Acoramidis

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Summary

What conditions is Acoramidis used for?
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.
How does Acoramidis work in the body?
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.
What are the usual dosages and routes of administration for Acoramidis?
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.
What are the most commonly observed adverse effects of Acoramidis?
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.
What are important safety warnings and contraindications for Acoramidis?
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.

Approvals

N/A

Essential Medicine

NO

Teratogenicity

N/A

Pharmaceutical Class

NA

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Drug Ingredient

Acoramidis

Know More About This Medicine -

Summary

What conditions is Acoramidis used for?
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.
How does Acoramidis work in the body?
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.
What are the usual dosages and routes of administration for Acoramidis?
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.
What are the most commonly observed adverse effects of Acoramidis?
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.
What are important safety warnings and contraindications for Acoramidis?
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

What is Acoramidis used for?

Acoramidis is indicated for the treatment of cardiomyopathy associated with wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults. This condition involves the buildup of amyloid deposits in the heart, leading to heart muscle disease and increased risk of cardiovascular death and hospitalization.

How does Acoramidis work?

Acoramidis acts as a selective stabilizer of transthyretin (TTR), a protein that can form amyloid deposits in the heart. 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 reduce amyloid deposits and their associated cardiac effects.

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 the Acoramidis group compared to the placebo group over 30 months. Improvements in functional capacity and health status were also observed.

How does one know if Acoramidis is working?

The benefit of Acoramidis is evaluated through clinical studies that measure outcomes such as all-cause mortality, cardiovascular-related hospitalizations, functional capacity, and health status. These outcomes are assessed using tools like the Six-Minute Walk Distance (6MWD) and the Kansas City Cardiomyopathy Questionnaire-Overall Summary score (KCCQ-OS).

Directions for Use

What is the usual dose of Acoramidis?

The usual daily dose for adults is 712 mg, taken orally twice daily. The safety and effectiveness of Acoramidis have not been established in pediatric patients, so there is no recommended dose for children.

How do I take Acoramidis?

Acoramidis should be taken orally at a dose of 712 mg twice daily, with or without food. The tablets should be swallowed whole and not cut, crushed, or chewed. There are no specific food restrictions mentioned, but patients should follow their healthcare provider's advice.

For how long do I take Acoramidis?

The typical duration of use for Acoramidis in clinical trials was 30 months. However, the actual duration of treatment may vary based on individual patient needs and should be determined by a healthcare provider.

How long does it take for Acoramidis to start working?

Increases in mean serum transthyretin (TTR) levels, indicating the pharmacodynamic effect of Acoramidis, were observed by Day 28 in patients. Near-complete in vitro TTR stabilization was also observed by this time, suggesting that the medication begins to exert its effects within the first month of treatment.

How should I store Acoramidis?

Acromidis should be stored at controlled room temperature, between 20°C to 25°C (68°F to 77°F), with permitted excursions to 15°C to 30°C (59°F to 86°F). It should be kept in its original blister card until use to protect from moisture.

Warnings and Precautions

Who should avoid taking Acoramidis?

There are no specific contraindications listed for Acoramidis. However, patients should be aware of potential drug interactions, particularly with UGT inducers and strong CYP3A inducers, which can decrease Acoramidis exposure. Patients should also be monitored for changes in serum creatinine and eGFR, as these can occur with treatment initiation.

Can I take Acoramidis with other prescription drugs?

Acoramidis is metabolized by UGT enzymes, and its exposure can be decreased by UGT inducers. It also inhibits CYP2C9, which may increase concentrations of CYP2C9 substrates. Patients should avoid concomitant use with UGT inducers and strong CYP3A inducers, and healthcare providers should monitor for increased exposure to CYP2C9 substrates.

Can I take Acoramidis with vitamins or supplements?

The provided content does not mention specific interactions between Acoramidis and vitamins or supplements. However, patients should always inform their healthcare provider about all medications and supplements they are taking to avoid potential interactions.

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 pregnancy to the BridgeBio reporting line and consult their healthcare provider for guidance.

Can Acoramidis be taken safely while breastfeeding?

There are 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. Consultation with a healthcare provider is recommended.

Is Acoramidis safe for the elderly?

No dosage adjustment is required for elderly patients (≥65 years). The clinical study included a large proportion of elderly participants, with 97% being 65 years and over, and a median age of 78 years. Elderly patients should follow the same dosage instructions as other adults, but should always consult their healthcare provider for personalized advice.

Is it safe to exercise while taking Acoramidis?

The provided content does not indicate that Acoramidis limits the ability to exercise. In fact, the medication is used to treat cardiomyopathy, which may improve exercise capacity. Patients should consult their healthcare provider for personalized advice on exercise.

Is it safe to drink alcohol while taking Acoramidis?

The provided content does not mention any specific interactions between alcohol and Acoramidis. However, it is generally advisable to consult with a healthcare provider regarding alcohol consumption while taking any medication, as alcohol can potentially affect the metabolism and effectiveness of drugs.