Tafamidis
Transthyretin-mediated Amyloidosis
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Drug Status
Government Approvals
US(FDA)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
NA
Controlled Drug Substance
No
Tafamidis
Summary
Tafamidis is used to treat a condition called transthyretin amyloid cardiomyopathy (ATTRCM). This is a condition where a protein builds up in the heart, making it difficult for the heart to pump blood.
Tafamidis works by stabilizing the transthyretin protein. This prevents the protein from depositing in the heart, which helps reduce heart-related issues like cardiovascular mortality and hospitalizations.
The usual daily dose for adults is either 80 mg of Tafamidis meglumine (four 20mg capsules) or 61 mg of Tafamidis (one capsule). It is taken orally once a day.
The document does not provide specific information on the most commonly observed adverse effects of Tafamidis.
Tafamidis can cause fetal harm, so pregnant women should be aware of potential risks. It is also present in rat milk, so breastfeeding women are advised not to take it. Additionally, it can increase the exposure and risk of toxicities related to drugs that are BCRP substrates, like methotrexate, rosuvastatin, and imatinib.
Indications and Purpose
How does Tafamidis work?
Tafamidis acts as a selective stabilizer of the transthyretin (TTR) protein. It binds to TTR at the thyroxine binding sites, stabilizing the tetramer and slowing its dissociation into monomers, which is the rate-limiting step in the amyloidogenic process. This stabilization helps prevent the formation of amyloid deposits in the heart.
Is Tafamidis effective?
Tafamidis has been shown to be effective in a multicenter, international, randomized, double-blind, placebo-controlled study involving 441 patients with wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM). The study demonstrated a significant reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations in patients treated with tafamidis compared to placebo. Additionally, improvements in functional capacity and health status were observed, supporting its effectiveness in managing ATTR-CM.
What is Tafamidis?
Tafamidis is used to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where a protein builds up in the heart, making it difficult to pump blood. It works by stabilizing the transthyretin protein, preventing its deposits in the heart. This helps reduce cardiovascular mortality and hospitalizations related to heart problems.
Directions for Use
For how long do I take Tafamidis?
Tafamidis is typically used as a long-term treatment for transthyretin amyloid cardiomyopathy (ATTR-CM). It is important to continue taking it even if you feel well, as it controls the condition but does not cure it. Always follow your doctor's guidance on the duration of use.
How do I take Tafamidis?
Tafamidis should be taken orally once a day, with or without food. It is important to take it at the same time every day. There are no specific food restrictions associated with tafamidis, but patients should follow their doctor's instructions regarding diet and medication use.
How should I store Tafamidis?
Tafamidis should be stored in its original container, tightly closed, and kept out of reach of children. It should be stored at room temperature, away from excess heat and moisture, and not in the bathroom. To prevent accidental ingestion by children, always lock safety caps and store the medication in a safe location.
What is the usual dose of Tafamidis?
The usual daily dose for adults is either 80 mg of tafamidis meglumine (four 20-mg capsules) or 61 mg of tafamidis (one capsule) taken orally once a day. The safety and effectiveness of tafamidis in children have not been established.
Warnings and Precautions
Can I take Tafamidis with other prescription drugs?
Tafamidis inhibits breast cancer resistant protein (BCRP) in humans. Coadministration with drugs that are BCRP substrates, such as methotrexate, rosuvastatin, and imatinib, may increase the exposure and risk of toxicities related to these substrates. Patients should be monitored for signs of BCRP substrate-related toxicities, and dosage adjustments may be necessary.
Can Tafamidis be taken safely while breastfeeding?
There are no available data on the presence of tafamidis in human milk, but it is present in rat milk. Due to the potential for serious adverse reactions in breastfed infants, it is recommended that women do not breastfeed while taking tafamidis. Patients should discuss alternative feeding options with their healthcare provider.
Can Tafamidis be taken safely while pregnant?
Tafamidis may cause fetal harm based on animal studies, although limited human data have not identified specific risks for major birth defects or miscarriage. Pregnant women should be advised of the potential risk to the fetus, and pregnancy should be reported to the Pfizer reporting line. Women of reproductive potential should consider pregnancy planning and prevention while on tafamidis.
Is Tafamidis safe for the elderly?
No dosage adjustment is required for elderly patients (65 years and older). In clinical studies, a significant portion of participants were elderly, with a median age of 75 years. However, as with any medication, elderly patients should be monitored closely for any adverse effects or interactions with other medications they may be taking.
Who should avoid taking Tafamidis?
There are no specific contraindications for tafamidis. However, patients should inform their doctor if they have liver disease, are pregnant, plan to become pregnant, or are breastfeeding. Tafamidis may cause fetal harm, so it is important to discuss pregnancy plans with a healthcare provider. Patients should also inform their doctor about all medications, vitamins, and supplements they are taking to avoid potential interactions.
