Vyndamax 61mg Capsules
It is used to treat a type of heart problem (cardiomyopathy).
Drug Class
Transthyretin Stabilizer
Pharmacologic Class
Selective transthyretin stabilizer
Pregnancy Category
Not available
FDA Approved
May 2019
DEA Schedule
Not Controlled
Overview
What is this medicine?
Vyndamax is a medicine used to treat a rare disease called transthyretin amyloid cardiomyopathy (ATTR-CM). This disease causes a protein called transthyretin to build up in the heart, making it stiff and unable to pump blood effectively. Vyndamax works by stabilizing this protein, preventing it from forming harmful deposits, and helping to reduce hospitalizations and improve survival for people with this condition.
How to Use This Medicine
Taking Your Medication Correctly
To get the most benefit from your medication, it's essential to take it exactly as directed by your doctor. Carefully read all the information provided with your prescription, and follow the instructions closely. When taking your medication, swallow the tablets whole - do not chew, break, or crush them. Continue taking your medication as prescribed by your doctor or healthcare provider, even if you start feeling well.
Storing and Disposing of Your Medication
To maintain the effectiveness and safety of your medication, store it at room temperature in a dry place, avoiding bathrooms. Keep all medications in a secure location, out of the reach of children and pets. Properly dispose of any unused or expired medication. Do not flush it down the toilet or pour it down the drain unless instructed to do so by your pharmacist or healthcare provider. If you have questions about disposing of your medication, consult your pharmacist, who can also inform you about potential drug take-back programs in your area.
What to Do If You Miss a Dose
If you miss a dose, take it as soon as you remember. However, if it's close to the time for your next scheduled dose, skip the missed dose and resume your regular dosing schedule. It's crucial not to take two doses at the same time or take extra doses to make up for the missed one.
To get the most benefit from your medication, it's essential to take it exactly as directed by your doctor. Carefully read all the information provided with your prescription, and follow the instructions closely. When taking your medication, swallow the tablets whole - do not chew, break, or crush them. Continue taking your medication as prescribed by your doctor or healthcare provider, even if you start feeling well.
Storing and Disposing of Your Medication
To maintain the effectiveness and safety of your medication, store it at room temperature in a dry place, avoiding bathrooms. Keep all medications in a secure location, out of the reach of children and pets. Properly dispose of any unused or expired medication. Do not flush it down the toilet or pour it down the drain unless instructed to do so by your pharmacist or healthcare provider. If you have questions about disposing of your medication, consult your pharmacist, who can also inform you about potential drug take-back programs in your area.
What to Do If You Miss a Dose
If you miss a dose, take it as soon as you remember. However, if it's close to the time for your next scheduled dose, skip the missed dose and resume your regular dosing schedule. It's crucial not to take two doses at the same time or take extra doses to make up for the missed one.
Lifestyle & Tips
- Take Vyndamax exactly as prescribed by your doctor, usually once a day.
- You can take Vyndamax with or without food.
- Do not crush, chew, or open the capsule; swallow it whole.
- Continue to follow your doctor's recommendations for managing your heart condition, including diet, exercise, and other medications.
- Report any new or worsening symptoms to your healthcare provider.
Available Forms & Alternatives
Available Strengths:
Dosing & Administration
Adult Dosing
Standard Dose:
61 mg orally once daily
Dose Range:
61 - 61 mg
Condition-Specific Dosing:
transthyretin_mediated_amyloidosis_cardiomyopathy:
61 mg orally once daily
Pediatric Dosing
Neonatal:
Not established
Infant:
Not established
Child:
Not established
Adolescent:
Not established
Dose Adjustments
Renal Impairment:
Mild:
No dose adjustment needed
Moderate:
No dose adjustment needed
Severe:
Not studied, use with caution
Dialysis:
Not studied, use with caution
Hepatic Impairment:
Mild:
No dose adjustment needed
Moderate:
No dose adjustment needed
Severe:
Not studied, use with caution
Pharmacology
Mechanism of Action
Tafamidis is a selective stabilizer of transthyretin (TTR). It binds to the thyroxine binding sites on the TTR tetramer, preventing its dissociation into monomers, which is the rate-limiting step in the amyloidogenic process. By stabilizing the TTR tetramer, tafamidis inhibits the formation of TTR amyloid fibrils, thereby reducing the deposition of amyloid in tissues and organs, particularly in the heart.
Pharmacokinetics
Absorption:
Bioavailability:
Not available (well absorbed)
Tmax:
2 hours
FoodEffect:
No clinically significant effect of food on absorption
Distribution:
Vd:
16 L
ProteinBinding:
More than 99%
CnssPenetration:
Limited
Elimination:
HalfLife:
Approximately 83 hours
Clearance:
0.22 L/h
ExcretionRoute:
Primarily feces (59%), urine (22%)
Unchanged:
Not available
Pharmacodynamics
OnsetOfAction:
Not precisely defined, but TTR stabilization occurs rapidly after administration.
PeakEffect:
Not precisely defined, but TTR stabilization occurs rapidly after administration.
DurationOfAction:
Due to long half-life, TTR stabilization is maintained with once-daily dosing.
Safety & Warnings
Side Effects
Urgent Side Effects: Seek Medical Help Right Away
While rare, some people may experience severe and potentially life-threatening side effects when taking this medication. If you notice any of the following symptoms, contact your doctor immediately or seek emergency medical attention:
* Signs of an allergic reaction, including:
+ Rash
+ Hives
+ Itching
+ Red, swollen, blistered, or peeling skin (with or without fever)
+ Wheezing
+ Tightness in the chest or throat
+ Trouble breathing, swallowing, or talking
+ Unusual hoarseness
+ Swelling of the mouth, face, lips, tongue, or throat
Other Possible Side Effects
Like all medications, this drug can cause side effects. However, many people do not experience any side effects or only have mild ones. If you have any side effects that bother you or persist, contact your doctor for advice.
Reporting Side Effects
This list is not exhaustive, and you may experience other side effects. If you have questions or concerns, discuss them with your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch. Your doctor is available to provide medical guidance on side effects.
While rare, some people may experience severe and potentially life-threatening side effects when taking this medication. If you notice any of the following symptoms, contact your doctor immediately or seek emergency medical attention:
* Signs of an allergic reaction, including:
+ Rash
+ Hives
+ Itching
+ Red, swollen, blistered, or peeling skin (with or without fever)
+ Wheezing
+ Tightness in the chest or throat
+ Trouble breathing, swallowing, or talking
+ Unusual hoarseness
+ Swelling of the mouth, face, lips, tongue, or throat
Other Possible Side Effects
Like all medications, this drug can cause side effects. However, many people do not experience any side effects or only have mild ones. If you have any side effects that bother you or persist, contact your doctor for advice.
Reporting Side Effects
This list is not exhaustive, and you may experience other side effects. If you have questions or concerns, discuss them with your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch. Your doctor is available to provide medical guidance on side effects.
Seek Immediate Medical Attention If You Experience:
- Signs of worsening heart failure (e.g., increased shortness of breath, swelling in legs/ankles, sudden weight gain)
- Unusual bleeding or bruising (though not a common side effect, report any unusual symptoms)
- Signs of liver problems (e.g., yellowing of skin or eyes, dark urine, severe stomach pain, unusual tiredness)
Before Using This Medicine
Before Taking This Medication: Important Information to Share with Your Doctor
It is essential to inform your doctor about the following:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the symptoms you experienced as a result of the allergy.
If you are breastfeeding, as you should not breastfeed while taking this medication.
To ensure safe treatment, it is crucial to discuss the following with your doctor and pharmacist:
All medications you are currently taking, including prescription and over-the-counter drugs, natural products, and vitamins.
Any existing health problems, as this medication may interact with other drugs or health conditions.
Before making any changes to your medication regimen, consult with your doctor to confirm it is safe to:
Start taking a new medication
Stop taking a medication
* Change the dose of any medication
It is essential to inform your doctor about the following:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the symptoms you experienced as a result of the allergy.
If you are breastfeeding, as you should not breastfeed while taking this medication.
To ensure safe treatment, it is crucial to discuss the following with your doctor and pharmacist:
All medications you are currently taking, including prescription and over-the-counter drugs, natural products, and vitamins.
Any existing health problems, as this medication may interact with other drugs or health conditions.
Before making any changes to your medication regimen, consult with your doctor to confirm it is safe to:
Start taking a new medication
Stop taking a medication
* Change the dose of any medication
Precautions & Cautions
It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication. If you are of childbearing age, you may need to use a reliable form of birth control to prevent pregnancy while taking this drug; discuss this with your doctor to determine the best option for you. This medication can cause harm to an unborn baby if taken during pregnancy. If you become pregnant or suspect you may be pregnant while taking this medication, contact your doctor immediately to discuss the potential risks and necessary next steps.
Overdose Information
Overdose Symptoms:
- No specific symptoms of overdose have been reported in clinical trials. In case of overdose, supportive measures should be taken.
What to Do:
Call 1-800-222-1222 (Poison Control Center) or seek emergency medical attention immediately.
Drug Interactions
Moderate Interactions
- BCRP substrates (e.g., methotrexate, rosuvastatin, imatinib, irinotecan, lapatinib, topotecan, sulfasalazine): Tafamidis is a BCRP inhibitor and may increase exposure to BCRP substrates.
- OATP1B1/1B3 substrates (e.g., statins like atorvastatin, rosuvastatin, pravastatin, simvastatin; valsartan, olmesartan, repaglinide): Tafamidis is an OATP1B1/1B3 inhibitor and may increase exposure to these substrates.
Monitoring
Baseline Monitoring
Diagnosis of ATTR-CM
Rationale: Confirm appropriate indication for treatment.
Timing: Prior to initiation
Cardiac function assessment (e.g., echocardiogram, MRI)
Rationale: Establish baseline cardiac status and disease severity.
Timing: Prior to initiation
Renal and hepatic function tests
Rationale: Assess baseline organ function, though no dose adjustment for mild/moderate impairment.
Timing: Prior to initiation
Routine Monitoring
Clinical assessment of ATTR-CM symptoms
Frequency: Regularly, as per clinical judgment (e.g., every 3-6 months)
Target: Stabilization or improvement of symptoms
Action Threshold: Worsening symptoms may indicate disease progression or need for re-evaluation.
Cardiac function assessment (e.g., echocardiogram)
Frequency: Periodically, as per clinical guidelines (e.g., annually or as needed)
Target: Stabilization or improvement of cardiac parameters
Action Threshold: Significant decline in cardiac function.
Symptom Monitoring
- Shortness of breath
- Fatigue
- Peripheral edema
- Palpitations
- Dizziness or lightheadedness
- Syncope
- Gastrointestinal symptoms (e.g., diarrhea, constipation, nausea)
- Peripheral neuropathy symptoms (e.g., numbness, tingling, pain)
Special Patient Groups
Pregnancy
There are no adequate and well-controlled studies of Vyndamax in pregnant women. Animal studies have shown developmental toxicity at exposures higher than clinical. Use during pregnancy only if the potential benefit justifies the potential risk to the fetus.
Trimester-Specific Risks:
First Trimester:
Potential risk based on animal data, but human data lacking.
Second Trimester:
Potential risk based on animal data, but human data lacking.
Third Trimester:
Potential risk based on animal data, but human data lacking.
Lactation
It is not known whether tafamidis is excreted in human milk. Due to the potential for serious adverse reactions in a breastfed infant, advise women not to breastfeed during treatment with Vyndamax.
Infant Risk:
Risk unknown, but potential for serious adverse reactions. Avoid breastfeeding.
Pediatric Use
Safety and effectiveness in pediatric patients have not been established. Not indicated for pediatric use.
Geriatric Use
No overall differences in safety or effectiveness were observed between patients 65 years of age and older and younger patients. No dose adjustment is needed based on age.
Clinical Information
Clinical Pearls
- Vyndamax (tafamidis meglumine) 61 mg is the recommended dose for ATTR-CM. Note that Vyndaqel (tafamidis meglumine) 20 mg and 80 mg are also available for ATTR polyneuropathy, but Vyndamax 61 mg is specifically for cardiomyopathy.
- Patients should be educated on the importance of consistent daily dosing due to the long half-life and the need for continuous TTR stabilization.
- This medication is extremely expensive, and patient assistance programs are crucial for access.
- While no specific lab monitoring is required for the drug itself, regular clinical assessment of cardiac function and symptoms is essential to monitor disease progression and treatment effectiveness.
Alternative Therapies
- Patisiran (Onpattro) - RNA interference therapeutic for hATTR amyloidosis with polyneuropathy (and some cardiac involvement in trials).
- Inotersen (Tegsedi) - Antisense oligonucleotide for hATTR amyloidosis with polyneuropathy.
- Vutrisiran (Amvuttra) - RNA interference therapeutic for hATTR amyloidosis with polyneuropathy (and some cardiac involvement in trials).
- Acoramidis (ATTRibute-CM trial, not yet approved for ATTR-CM) - another TTR stabilizer.
- Bridge to transplant (heart transplant) or supportive care for advanced disease.
Cost & Coverage
Average Cost:
Highly expensive (e.g., >$20,000 - $30,000) per 30 capsules
Insurance Coverage:
Specialty Tier (requires prior authorization, often with strict criteria)
General Drug Facts
If your symptoms or health issues persist or worsen, it's essential to contact your doctor for further guidance. To ensure your safety, never share your medication with others or take someone else's medication. Some medications may come with an additional patient information leaflet, so be sure to check with your pharmacist for more information. If you have any questions or concerns about your medication, don't hesitate to reach out to your doctor, nurse, pharmacist, or other healthcare provider. In the event of a suspected overdose, immediately call your local poison control center or seek medical attention. When seeking help, be prepared to provide details about the medication taken, the amount, and the time it happened.