Insurance Review and Assessment Service (HIRA) on October 10, 2024. The committee deemed the drug appropriate for reimbursement for both wild-type and hereditary ATTR-CM. Industry sources indicate that Pfizer Korea has completed price negotiations with the National Health Insurance Service (NHIS), and following a final review by the Health Insurance Policy Review Committee, the drug is expected to be covered starting in March.

Transthyretin amyloidosis (ATTR) is a rare disorder caused by the structural instability of transthyretin (TTR), a plasma protein, due to genetic mutations or aging. This leads to amyloid deposits accumulating primarily in nerves and cardiac muscles, resulting in two subtypes: transthyretin amyloid polyneuropathy (ATTR-PN) and ATTR-CM. Some patients experience a mixed form where both systems are affected.

Patients with ATTR often present with orthostatic hypotension, arrhythmia, syncope, sensory abnormalities, and gastrointestinal symptoms. Due to its similarity to primary (AL) amyloidosis, diagnosis typically requires a multidisciplinary approach involving nuclear medicine, cardiology, and pathology. Although diagnostic rates have improved, with approximately 75 new cases identified annually (2018–2021), the exact number of patients in South Korea remains unclear.

Vyndamax is the standard treatment for ATTR-CM and mixed-type (ATTR-CM + ATTR-PN) patients. However, its high annual cost exceeding $75,000 and lack of insurance coverage have significantly limited patient access. By contrast, Vyndaqel (tafamidis meglumine), which treats ATTR-PN, has been reimbursed since 2018, reducing the monthly out-of-pocket cost to approximately $320.

To alleviate financial strain, Pfizer Korea has provided Vyndamax free of charge to about 50 patients. However, many remain unable to access the drug, with some resorting to importing aspirin-based alternatives through the Korea Orphan & Essential Drug Center.

With Vyndamax set for insurance coverage, a significant gap in treatment access is expected to be addressed. However, experts stress the need for improved diagnostic rates and more flexible reimbursement policies.

A pharmaceutical company representative developing ATTR treatments stated, "In 2022, the Korean Society of Heart Failure incorporated an ATTR cardiac amyloidosis diagnostic algorithm into its heart failure treatment guidelines. Awareness is growing within the medical community, but broader public education is needed."

The representative further noted, "Some ATTR patients receive a confirmed diagnosis but do not qualify for insurance coverage due to criteria such as the extent of amyloid deposition. Ongoing discussions are necessary to refine reimbursement policies and ensure wider patient access."