Amvuttra, Onpattro, Tegsedi, and More: 6 Medications for Hereditary ATTR
Hereditary ATTR amyloidosis is a rare and underdiagnosed inherited condition that commonly affects the heart and nerves. Here are six hATTR treatments to know.
Amvuttra
Used for Hereditary Transthyretin-Mediated Amyloidosis
Used for Hereditary Transthyretin-Mediated Amyloidosis
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.
What is Amvuttra (vutrisiran)?
What is Amvuttra (vutrisiran) used for?
- Polyneuropathy (nerve damage) from hereditary ATTR amyloidosis (hATTR)
- Cardiomyopathy (heart muscle problems) from wild-type transthyretin-mediated amyloidosis (ATTRwt) or hATTR
How Amvuttra (vutrisiran) works
Amvuttra (vutrisiran) is a small interfering ribonucleic acid (siRNA). It works by blocking the genetic message (mRNA) that tells your body to make a harmful protein called transthyretin (TTR). When too much TTR builds up, it can form clumps that damage your nerves and heart. By lowering TTR levels, Amvuttra (vutrisiran) helps reduce nerve damage and heart muscle problems.
When does Amvuttra (vutrisiran) start working?Time passed since treatment started:
Drug facts
| Common Brands | Amvuttra |
|---|---|
| Drug Class | Small interfering ribonucleic acid (siRNA) |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
Side effects of Amvuttra (vutrisiran)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Pain in arms and legs (15%)
- Joint pain (11%)
- Shortness of breath (7%)
- Lower vitamin A levels (7%)
Less Common Side Effects
When do Amvuttra (vutrisiran) possible side effects begin and end?
Time passed since treatment started:
M
H
D
W
M
Injection-site reactions
The following Amvuttra (vutrisiran) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Low vitamin A level—change in vision, dry eyes, dry skin, frequent infections
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Pros and cons of Amvuttra (vutrisiran)
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
Pharmacist tips for Amvuttra (vutrisiran)
- Amvuttra (vutrisiran) is injected under the skin in your stomach, thighs, or upper arms by a healthcare professional every 3 months. Mark your appointments on a calendar so you don’t forget.
- If you miss a dose of Amvuttra (vutrisiran), contact your prescriber as soon as possible to reschedule your appointment. Your next doses should then be scheduled every 3 months from that date.
- Amvuttra (vutrisiran) can lower the amount of vitamin A in your blood. So your prescriber might recommend that you take a vitamin A supplement every day.
- Tell your prescriber right away if you notice any vision changes, such as night blindness, dry eyes, cloudy corneas, or new spots in your vision. These might be signs that your vitamin A levels are too low.
- Don't take more vitamin A than what your prescriber recommends. Too much vitamin A can cause symptoms such as severe headache, nausea, dizziness, and muscle aches.
- Amvuttra (vutrisiran) hasn’t been studied in pregnant women, but animal studies show it could harm unborn babies. And since this medication can also lower vitamin A levels, it might also affect the baby's development. If you’re pregnant or planning to become pregnant, talk with your prescriber about the risks and benefits of taking this medication.
Risks and warnings for Amvuttra (vutrisiran)
Amvuttra (vutrisiran) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Low vitamin A levels
Amvuttra (vutrisiran) can lower the vitamin A in your blood, which might cause problems like poor night vision and raise your risk of infections. Your prescriber might recommend a vitamin A supplement while you're taking this medication. But don't take more than directed since too much vitamin A causes severe headache, nausea, dizziness, and muscle aches. Let your care team know if you notice blurry vision or trouble seeing at night because these can be signs of low vitamin A.
Amvuttra (vutrisiran) dosage
Typical dosage for Amvuttra (vutrisiran)
The typical dose is 25 mg injected under the skin by a healthcare professional once every 3 months.
What are alternatives to Amvuttra (vutrisiran)?
There are a number of medications that your doctor can prescribe in place of Amvuttra (vutrisiran). Compare a few possible alternatives below.
News about Amvuttra (vutrisiran)
Amvuttra (vutrisiran) images
