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ATTR Amyloidosis Treatments Compared

Name: ATTR Amyloidosis Treatments Compared
Creator: Jason Hunter
Published: 2026-10-01

5 FDA-approved drugs and 1 in development, side by side from the current prescribing labels. Newest label Jun 2026. Checked October 1, 2026.

Nerve form, TTR silencersOnpattro · Amvuttra · Wainua

Heart form, TTR stabilizersVyndaqel / Vyndamax · Attruby

Both formsAmvuttra

Five drugs are FDA-approved for transthyretin (ATTR) amyloidosis, and which ones apply depends on the form. For the nerve form, hereditary ATTR polyneuropathy, the options are Onpattro, Amvuttra and Wainua, which silence production of the TTR protein. For the heart form, ATTR cardiomyopathy, the options are Vyndaqel/Vyndamax and Attruby, pills that stabilize the protein, plus Amvuttra, the only drug approved for both forms. Onpattro is a clinic infusion every 3 weeks, Amvuttra a clinic shot every 3 months, Wainua a monthly self-injection, and the 2 stabilizers are daily pills. The nerve-form trials measured a neuropathy score and the heart-form trials measured deaths and hospitalizations, so results across forms cannot be compared.

Side by side

5 approved ATTR amyloidosis treatments at a glance

How each drug is given, how often, who it is approved for and when it was first approved, from the current FDA label.

ATTR Amyloidosis (Transthyretin Amyloidosis) treatments compared: route, schedule, approved use and approval date
Drug	How it is given	How often	Approved for	First approved
Onpattro patisiran Alnylam Pharmaceuticals	IV infusion at a clinic every 3 weeks, with premedicationAbout 80 minutes per dose	0.3 mg/kg (30 mg if 100 kg or more) by IV infusion once every 3 weeks; steroid, acetaminophen and antihistamines given 60 minutes before each dose	Adults with polyneuropathy (nerve damage) from hereditary ATTR amyloidosis	August 10, 2018
Amvuttra vutrisiran Alnylam Pharmaceuticals	Under-the-skin shot by a healthcare professional every 3 months	25 mg subcutaneous injection once every 3 months	Adults with polyneuropathy from hereditary ATTR amyloidosis, and adults with cardiomyopathy from wild-type or hereditary ATTR amyloidosis	June 13, 2022
Vyndaqel / Vyndamax tafamidis meglumine / tafamidis Pfizer	1 capsule by mouth once a day (Vyndamax) or 4 capsules once a day (Vyndaqel)	Vyndamax 61 mg once daily, or Vyndaqel 80 mg (four 20 mg capsules) once daily	Adults with cardiomyopathy from wild-type or hereditary ATTR amyloidosis, to reduce cardiovascular death and hospitalization	May 3, 2019
Attruby acoramidis BridgeBio Pharma	2 tablets by mouth twice a day	712 mg (two 356 mg tablets) twice daily, with or without food	Adults with cardiomyopathy from wild-type or variant ATTR amyloidosis, to reduce cardiovascular death and hospitalization	November 22, 2024
Wainua eplontersen AstraZeneca (with Ionis Pharmaceuticals)	Under-the-skin shot once a month; autoinjector can be given at home	45 mg subcutaneous injection once monthly	Adults with polyneuropathy from hereditary ATTR amyloidosis	December 21, 2023

The calendar

A year on each ATTR amyloidosis treatment

Every dose in a year, drawn to the label schedule, so the difference between a daily pill and a twice-yearly infusion is the picture.

Onpattro

Infusion, at a clinic

17 doses a year

Amvuttra

Injection, at a clinic

4 doses a year

Vyndaqel / Vyndamax

By mouth, at home

365 doses a year

Attruby

By mouth, at home

730 doses a year

Wainua

Injection

12 doses a year

Counts follow the label schedule for a full year on the maintenance dose. Drugs given in cycles or adjusted to blood tests have no fixed count, so their schedule is written out instead.

The evidence

What the pivotal ATTR amyloidosis trials showed

The primary result from the trial each FDA approval rests on, as reported on the label.

Before you compare

How to read the ATTR amyloidosis trial results

1
The 2 forms use different endpoints
Nerve-form trials report the mNIS+7 neuropathy score, where a smaller rise, or a fall, means less worsening. Heart-form trials report deaths and cardiovascular events over 30 to 36 months. A number from one column cannot be compared with a number from the other.
2
Two nerve-form trials were open-label
HELIOS-A for Amvuttra and NEURO-TTRansform for Wainua compared their drug against the placebo group from the earlier APOLLO trial, while Onpattro's APOLLO trial had its own placebo arm.
3
Vyndaqel and Vyndamax are one drug
Vyndaqel and Vyndamax are the same molecule, tafamidis, in 2 capsule strengths that are not interchangeable milligram for milligram. All 3 silencers lower vitamin A and the labels advise taking a supplement.

ATTR Amyloidosis (Transthyretin Amyloidosis) pivotal trial results by drug
Drug	Pivotal trial	Primary result
Onpattro patisiran Alnylam Pharmaceuticals	APOLLO (NCT01960348), 225 adults Trial record or paper	mNIS+7 nerve score improved 6.0 points on Onpattro vs worsened 28.0 points on placebo at 18 months (34-point difference)
Amvuttra vutrisiran Alnylam Pharmaceuticals	HELIOS-A (NCT03759379), 164 adults with polyneuropathy; HELIOS-B (NCT04153149), 654 adults with cardiomyopathy Trial record or paper	Nerve form: mNIS+7 improved 2.2 points vs worsened 14.8 points on external placebo at 9 months (17-point difference). Heart form: 28% lower risk of death or cardiovascular events vs placebo over up to 36 months (HR 0.72)
Vyndaqel / Vyndamax tafamidis meglumine / tafamidis Pfizer	ATTR-ACT (NCT01994889), 441 adults Trial record or paper	70.5% alive at 30 months vs 57.1% on placebo; 30% lower risk of death (HR 0.70) and 32% fewer cardiovascular hospitalizations
Attruby acoramidis BridgeBio Pharma	ATTRibute-CM (NCT03860935), 611 adults Trial record or paper	Fewer deaths (19% vs 26%) and fewer people with cardiovascular hospitalizations (27% vs 43%) than placebo over 30 months; 35.5% lower risk of death or first cardiovascular hospitalization (HR 0.645)
Wainua eplontersen AstraZeneca (with Ionis Pharmaceuticals)	NEURO-TTRansform (NCT04136184), 168 adults Trial record or paper	mNIS+7 nerve score held steady (+0.2 points) on Wainua vs worsened 9.2 points on external placebo at week 35 (9-point difference)

Safety

ATTR Amyloidosis (Transthyretin Amyloidosis) treatment side effects and warnings

None of these drugs carries a boxed warning. Each label's main precautions are listed.

ATTR Amyloidosis (Transthyretin Amyloidosis) treatments: most common side effects and key warnings from the FDA labels
Drug	Most common side effects	Key warning
Onpattro patisiran Alnylam Pharmaceuticals	upper respiratory tract infections 29%, infusion-related reactions 19% and indigestion 8%	No boxed warning. Infusion-related reactions (19% vs 9% on placebo); lowers vitamin A, so daily vitamin A supplement is advised.
Amvuttra vutrisiran Alnylam Pharmaceuticals	pain in arms or legs 15%, joint pain 11% and shortness of breath 7%	No boxed warning. Lowers vitamin A, so daily vitamin A supplement is advised; see an eye doctor for night blindness.
Vyndaqel / Vyndamax tafamidis meglumine / tafamidis Pfizer	label reports no side effect more common than placebo and 7% stopped for side effects vs 6% on placebo	No boxed warning and no Warnings and Precautions section on the label.
Attruby acoramidis BridgeBio Pharma	diarrhea 11.6% (vs 7.6% placebo), upper abdominal pain 5.5% (vs 1.4% placebo) and rise in creatinine (kidney blood test) in first 4 weeks, reversible	No boxed warning and no Warnings and Precautions section on the label. Causes a small, stable rise in creatinine and drop in eGFR that reverses if stopped.
Wainua eplontersen AstraZeneca (with Ionis Pharmaceuticals)	low vitamin A 15%, vomiting 9% and protein in urine 8%	No boxed warning. Lowers vitamin A, so daily vitamin A supplement is advised; see an eye doctor for night blindness or dry eyes.

Not yet approved

ATTR amyloidosis treatments in Phase 3 or under FDA review

Investigational drugs with a Phase 3 trial, shown separately because their results are not from a label. Status as of the date in each row.

ATTR Amyloidosis (Transthyretin Amyloidosis) drugs in development
Drug	Status	How it works and is given	Pivotal trial	Result so far	What is next
nexiguran ziclumeran (nex-z, NTLA-2001) Intellia Therapeutics / Regeneron	Phase 3as of October 1, 2026	One-time CRISPR gene editing that switches off the TTR gene in the liverSingle IV infusion, given once	MAGNITUDE (cardiomyopathy), Phase 3, about 1,200 people; MAGNITUDE-2 (polyneuropathy), Phase 3, about 60 peoplePrimary endpoint: MAGNITUDE: composite of cardiovascular death and cardiovascular events (18 months to about 5 years). MAGNITUDE-2: mNIS+7 at 18 months and serum TTR at day 29 ClinicalTrials.gov	No trial results published yet	No date announced Intellia press release, March 2, 2026

Cost

Help paying for ATTR amyloidosis treatment

Drugmaker copay and free-drug programs, plus charity funds for the condition, checked every week.

Questions

ATTR Amyloidosis (Transthyretin Amyloidosis) treatment questions people ask

Which ATTR amyloidosis drugs treat the heart?

Three. Vyndaqel/Vyndamax (tafamidis) and Attruby (acoramidis) are daily pills approved for ATTR cardiomyopathy, and Amvuttra (vutrisiran) was approved for the heart form in 2025 on top of its nerve-form approval. In their trials, tafamidis cut the risk of death by 30% and Attruby lowered deaths from 26% to 19% and cardiovascular hospitalizations from 43% to 27% against placebo over 30 months.

Which ATTR amyloidosis drugs treat the nerves?

Onpattro (patisiran), Amvuttra (vutrisiran) and Wainua (eplontersen), all approved for the polyneuropathy of hereditary ATTR. All 3 lower production of the TTR protein. In their trials the mNIS+7 nerve score improved or held steady on the drug while it worsened by 9 to 28 points on placebo.

Is Amvuttra better than Onpattro?

They are both Alnylam TTR silencers, and in HELIOS-A Amvuttra's effect on the nerve score was measured against the placebo group from Onpattro's earlier APOLLO trial, with Onpattro included only as a reference arm, so there is no head-to-head result. The practical difference is delivery: Onpattro is an IV infusion every 3 weeks with premedication, Amvuttra is a shot under the skin every 3 months. Amvuttra is also approved for the heart form; Onpattro is not.

Can Wainua be taken at home?

Yes. Wainua is a 45 mg injection under the skin once a month from an autoinjector that patients or caregivers can give at home. It is the only ATTR silencer that does not require a clinic visit for each dose.

What ATTR amyloidosis treatments are in development?

Nexiguran ziclumeran (nex-z), a one-time CRISPR gene-editing infusion from Intellia and Regeneron, is in 2 Phase 3 trials, MAGNITUDE for the heart form and MAGNITUDE-2 for the nerve form, both recruiting as of October 2026 after a clinical hold for liver enzyme rises was lifted in early 2026. No Phase 3 results have been published yet.

Go deeper

More on ATTR Amyloidosis (Transthyretin Amyloidosis) at Trial Friend

Sources

Where every number on this page comes from

Approved-drug facts are from the current FDA prescribing information on DailyMed, with the label's effective date. Investigational-drug facts are from ClinicalTrials.gov and the sources named in each row.

Onpattro (patisiran) prescribing information, Alnylam Pharmaceuticals, label dated Sep 2025. DailyMed. Pivotal trial: ClinicalTrials.gov
Amvuttra (vutrisiran) prescribing information, Alnylam Pharmaceuticals, label dated Nov 2025. DailyMed. Pivotal trial: ClinicalTrials.gov
Vyndaqel / Vyndamax (tafamidis meglumine / tafamidis) prescribing information, Pfizer, label dated May 2026. DailyMed. Pivotal trial: ClinicalTrials.gov
Attruby (acoramidis) prescribing information, BridgeBio Pharma, label dated Jun 2026. DailyMed. Pivotal trial: ClinicalTrials.gov
Wainua (eplontersen) prescribing information, AstraZeneca (with Ionis Pharmaceuticals), label dated Apr 2026. DailyMed. Pivotal trial: ClinicalTrials.gov
nexiguran ziclumeran (nex-z, NTLA-2001), Intellia Therapeutics / Regeneron: ClinicalTrials.gov record; Intellia press release, March 2, 2026. Checked October 1, 2026.

This page compares what the FDA labels and pivotal trials report. It is not medical advice, and no trial on this page compared these drugs with each other. Written and checked by Jason Hunter; the method is on the editorial standards page.