Tag: Tafamidis

In May 2019, the FDA approved Tafamidis, marking the first time a medication was specifically approved to treat transthyretin amyloid cardiomyopathy (ATTR-CM). This article explores the specific clinical scenarios for which this groundbreaking drug is prescribed, detailing the disease it targets and its significant impact on patient outcomes.

Over the last decade, there has been a significant expansion in the number of FDA-approved drugs for amyloidosis, with distinct treatments now available for different types of the disease. The answer to **What drugs are FDA approved for amyloidosis?** depends on whether a patient has transthyretin (ATTR) or light chain (AL) amyloidosis, as the treatments for each type differ substantially.

The wholesale acquisition cost (WAC) for a 30-day supply of Vyndamax was over $22,000 as of January 2025. While this list price for the vital medication is exceptionally high, the actual out-of-pocket cost for patients depends heavily on their insurance, financial situation, and access to patient support programs.

In transthyretin-mediated amyloidosis (ATTR), a progressive and often fatal disease, the transthyretin (TTR) protein misfolds and forms harmful amyloid deposits. The therapeutic effectiveness of Vyndaqel hinges on its unique ability to disrupt this process, making understanding what is the mechanism of action of Vyndaqel crucial for both patients and clinicians.