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Medications for Transthyretin-Related Amyloidosis

Other names: hATTR; Hereditary Transthyretin-Related Amyloidosis; Transthyretin-Related Amyloidosis

Medically reviewed by Carmen Pope, BPharm. Last updated on Feb 19, 2024.

Patisiran (Onpattro), vutrisiran (Amvuttra), inotersen (Tegsedi), and eplontersen (Waiuna) may be used to stop or reverse the progression of polyneuropathy (damage to multiple nerves) associated with hereditary transthyretin-related amyloidosis (hATTR).

Tafamidis (Vyndamax, Vyndaqel) helps to stabilize the transthyretin protein, and may be used to treat cardiomyopathy associated with a build-up of TTR.

Transthyretin-related amyloidosis (ATTR) is characterized by the deposition in organs of a normal or mutated form of transthyretin protein (this is a transport proteins that transports thyroid hormone [T4] and retinol throughout the body). There are 2 types – hereditary hATTR which is caused by a genetic mutation in the transthyretin gene, and wild type (wATTR), which is caused by the normal transthyretin protein becoming structurally unstable with age and depositing in organs.

Drugs used to treat Transthyretin-Related Amyloidosis

The medications listed below are related to or used in the treatment of this condition.

Filter
Drug name Rating Reviews Activity ? Rx/OTC Pregnancy CSA Alcohol
Onpattro Rate Add review
Rx N
Generic name:
patisiran systemic
Drug class:
miscellaneous metabolic agents
For consumers:
dosage, side effects
For professionals:
Prescribing Information
Tegsedi Rate Add review
Rx N
Generic name:
inotersen systemic
Drug class:
miscellaneous metabolic agents
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
eplontersen Rate Add review
Rx N
Generic name:
eplontersen systemic
Brand name:
Wainua
Drug class:
miscellaneous metabolic agents
For consumers:
dosage, side effects
For professionals:
AHFS DI Monograph
Vyndamax 10 1 review for Vyndamax to treat Transthyretin-Related Amyloidosis
Rx N
Generic name:
tafamidis systemic
Drug class:
transthyretin stabilizers
For consumers:
dosage, interactions, side effects
tafamidis 10 1 review for tafamidis to treat Transthyretin-Related Amyloidosis
Rx N
Generic name:
tafamidis systemic
Brand names:
Vyndamax, Vyndaqel
Drug class:
transthyretin stabilizers
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
inotersen Rate Add review
Rx N
Generic name:
inotersen systemic
Brand name:
Tegsedi
Drug class:
miscellaneous metabolic agents
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
patisiran Rate Add review
Rx N
Generic name:
patisiran systemic
Brand name:
Onpattro
Drug class:
miscellaneous metabolic agents
For consumers:
dosage, side effects
For professionals:
AHFS DI Monograph
Vyndaqel Rate Add review
Rx N
Generic name:
tafamidis systemic
Drug class:
transthyretin stabilizers
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
vutrisiran Rate Add review
Rx N
Generic name:
vutrisiran systemic
Drug class:
miscellaneous metabolic agents
For consumers:
dosage, side effects
For professionals:
AHFS DI Monograph
Wainua Rate Add review
Rx N
Generic name:
eplontersen systemic
Drug class:
miscellaneous metabolic agents
For consumers:
dosage, side effects
For professionals:
Prescribing Information

Frequently asked questions

Learn more about Transthyretin-Related Amyloidosis

Care guides

Legend

Rating For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective).
Activity Activity is based on recent site visitor activity relative to other medications in the list.
Rx Prescription only.
OTC Over-the-counter.
Rx/OTC Prescription or Over-the-counter.
Off-label This medication may not be approved by the FDA for the treatment of this condition.
EUA An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.
Expanded Access Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Pregnancy Category
A Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
B Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
C Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
D There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
X Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
N FDA has not classified the drug.
Controlled Substances Act (CSA) Schedule
M The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
U CSA Schedule is unknown.
N Is not subject to the Controlled Substances Act.
1 Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
2 Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
3 Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
4 Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
5 Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
Alcohol
X Interacts with Alcohol.

Further information

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