Amvuttra vs. Vyndaqel for Hereditary Transthyretin-Mediated Amyloidosis and Heart Failure: Important Differences and Potential Risks.

Amvuttra

(vutrisiran)

Vyndaqel

(tafamidis)

Summary for Amvuttra

Prescription only

Amvuttra (vutrisiran) is a prescription injection used to treat nerve damage (polyneuropathy) in adults with a rare condition called hereditary transthyretin-mediated amyloidosis (hATTR). It's also FDA approved to treat heart muscle problems (cardiomyopathy) in adults with either wild-type transthyretin-mediated amyloidosis (ATTRwt) or hATTR. This medication belongs to a drug class called small interfering ribonucleic acids (siRNAs) and is injected under the skin once every 3 months by a healthcare professional. Some common side effects include joint pain and shortness of breath.

Indications for Amvuttra

Polyneuropathy (nerve damage) from hereditary ATTR amyloidosis (hATTR)
Cardiomyopathy (heart muscle problems) from wild-type transthyretin-mediated amyloidosis (ATTRwt) or hATTR

Pros and Cons for Amvuttra

Pros

Injected once every 3 months
Given under the skin, instead of through the veins
Doesn't require pretreatment medications
Treats both nerve and heart problems from ATTR

Cons

Likely to cause low vitamin A levels
Needs to be given by a healthcare professional
Unknown if safe or works well in children
Maybe not safe during pregnancy

Common Side Effects for Amvuttra

Pain in arms and legs (15%)
Joint pain (11%)
Shortness of breath (7%)
Lower vitamin A levels (7%)

Warnings for Amvuttra

Low vitamin A levels

Summary for Vyndaqel

Prescription only

Vyndaqel (tafamidis) is a medication taken by mouth used for a rare type of heart disease called transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). It's available as two different brand names and is taken once per day.

Indications for Vyndaqel

Transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM, a rare heart muscle disease)

Pros and Cons for Vyndaqel

Pros

The first once-daily, oral treatment for ATTR-CM
Can help lower chance of hospitalizations and death in people with ATTR-CM
Can be taken with or without food
Manufacturer financial assistance available

Cons

Need to take four capsules for one dose of Vyndaqel (tafamidis)
Available as brand names only, which can be very expensive
Not recommended for use during pregnancy

Common Side Effects for Vyndaqel

The following side effects listed in the supplemental appendix of a major clinical study were similar in people taking Vyndaqel (tafamidis) and people taking placebo. Side effects were generally mild to moderate in severity.