This page contains brief details about the drug , it's indication, dosage & administaration, mechanism of action, related brands with strength, warnings and common side effects.
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 May 3, 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 a significant advancement for individuals living with this rare disease.
Mechanism of Action of undefined
This medicine works by binding to a protein in the body called transthyretin. Normally, transthyretin helps carry substances like vitamin A and thyroid hormones through the blood. However, in certain diseases, this protein can break apart and form clumps that damage organs, especially the heart. By stabilising the transthyretin protein, Tafamidis helps prevent these harmful clumps from forming. This way, it slows down the damage to the heart and improves the symptoms associated with the disease.
Uses of undefined
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).
undefined Drug administaration and Dosage available
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.
Warnings, Precautions and Side Effects of undefined
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.
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.
Word Of Advice
Taking Tafamidis consistently at the same time each day can help maintain its effectiveness. It should be swallowed whole and not crushed or split. Patients should maintain regular appointments with their doctor to monitor heart function and liver health. Eating a balanced diet and staying physically active, as advised by a doctor, can further support heart health. Always inform any new physician that you are taking this medicine before starting other treatments.
Frequently Asked Question
References
Disclaimer
The content provided here is for informational purposes only and should not be considered a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of your physician or another qualified healthcare provider with any questions you may have regarding a medical condition.
