Acoramidis
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Drug Status
Government Approvals
US(FDA)
WHO Essential Medicine
NO
Known Teratogen
NO
Pharmaceutical Class
None
Controlled Drug Substance
NO
Acoramidis
Learn more about this medicine -
Click HereSummary
Acoramidis is used to treat a condition called cardiomyopathy associated with wild-type or variant transthyretin-mediated amyloidosis (ATTRCM) in adults. This condition involves the buildup of harmful protein deposits, called amyloid, in the heart.
Acoramidis works by stabilizing a protein called transthyretin. This protein can form harmful amyloid deposits in the heart. Acoramidis binds to this protein and slows down its breakdown, which reduces the formation of these deposits.
The usual dose for adults is 712 mg of Acoramidis taken orally twice daily. The tablets should be swallowed whole and can be taken with or without food. It's important to follow your healthcare provider's advice on how to take this medication.
The most commonly reported side effects of Acoramidis are gastrointestinal, including diarrhea (11.6%) and upper abdominal pain (5.5%). These side effects are generally mild and resolve without stopping the medication.
Patients should avoid using Acoramidis with UGT inducers and strong CYP3A inducers, as these can decrease its effectiveness. Also, it's important to monitor for changes in serum creatinine and eGFR, as these can occur with treatment initiation. Always consult your healthcare provider for personalized advice.
Indications and Purpose
How does Acoramidis work?
Acoramidis acts as a selective stabilizer of transthyretin (TTR), a protein that can misfold and form amyloid deposits. It binds to TTR at thyroxine binding sites, slowing the dissociation of the TTR tetramer into monomers, which is the rate-limiting step in amyloid formation. This stabilization helps prevent the progression of amyloidosis.
How does one know if Acoramidis is working?
The benefit of Acoramidis is evaluated through clinical studies that measure its impact on all-cause mortality and cardiovascular-related hospitalizations. Additionally, improvements in functional capacity and health status, such as the Six-Minute Walk Distance and Kansas City Cardiomyopathy Questionnaire scores, are used to assess its effectiveness.
Is Acoramidis effective?
The efficacy of Acoramidis was demonstrated in a multicenter, international, randomized, double-blind, placebo-controlled study involving 611 adult patients with wild-type or variant ATTR-CM. The study showed a statistically significant reduction in all-cause mortality and cardiovascular-related hospitalizations in patients treated with Acoramidis compared to placebo. Improvements in functional capacity and health status were also observed, supporting its effectiveness.
What is Acoramidis?
Acoramidis is used to treat cardiomyopathy caused by transthyretin-mediated amyloidosis (ATTR-CM) in adults, reducing cardiovascular death and hospitalizations. It works by stabilizing transthyretin, a protein that can misfold and form amyloid deposits in the heart. By binding to transthyretin, Acoramidis slows the dissociation of its tetramer structure, preventing amyloid formation.
What is Acoramidis used for?
Acoramidis is indicated for the treatment of cardiomyopathy caused by wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults. It is used to reduce the risk of cardiovascular death and hospitalizations related to heart problems in these patients.
Directions for Use
For how long do I take Acoramidis?
Acoramidis is typically used for a duration of 30 months, as demonstrated in clinical studies. However, the exact duration may vary based on individual patient needs and should be determined by a healthcare provider.
How do I take Acoramidis?
Take Acoramidis exactly as prescribed by your healthcare provider, usually 712 mg orally twice a day. It can be taken with or without food. Swallow the tablets whole without cutting, crushing, or chewing them. There are no specific food restrictions mentioned, but always consult your healthcare provider for personalized advice.
How long does it take for Acoramidis to start working?
Acoramidis begins to stabilize transthyretin as early as Day 28, with near-complete stabilization observed in clinical studies. However, the full therapeutic effects, such as reduced mortality and hospitalizations, are typically evaluated over a longer period, such as 30 months.
How should I store Acoramidis?
Store Acoramidis tablets at controlled room temperature between 68°F to 77°F (20°C to 25°C). Keep the tablets in their original blister card until use to protect them from moisture. Ensure that the medication is kept out of reach of children.
What is the usual dose of Acoramidis?
The usual daily dose for adults is 712 mg taken orally twice a day. The safety and effectiveness of Acoramidis in children have not been established, so there is no recommended dose for pediatric patients.
Warnings and Precautions
Can Acoramidis be taken safely while breastfeeding?
There is no available data on the presence of Acoramidis in human or animal milk, or its effects on the breastfed infant or milk production. The benefits of breastfeeding should be weighed against the mother's need for Acoramidis and any potential adverse effects on the child. Consult a healthcare provider for personalized advice.
Can Acoramidis be taken safely while pregnant?
There is insufficient data on the use of Acoramidis in pregnant women to establish a risk of major birth defects or miscarriage. Animal studies in rats and rabbits did not show embryofetal abnormalities at high exposures. Pregnant women should report their condition to the BridgeBio reporting line and consult their healthcare provider for personalized advice.
Can I take Acoramidis with other prescription drugs?
Acoramidis is metabolized by UGT enzymes, so using it with UGT inducers can decrease its exposure. Strong CYP3A inducers can also affect UGT enzymes. Acoramidis inhibits CYP2C9, which may increase concentrations of CYP2C9 substrates. Patients should be monitored for signs of increased exposure when taking these drugs together.
Is Acoramidis safe for the elderly?
No dosage adjustment is required for elderly patients (65 years and older). In clinical studies, 97% of participants were 65 years and over, with a median age of 78 years. However, elderly patients should still be monitored closely for any adverse effects or interactions with other medications they may be taking.
Who should avoid taking Acoramidis?
There are no specific contraindications for Acoramidis. However, patients should be aware of potential drug interactions, especially with UGT inducers and strong CYP3A inducers, which can decrease Acoramidis exposure. Patients should also monitor for gastrointestinal side effects and changes in kidney function, as these have been observed in clinical trials.