ATTR Amyloidosis Treatment in 2026, 5 Approved Drugs Compared on Survival, Dosing and Side Effects

Transthyretin is a protein the liver makes to ferry thyroid hormone and vitamin A around the body. When it misfolds and piles up in the heart or nerves, the result is transthyretin amyloidosis, or ATTR. There are families in which a person inherits a disease-causing version of the gene and rarely develops the disease. They carry a second change in the same gene, called T119M, which makes the protein unusually stable. That accident of biology is the whole idea behind acoramidis, a pill designed to make everyone's transthyretin behave like theirs. It is one of 2 ways medicine has learned to fight this disease. The other is blunter: stop the liver from making the protein at all.

Both approaches now have FDA-approved drugs behind them, 6 since 2018, and the choice between them is the central decision for anyone diagnosed with ATTR amyloidosis today. It is also a decision most patients are asked to make with numbers from 7 different trials that were never meant to be compared. This guide puts those numbers side by side, states plainly where a comparison is fair and where it is not, and draws the parts that are easier to see than to read.

ATTR Amyloidosis Is 2 Diseases, and Whether Yours Is Heart or Nerve Decides Which Drugs You Can Get

Transthyretin, or TTR, is a protein the liver makes to carry thyroid hormone and vitamin A through the blood. It travels as a 4-part cluster, and it only causes trouble when the cluster falls apart. The loose pieces misfold, stick together, and settle into tissue as amyloid. Where they settle decides which disease you have. In the heart muscle they cause a stiff, thickened heart that cannot fill properly (ATTR cardiomyopathy, or ATTR-CM). In the nerves of the hands, feet and gut they cause numbness, pain, diarrhea and blood pressure that collapses on standing (ATTR polyneuropathy, or ATTR-PN).

A second split matters just as much. In hereditary ATTR (hATTR or ATTRv), a variant in the TTR gene makes the cluster unstable from birth, and the disease can hit the heart, the nerves or both, often in middle age. In wild-type ATTR (ATTRwt), the gene is normal and the protein simply becomes unstable with age; it mostly affects the heart, and mostly in men over 70. Trials reflect that: 76% of the people in tafamidis's heart trial had wild-type disease, and 88% in vutrisiran's did (Maurer et al., 2018; Fontana et al., 2025).

ATTR-CM, wild-type or hereditary

Approved: tafamidis (Vyndamax, Vyndaqel), acoramidis (Attruby), vutrisiran (Amvuttra).

hATTR-PN, hereditary with nerve damage

Approved: patisiran (Onpattro), vutrisiran (Amvuttra), eplontersen (Wainua). Tafamidis is not approved for the nerve form in the United States, though it was in Europe in 2011.

Both heart and nerves

Only vutrisiran carries both indications on its FDA label. Many people with hereditary disease have both problems, which is one reason the silencer drugs get used in them.

Wild-type with nerve symptoms

No drug is approved for this. Carpal tunnel syndrome and some neuropathy are common in wild-type disease, but the nerve-form approvals cover the hereditary disease only.

The diagnosis numbers are the striking part. In 2019 Pfizer put the US population with ATTR-CM at about 100,000 and said only 1% to 2% had been diagnosed. Alnylam's 2025 estimate is about 150,000. The disease hides inside ordinary heart failure in older men, and until a scan called a technetium pyrophosphate scan made it possible to find it without a biopsy, most people never learned what they had. A 2016 Mayo Clinic series of 360 wild-type patients, all diagnosed before any treatment existed, found a median survival of 3.6 years from diagnosis (Grogan et al., 2016).

The 2 silencer technologies differ in how they reach the liver. Patisiran and vutrisiran use RNA interference (RNAi); patisiran packs its RNA into a fatty nanoparticle that must be infused into a vein, while vutrisiran attaches a sugar called GalNAc that liver cells pull in on their own, which is why it works as a small injection under the skin every 3 months. Eplontersen is an antisense oligonucleotide, a single-stranded cousin of the same idea, also GalNAc-linked, injected monthly. Inotersen was an older antisense drug without the sugar; it needed weekly injections and weekly blood tests, and it is no longer sold.

8 Years of ATTR Drug Approvals in 1 Timeline, From Onpattro to the Wainua Heart Trial That Failed

Vyndamax, Attruby and Amvuttra Heart Trials Side by Side, and Why the Placebo Groups Kept Getting Healthier

The 3 drugs approved for ATTR-CM were each tested against placebo for 30 to 36 months, and each won. The temptation is to line up the results and crown a winner. Resist it, at least a little. The trials enrolled different people at different times, and the biggest difference is one the charts cannot show: what the placebo patients were allowed to take.

Look at the placebo columns. In ATTR-ACT, 42.9% of untreated patients died within 30 months, and 6 years later in ATTRibute-CM, 26% did. The disease had not become kinder. Patients were being found earlier, thanks to the scan, and in the acoramidis trial patients in either group could start tafamidis after 12 months: 22.8% of the placebo group did, and 14.9% of the acoramidis group (Attruby label). In HELIOS-B the shift went further: 40% of everyone enrolled was already taking tafamidis on day 1, and another 21% of the placebo group started it during the study (Fontana et al., 2025).

That is why the bars shrink from left to right in the walk and quality-of-life columns. Tafamidis was measured against nothing; vutrisiran was measured, for many patients, against tafamidis plus placebo. A smaller advantage over a treated group is not a smaller drug. The honest summary is that all 3 lowered the risk of death and hospital stays by roughly a quarter to a third in their own trials, and no trial has ever put 2 of them head to head.

Onpattro, Amvuttra and Wainua Nerve Trials Measured Numbness in Points, and Treatment Did More Than Slow It

Nerve damage from ATTR is scored on a scale called mNIS+7, where a higher number means worse function and a rising score means the disease is advancing. In the trials with 15 to 18 months of placebo data, the placebo group's score climbed by 25 to 28 points, while the treated group's barely moved or improved. The gap between them is the treatment effect.

That chart needs 2 cautions. The vutrisiran number is at 9 months, not 18, because that was the trial's primary timepoint; by 18 months its effect was described as significant on every measure and its TTR lowering matched patisiran's within the same study (Adams et al., 2023). Inotersen's smaller effect came with a much bigger price: in its trial there were 5 deaths on inotersen and none on placebo, 1 of them linked to a severe platelet drop, and 3 patients developed kidney inflammation (Benson et al., 2018). None of the 3 silencers still sold carries a boxed warning; their trials did not show inotersen's platelet or kidney problems.

A Year on Each ATTR Amyloidosis Treatment, Drawn to Scale

Efficacy tables hide the part of treatment that fills a calendar. Each square below is 1 dose. The rows are drawn at the same scale, so a year of acoramidis is a block and a year of vutrisiran is 4 dots. Neither is better because of that; a pill you take at your kitchen table and an injection a nurse gives 4 times a year are different kinds of burden, and people weigh them differently. The picture just makes the trade visible.

In the first quarter of 2026, Alnylam reported $890 million in global Amvuttra sales, up 187% from a year earlier, while global Onpattro sales fell 59% to $20 million (Alnylam Q1 2026 results). Alnylam credited Amvuttra's growth mainly to new heart-form patients in the United States and noted fewer patients on Onpattro.

ATTR Drug Side Effects, From Tafamidis, Which Looks Like Placebo, to Tegsedi, Now Discontinued

Tafamidis sits at one end. Its label has no warnings section at all, and in its 30-month trial side effects occurred at rates similar to placebo. Acoramidis is close behind: diarrhea in 11.6% of patients versus 7.6% on placebo, upper stomach pain in 5.5% versus 1.4%, and a small, expected rise in the kidney value creatinine in the first 4 weeks that levels off and reverses if the drug stops (Attruby label). About 9% of patients stopped acoramidis for a side effect, about the same as placebo.

The silencers share 2 issues worth knowing. First, vitamin A. Because TTR carries it, silencing TTR drops blood vitamin A in nearly everyone: 99% of patisiran patients with normal levels developed low ones, 95% on eplontersen and 80% on vutrisiran in its heart trial (Onpattro, Wainua and Amvuttra labels). Labels tell patients to take the recommended daily allowance, not more, and to see an eye doctor for night blindness or dry eyes. Second, each label reports a slow heartbeat from a conduction problem called AV block as a serious event in a small number of patients: 2.7% on patisiran (none on placebo), 2% on eplontersen and 1.6% on vutrisiran in their nerve trials, which had no concurrent placebo group. Heart rhythm problems are also part of ATTR itself, and the labels do not attribute AV block to how the drugs work. Patisiran alone adds infusion reactions, in 19% of patients versus 9% on placebo, mostly during the first 2 infusions and the reason for all that premedication.

How Long You Can Live With ATTR Amyloidosis Depends on the Stage and the Year You Were Diagnosed

This is the question people search for, and the honest answer has 2 halves. The first is that survival without treatment depends heavily on how far the disease has progressed at diagnosis, which doctors estimate with 2 blood tests. The UK National Amyloidosis Centre staged 869 patients using NT-proBNP (a heart strain marker) and kidney function, and the spread was wide (Gillmore et al., 2018).

The second half is that every one of those numbers predates the drugs. In ATTR-ACT, 70.5% of patients on tafamidis were alive at 30 months versus 57.1% on placebo, consistent with a drug that prevents new damage rather than undoing old damage (Maurer et al., 2018). The people diagnosed at stage I, who have the most heart left to protect, are the ones with the most to gain from starting early. That is the practical reason cardiologists now scan older men with unexplained heart failure, carpal tunnel surgery in their history, or a thick heart wall on an echo: stage I patients had the longest survival, and the treatment trials suggest benefit builds over time, so earlier diagnosis gives treatment more time to work.

Taking a TTR Stabilizer and a Silencer Together, and What HELIOS-B and CARDIO-TTRansform Found

Whether to combine them is the obvious question. The 2 classes attack different steps, so adding them should help. No FDA label specifically addresses combining them, though Amvuttra's heart approval came from a trial in which 40% of patients were also on tafamidis. Only 2 trials speak to the question, and neither settles it.

The cautious reading is that the benefit of adding a silencer to a stabilizer is uncertain: HELIOS-B suggested a smaller but consistent effect, and CARDIO-TTRansform found none for eplontersen, a different drug. Combination is unproven, means paying for 2 specialty drugs, and is something to raise with an amyloidosis specialist rather than assume. The Amyloidosis Research Consortium is a good starting point for finding an amyloidosis center.

What Is Next for ATTR Amyloidosis, From Nucresiran to the Nex-z Gene Edit

Open and recruiting ATTR studies, with their sites, are on the ATTR amyloidosis trials page and can be sorted by distance on the near-you page.

Paying for ATTR Amyloidosis Drugs, and the Medicare Detail That Depends on Which One You Take

Every one of the 5 drugs has a manufacturer support program, and as of September 24, 2026, 6 charity funds for amyloidosis were open: 2 at the HealthWell Foundation (one specifically for people on Medicare with cardiomyopathy), 1 at TotalAssist, 2 at NORD and 1 at The Assistance Fund. The patient assistance finder lists all of them with the date each was last checked, and filters them by your insurance.

Vyndamax and Vyndaqel (tafamidis)

Pfizer's VynAssist program, 1-888-863-1177: copay help for commercial insurance and free medicine through the Pfizer Patient Assistance Foundation for eligible patients, including some on Medicare. Details.

Attruby (acoramidis)

BridgeBio's ForgingBridges, 1-888-552-7434: commercial copay support, a bridge supply during coverage delays, and free drug for uninsured or underinsured patients. BridgeBio also provides Attruby free for life to US participants in its clinical trials. Details.

Amvuttra (vutrisiran) and Onpattro (patisiran)

Alnylam Assist, 1-833-256-2748: commercial copay program, help during coverage delays (a Quick Start dose at no cost for eligible new Amvuttra patients, and a Bridge Program for both drugs), and free drug for the uninsured. A case manager calls within 2 business days of the doctor's Start Form. Details.

Wainua (eplontersen)

AstraZeneca's WAINUA WAY program, 1-844-2-WAINUA (1-844-292-4682): AstraZeneca Access 360 help with coverage and costs, a copay program that lets commercially insured patients pay as little as $0 a month, and free medicine through AZ&Me for eligible patients, including some who are uninsured or on Medicare. The 1-800-236-9933 number on the label is AstraZeneca's general information line. The Wainua drug page has details.