What is the New Drug for Amyloidosis?

October 7, 2025 •

4 min read

An estimated 4,000 people develop primary amyloidosis in the U.S. each year, a rare disease that has seen a recent surge in treatment options [1.10.3]. So, what is the new drug for amyloidosis? The answer depends on the type, with several groundbreaking therapies approved in late 2023 and 2024 [1.3.1, 1.4.1].

Quick Summary

Recent advancements in pharmacology offer new hope for amyloidosis patients. This overview covers the latest FDA-approved drugs for both ATTR and AL amyloidosis, their mechanisms, and what they mean for managing this once-fatal disease.

Key Points

New ATTR-CM Drugs: The FDA recently approved acoramidis (November 2024) and expanded vutrisiran's use (March 2025) for ATTR amyloidosis with cardiomyopathy, a major cause of heart failure in patients [1.2.2, 1.4.1].
New hATTR-PN Drug: Eplontersen (Wainua) was approved in December 2023 as a self-administered monthly injection for the polyneuropathy of hereditary ATTR amyloidosis [1.3.1, 1.3.4].
Two Main Mechanisms: New ATTR drugs work either by 'silencing' the gene that produces the TTR protein (e.g., Wainua, Amvuttra) or by 'stabilizing' the protein to prevent misfolding (e.g., Acoramidis) [1.2.1, 1.3.2].
AL Amyloidosis Advance: The standard of care for newly diagnosed AL amyloidosis now includes daratumumab, an antibody that targets the underlying plasma cells causing the disease [1.7.1, 1.7.4].
Improved Patient Outcomes: These new therapies significantly slow disease progression, reduce hospitalizations, and improve quality of life, transforming amyloidosis into a manageable condition [1.2.1, 1.3.4].
Future Research: The next wave of treatments includes therapies to actively remove amyloid deposits from organs and gene-editing technologies to permanently reduce TTR protein production [1.2.1, 1.2.4].

Amyloidosis is a group of rare, serious diseases caused by the buildup of abnormal proteins, called amyloid, in organs and tissues [1.10.3]. This protein deposition can disrupt the normal function of the heart, kidneys, nerves, and other organs [1.8.2]. For years, treatment was limited, but the therapeutic landscape has been revolutionized. Recent FDA approvals have introduced targeted therapies that are turning what was often a rapidly fatal diagnosis into a manageable, chronic condition [1.2.1]. The two most common forms are light-chain (AL) amyloidosis and transthyretin (ATTR) amyloidosis [1.10.1].

The New Wave of TTR Gene Silencers for ATTR Amyloidosis

Transthyretin (ATTR) amyloidosis occurs when a protein called transthyretin (TTR), produced by the liver, becomes unstable and misfolds [1.8.2]. A new class of drugs, known as TTR gene silencers, works by reducing the body's production of this protein. These include small interfering RNA (siRNA) and antisense oligonucleotide (ASO) therapies [1.5.1, 1.3.2].

Eplontersen (Wainua) Approved by the FDA in December 2023, Wainua (eplontersen) is an antisense oligonucleotide (ASO) therapy for adults with the polyneuropathy of hereditary ATTR amyloidosis (hATTR-PN) [1.3.1]. It is designed to reduce the production of the TTR protein at its source [1.3.4]. Administered as a once-monthly subcutaneous self-injection, it has been shown to halt neuropathy disease progression and improve quality of life [1.3.1, 1.3.4]. A common side effect is a decrease in Vitamin A levels, requiring supplementation [1.3.3].

Vutrisiran (Amvuttra) Approved for hATTR-PN in 2022 and later for ATTR with cardiomyopathy (ATTR-CM) in March 2025, vutrisiran is an RNA interference (RNAi) therapeutic [1.2.2, 1.5.2]. It uses a double-stranded siRNA to cause the degradation of TTR mRNA, which reduces serum TTR protein levels [1.5.3]. It is administered via subcutaneous injection once every three months [1.5.4]. Clinical trials demonstrated that vutrisiran significantly reduces the risk of all-cause mortality and recurrent cardiovascular events in patients with ATTR-CM [1.2.1].

Breakthrough TTR Stabilizers for Heart-Related Amyloidosis (ATTR-CM)

Another major therapeutic strategy is to stabilize the TTR protein, preventing it from misfolding and forming amyloid deposits in the first place. This approach has proven highly effective for patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM), a condition that stiffens the heart muscle [1.4.1].

Acoramidis (Attruby) The FDA approved acoramidis in November 2024 for adults with cardiomyopathy of both wild-type and hereditary ATTR amyloidosis [1.4.1]. As a potent TTR stabilizer, it binds to the TTR protein to prevent its dissociation [1.9.2]. In its pivotal clinical trial, acoramidis demonstrated a significant reduction in all-cause mortality and cardiovascular-related hospitalizations compared to placebo [1.4.1, 1.4.3]. It is an oral therapy taken twice daily [1.4.1].

Comparison of New Amyloidosis Drugs

The expansion of treatment options gives physicians and patients more choices tailored to the specific type of amyloidosis and patient lifestyle.


Drug (Brand/Generic)	Type of Amyloidosis	Mechanism	Administration	Key Benefit
Acoramidis (Attruby)	ATTR-CM (wild-type & hereditary) [1.4.1]	TTR Protein Stabilizer [1.4.1]	Oral, twice daily [1.4.1]	Reduces death and cardiovascular hospitalizations [1.4.1].
Eplontersen (Wainua)	hATTR-PN (hereditary with polyneuropathy) [1.3.1]	TTR Gene Silencer (ASO) [1.3.2]	Subcutaneous injection, monthly [1.3.1]	Halts neuropathy progression; self-administered [1.3.4].
Vutrisiran (Amvuttra)	hATTR-PN & ATTR-CM [1.5.3]	TTR Gene Silencer (RNAi) [1.5.3]	Subcutaneous injection, every 3 months [1.5.3]	Reduces death and cardiovascular events in ATTR-CM [1.2.1].
Tafamidis (Vyndaqel/Vyndamax)	ATTR-CM (wild-type & hereditary) [1.2.5]	TTR Protein Stabilizer [1.2.5]	Oral, once daily	First approved stabilizer, reducing mortality and disease progression [1.2.5].

Advancements in AL Amyloidosis Treatment

AL amyloidosis is caused by abnormal plasma cells in the bone marrow that produce misfolded light chains [1.7.1]. Treatment, therefore, focuses on targeting this underlying plasma cell clone, similar to treatments for multiple myeloma [1.7.3].

Daratumumab (Darzalex Faspro) In 2021, the FDA approved the first-ever treatment for newly diagnosed AL amyloidosis: a combination of daratumumab with cyclophosphamide, bortezomib, and dexamethasone (D-VCD) [1.7.1]. Daratumumab is a monoclonal antibody that targets CD38, a protein on the surface of plasma cells, causing their death [1.7.1]. The addition of daratumumab to the standard care regimen resulted in significantly higher rates of hematologic complete response and organ response (both heart and kidney) compared to the standard regimen alone [1.7.4].

The Future of Amyloidosis Treatment

The therapeutic landscape for amyloidosis is evolving rapidly. Future research is focused on several promising areas:

Combination Therapies: Studies are exploring the potential benefits of using a TTR silencer and a TTR stabilizer together to provide complementary effects [1.2.1].
Amyloid Depleters: A new class of drugs is in development that aims to actively remove existing amyloid deposits from organs, which could potentially reverse disease progression [1.2.1, 1.8.2].
Gene Editing: CRISPR-Cas9-based therapies, such as ziclumeran and NTLA-2001, are being investigated in clinical trials to permanently reduce TTR protein production [1.2.4, 1.2.2].

Conclusion

The question 'What is the new drug for amyloidosis?' now has multiple answers. The approvals of eplontersen (Wainua), acoramidis (Attruby), and the expanded indication for vutrisiran (Amvuttra) have provided powerful, targeted options for patients with different forms of ATTR amyloidosis. For AL amyloidosis, daratumumab-based regimens have set a new standard of care. These breakthroughs have fundamentally changed the prognosis for patients, offering not just a delay in progression but a significant improvement in quality of life and survival. With ongoing research into combination therapies and amyloid removal, the future for managing amyloidosis looks brighter than ever.

For more information and patient support, one authoritative resource is the Amyloidosis Research Consortium [1.4.4].

Frequently Asked Questions

As of 2025, two very new options are available. Acoramidis (Attruby) was approved in November 2024, and Vutrisiran (Amvuttra) was approved for ATTR-CM in March 2025. Both are for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM) [1.2.2, 1.4.1].

Currently, there is no cure for amyloidosis. However, new treatments can effectively slow or halt the progression of the disease, manage symptoms, and significantly improve survival and quality of life, turning it into a manageable chronic condition [1.2.1, 1.2.3].

Wainua (eplontersen) is a ligand-conjugated antisense oligonucleotide (LICA) medicine. It works by reducing the liver's production of the transthyretin (TTR) protein, which is the source of the amyloid deposits in ATTR amyloidosis [1.3.2, 1.3.4].

No, the new drugs for ATTR amyloidosis, like acoramidis, eplontersen, and vutrisiran, are not chemotherapy. They are targeted therapies, working as either 'gene silencers' or 'protein stabilizers' [1.2.1]. For AL amyloidosis, treatment often involves chemotherapy agents in combination with targeted antibody therapies like daratumumab [1.7.4].

A TTR gene silencer, like eplontersen or vutrisiran, works upstream to reduce the liver's production of the TTR protein [1.3.2, 1.5.3]. A protein stabilizer, like acoramidis or tafamidis, works on the TTR protein already in circulation, binding to it to prevent it from misfolding and forming amyloid deposits [1.2.5, 1.4.1].

AL amyloidosis is caused by abnormal plasma cells in the bone marrow. Treatment targets these cells, often using a combination of drugs including the antibody therapy daratumumab [1.7.4]. ATTR amyloidosis is caused by a misfolding liver protein, so treatments target that specific TTR protein [1.8.2].

The primary goal and demonstrated effect of current approved therapies (stabilizers and silencers) is to slow or halt disease progression [1.2.1]. They do not reverse existing significant organ damage. However, a future class of drugs called 'amyloid depleters,' currently in clinical trials, is being investigated for its potential to clear existing amyloid deposits [1.2.1].