What is the use of Vyndamax (Tafamidis)?

From the Guidelines

Vyndamax (tafamidis) is recommended as a first-line treatment for patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM) with NYHA class I to III symptoms, due to its ability to improve cardiovascular outcomes and reduce mortality, as demonstrated in the ATTR-ACT clinical trial 1.

Key Points

• The standard dose of Vyndamax is 61 mg taken orally once daily, with or without food.
• Vyndamax works by stabilizing the transthyretin protein, preventing it from breaking apart and forming harmful amyloid deposits in the heart tissue.
• The medication has been shown to slow the progression of heart failure symptoms and may improve survival in patients with ATTR-CM, with a lower all-cause mortality rate (29.5% versus 42.9%) and lower cardiovascular-related hospitalization rate (0.48 versus 0.70 per year) after 30 months, as demonstrated in the ATTR-ACT clinical trial 1.
• Common side effects include respiratory tract infections, dizziness, and diarrhea.
• Patients should continue taking their other heart failure medications as prescribed alongside Vyndamax.
• It's essential to note that Vyndamax is specifically for the cardiomyopathy form of transthyretin amyloidosis and not for other types of amyloidosis.
• Regular follow-up with a cardiologist is necessary to monitor heart function and assess treatment response.
• The medication is expensive, so insurance coverage verification is recommended before starting treatment.

Important Considerations

• Tafamidis is available in two formulations: tafamidis meglumine (20-mg capsules) and tafamidis (61-mg capsules), with the FDA-approved dose being 80 mg (4 capsules) once daily for the meglumine formulation and 61 mg once daily for the new formulation 1.
• The cost-effectiveness of tafamidis has been evaluated in model-based analyses, which estimated that the medication increases average survival by 1.97 years and QALY by 1.29, despite having an incremental cost-effectiveness ratio >$180,000 per QALY gained 1.
• The medication is not FDA-approved for the treatment of amyloid neuropathy, and its use in this context is not supported by current evidence 1.

From the FDA Drug Label

VYNDAQEL and VYNDAMAX are indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. Vyndamax is a medication used to treat adults with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM). The main goal of the treatment is to reduce cardiovascular mortality and cardiovascular-related hospitalization 2.

• Key points:
  • Indication: Treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults.
  • Goal of treatment: Reduce cardiovascular mortality and cardiovascular-related hospitalization.

From the Research

Overview of Vyndamax

• Vyndamax, also known as tafamidis, is a medication used to treat transthyretin amyloid cardiomyopathy (ATTR-CM) 3, 4.
• It works by stabilizing both wild-type and mutant transthyretin (TTR), inhibiting the formation of TTR amyloid fibrils 3.

Efficacy of Vyndamax

• In the pivotal phase III ATTR-ACT trial, tafamidis significantly reduced all-cause mortality and frequency of cardiovascular-related hospitalizations relative to placebo in patients with ATTR-CM 3.
• Tafamidis recipients also experienced significantly less deterioration in 6-minute walk test distance and quality of life than placebo recipients over the 30-month treatment period 3.
• Treatment benefits were largely consistent between patients with wild-type TTR and patients with a variant TTR genotype 3.

Safety and Tolerability of Vyndamax

• Tafamidis was generally well tolerated in patients with ATTR-CM and had a safety profile similar to that of placebo 3.
• This makes tafamidis suitable for long-term use in patients with ATTR-CM 3.

Current and Future Therapies for ATTR-CM

• Tafamidis is currently the only approved drug for the treatment of ATTR-CM, but other therapies such as patisiran and inotersen are being investigated 4, 5, 6.
• Novel therapies using RNA interference and monoclonal antibodies are also being developed and may offer clinical promise 5, 6.
• Despite the availability of these therapies, the high cost and limited accessibility may pose challenges for long-term treatment 4.