Vyndamax for Transthyretin Amyloid Cardiomyopathy

Published: Aug 21, 2024
Category
Reference Materials
Key Information
Source
WebMD
Year
2024
summary/abstract

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a type of amyloidosisexternal link, opens in a new tab, which is a group of diseases where abnormal proteins (amyloids) build up and affect areas of the body. ATTR-CM is a long-term, progressive heart condition where the amyloids build up in the heart. This makes it harder for the heart to function properly.  

Vyndamaxexternal link, opens in a new tab was the first once-daily medicine for ATTR-CM that helps slow the progression of the disease and improve your quality of life.

There are two types of ATTR-CM (see table below). In both types, there is a buildup of a protein called transthyretin (TTR). TTR protein is made in the liver, brain, and retina of the eye. It helps to carry a thyroid hormone (thyroxine) and vitamin A (retinol) around the body. In ATTR-CM, the TTR protein is weak and unstable. This causes the protein to build up in the heart and other organs. The heart muscle becomes thick and stiffens, which leads to heart failureexternal link, opens in a new tab. 

ATTR-CM

Details

Symptoms

Wild-type 

(wtATTR)

  • Also called senile systemic amyloidosis
  • Occurs in older people due to age-related changes to the TTR protein
  • More commonly seen in White men over 60

 

Heart Failure Symptoms

  • Unusual tiredness (fatigue)
  • Shortness of breath or difficulty breathing
  • Swelling of feet, ankles, or legs
  • Unusually fast weight gain

 

Other Symptoms

  • Numbness, tingling or pain in your fingers, lower back, feet, or toes
  • Diarrhea or constipation
  • Nausea or feeling full quickly

Hereditary 

(hATTR)

  • Caused by a change (mutation) in your TTR gene
  • Hereditary means that the abnormal gene can be passed down from a close family member 
  • Most common mutation (V122I) is found almost always in Black people
  • Can affect other organs in the body besides the heart

Your health care provider will focus on treating your symptoms and recommend lifestyle changes for diet and exercise. Until the approval of Vyndamax, there was only symptom management or heart or liver transplants. Now, there are new medicines that reduce the amount of abnormal TTR proteins. Vyndamax contains tafamidis, which was the first active ingredient developed for ATTR-CM. Vyndamax helps to stabilize the TTR protein. This reduces the buildup of the protein and slows the progression of the disease. 

Authors
Beth Johnston, PharmD, BCPS