TAFAMIDIS
Background and Date of Approval
Tafamidis is a medicine developed to treat a rare but serious heart condition linked to abnormal protein buildup in the body. It works by stabilising specific proteins, preventing them from clumping together and causing damage. The U.S. Food and Drug Administration (FDA) approved it on 3 May 2019, after clinical trials showed that it helped reduce hospitalisation rates and improved survival among patients with heart conditions caused by transthyretin amyloidosis. One key trial, known as the ATTR-ACT study, demonstrated that patients taking this medication had better outcomes compared to those who did not. Tafamidis represents an advancement for patients with this rare disease.
Uses
Tafamidis is approved for the treatment of: Transthyretin amyloid cardiomyopathy (ATTR-CM) in adults (a condition where abnormal proteins build up in the heart, making it harder for the heart to pump blood properly). Wild-type transthyretin amyloidosis (wtATTR) (this happens naturally with ageing, without any inherited cause, and affects the heart). Hereditary transthyretin amyloidosis (hATTR) (this form is passed down through families and also causes heart problems due to protein buildup).
How is it administered?
Tafamidis is taken orally, meaning it is swallowed as a capsule with water. It should ideally be taken once daily, at the same time each day, to maintain consistent levels in the body. It is available in two strengths: 20 mg and 61 mg capsules. Healthcare providers typically recommend the 61 mg dose for most adults with heart-related conditions linked to transthyretin amyloidosis.
Side Effects
Common Side Effects: Diarrhea Urinary tract infection Muscle pain Fatigue Cough Stomach pain Serious Side Effects: Liver function abnormalities Shortness of breath Swelling in the lower legs or ankles (edema) Worsening of heart failure symptoms Severe allergic reactions (rare) It is important to contact a healthcare provider if any serious side effects are noticed.
Warnings
Liver issues: Regular monitoring of liver function is advised. Pregnancy and breastfeeding: Should not be used during pregnancy or while breastfeeding unless clearly needed. Drug interactions: Caution is needed when taking medications that affect liver enzymes, such as rifampin or carbamazepine. Severe heart failure: Patients with very advanced heart failure should use with caution.
Precautions
Before starting Tafamidis, doctors usually assess liver function and overall heart health. Women who are pregnant or planning to become pregnant should discuss potential risks with their doctor, as the safety for unborn babies is not well-established. It is also important for patients to share a full list of all medications and supplements they are taking to avoid potential interactions. Regular follow-ups are necessary to monitor the progression of the disease and the body's response to the medication.
Word of Advice
Consistent dosing of tafamidis at the same time each day helps maintain therapeutic levels. The tablet should be swallowed whole; do not crush or split. Patients with cardiac amyloidosis require regular monitoring appointments to assess heart function and liver health. A balanced diet and physical activity, as recommended by the treating physician, may provide additional cardiovascular support. Inform all new physicians that you are taking tafamidis before commencing any additional treatments.
References
https://www.pfizermedicalinformation.com/vyndaqel-or-vyndamax https://www.drugs.com/history/vyndamax.html https://www.nejm.org/doi/full/10.1056/NEJMoa1805689 https://pmc.ncbi.nlm.nih.gov/articles/PMC8048553/ https://pmc.ncbi.nlm.nih.gov/articles/PMC10783421/
Frequently Asked Questions
What is Tafamidis used for?
Tafamidis is used to treat a heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). It helps prevent abnormal protein buildup in the heart, which can make it easier for the heart to work properly and may reduce the risk of hospitalisations and heart-related problems.
What is the cost of Tafamidis in India?
Tafamidis is a high-cost medication in India. The price can vary depending on the brand and source, but it typically costs between â¹5,00,000 and â¹7,00,000 per month. MrMed offers Tafamidis at a discounted price of around â¹1,17,000 , making it more affordable for patients. It is important to check with authorised pharmacies or hospitals for exact pricing and availability.
What is the best time to take Tafamidis?
It is best to take Tafamidis at the same time every day, preferably in the morning. Consistency helps keep the medication levels steady in the body.
Should Tafamidis be taken before or after meals?
Tafamidis can be taken with or without food. However, taking it with a meal may help if you experience stomach upset.
How long do you have to take Tafamidis?
Tafamidis is usually prescribed as a long-term treatment. Patients are advised to continue taking it as directed by their healthcare provider unless told otherwise.