Vyndaqel

Vyndaqel is used to to reduce death and hospitalizations related to heart problems in adults with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM occurs when abnormal proteins clump together and form amyloid fibrils which travel through the bloodstream and are deposited in many organs, including the heart. As fibrils accumulate in the tissue, they thicken and stiffen the wall of the heart. This causes a cardiomyopathy and, ultimately, heart failure.

Vyndaqel belongs to a class of drugs called selective TTR stabilizers. Vyndaqel binds to the transthyretin protein that causes ATTR-CM, slowing its breakdown and the buildup of dangerous deposits. By doing so, Vyndaqel can slow the progression of the disease.

Vyndaqel is available as four 20-mg capsules, taken once a day, with or without food. Swallow Vyndaqel capsules whole, do not crush or cut.

No side effects were identified during treatment with Vyndaqel in clinical trials.

Vyndaqel is a prescription medication used to treat adults with cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) to reduce death and hospitalization related to heart problems.

This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.

No side effects were identified during treatment with Vyndaqel in clinical trials.

Tell your doctor if you have any side effect that bothers you or does not go away.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Tell your healthcare provider about all the medicines you take including any prescription or over-the-counter medicines, vitamins, and herbal supplements.

Especially tell your healthcare provider if you take any of the following medicines:

• non-steroidal anti-inflammatory drugs
• diuretic medicines (e.g. furosemide, bumetanide)
• anti-cancer medicines (e.g. methotrexate, imatinib)
• statins (e.g. rosuvastatin)
• anti-viral medicines (e.g. oseltamivir, tenofovir, ganciclovir, adefovir, cidofovir, lamivudine, zidovudine, zalcitabine)

This is not a complete list of Vyndaqel drug interactions. Ask your doctor or pharmacist for more information.

Do not use Vyndaqel for a condition for which it was not prescribed. Do not give this medication to other people, even if they have the same symptoms you have. It may harm them.

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of Vyndaqel, there are no specific foods that you must exclude from your diet when receiving this medication.

Before taking Vyndaqel, tell your healthcare provider about all your medical conditions, including if you:

• have liver problems.
• are pregnant or plan to become pregnant.
• are breastfeeding or plan to breastfeed.

Tell your healthcare provider about all the medicines you take including any prescription or over‑the‑counter medicines, vitamins, and herbal supplements.

Tell your doctor if you are pregnant or plan to become pregnant.

There are no well-done studies that have been done in humans with Vyndaqel. In animal studies, pregnant animals were given this medication and several problems where observed related to this medication. Additionally, in some situations, fetal death was observed. Based on findings from animal studies, Vyndaqel may cause harm to the unborn baby when administered to a pregnant woman. Therefore, this medication may be used, but only if the potential benefits to the mother outweigh the potential risks to the unborn child.

Tell your doctor if you are breastfeeding or plan to breastfeed.

It is not known if Vyndaqel crosses into human milk. Because many medications can cross into human milk and because the possibility for serious adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will decide if the benefits outweigh the risk of using Vyndaqel.

• Take Vyndaqel exactly as your healthcare provider tells you to.
• Take Vyndaqel capsules 1 time a day.
• Vyndaqel capsules should be swallowed whole and not crushed or cut.
• If you miss a dose, take it as soon as you remember. If it is almost time for your next dose, skip the missed dose and take the next dose at your regularly scheduled time. Do not take 2 doses at the same time.
• There is a Transthyretin Amyloidosis Outcome Survey (THAOS) registry for people who receive treatment with Vyndaqel. Talk to your healthcare provider about how you can take part in this registry. For more information about this registry, go to https://clinicaltrials.gov.

Take Vyndaqel exactly as prescribed by your doctor. Follow the directions on your prescription label carefully.

The recommended dosage is Vyndaqel 80 mg orally once daily.

If you take too much of this medication, call your healthcare provider or local Poison Control Center, or seek emergency medical attention right away.

• Store Vyndaqel capsules at room temperature between 68°F to 77°F (20°C to 25°C).
• Keep Vyndaqel and all medicines out of the reach of children.