What drugs are FDA approved for amyloidosis?

October 10, 2025 •

3 min read

Over the last decade, there has been a significant expansion in the number of FDA-approved drugs for amyloidosis, with distinct treatments now available for different types of the disease. The answer to What drugs are FDA approved for amyloidosis? depends on whether a patient has transthyretin (ATTR) or light chain (AL) amyloidosis, as the treatments for each type differ substantially.

Quick Summary

The U.S. Food and Drug Administration has approved several drugs for specific types of amyloidosis, including Darzalex Faspro for light chain (AL) and multiple options like Vyndaqel/Vyndamax, Attruby, Amvuttra, and Onpattro for transthyretin (ATTR) amyloidosis.

Key Points

AL Amyloidosis Treatment: Darzalex Faspro, in combination with other chemotherapy drugs (VCd), is FDA-approved for newly diagnosed light chain (AL) amyloidosis.
ATTR Stabilizers for Cardiomyopathy: Tafamidis (Vyndaqel/Vyndamax) and acoramidis (Attruby) are oral medications that stabilize the TTR protein for patients with ATTR cardiomyopathy (ATTR-CM).
TTR Gene Silencers: RNAi-based therapies like patisiran (Onpattro), vutrisiran (Amvuttra), and eplontersen (Wainua), along with the ASO inotersen (Tegsedi), are FDA-approved to reduce TTR protein production.
Dual ATTR Indication: Vutrisiran (Amvuttra) is notably approved for both hATTR polyneuropathy and ATTR cardiomyopathy.
Targeted Therapies: Approved treatments for amyloidosis work by either targeting the abnormal plasma cells in AL amyloidosis or by stabilizing/reducing the specific misfolded protein in ATTR amyloidosis.
Specialized Care: Given the complexity of amyloidosis, treatment decisions should be made in consultation with specialists at a dedicated amyloidosis center.

Amyloidosis is a rare disease characterized by the buildup of abnormal protein deposits called amyloid in organs and tissues. The two primary systemic forms are transthyretin (ATTR) amyloidosis and light chain (AL) amyloidosis. The FDA has approved targeted therapies that have significantly improved patient outcomes.

FDA-Approved Treatments for Transthyretin (ATTR) Amyloidosis

ATTR amyloidosis results from misfolded transthyretin (TTR) protein and can affect the heart (ATTR-CM) or nerves (hATTR-PN). Treatments either stabilize the TTR protein or reduce its production.

TTR Stabilizers for ATTR Cardiomyopathy (ATTR-CM)

These drugs bind to and stabilize the TTR protein.

Tafamidis (Vyndaqel, Vyndamax): An oral medication approved in 2019 for adults with ATTR-CM, showing reduced mortality and hospitalizations. Different formulations are not interchangeable.
Acoramidis (Attruby): Approved in November 2024, this oral stabilizer for ATTR-CM reduced cardiovascular events in trials.

TTR Gene Silencers for hATTR-PN and ATTR-CM

These therapies use RNAi or ASOs to reduce TTR protein production.

Patisiran (Onpattro): Approved in 2018, the first RNAi therapeutic for hATTR-PN, administered intravenously.
Inotersen (Tegsedi): An ASO approved in 2018 for hATTR-PN, given via subcutaneous injection. It is available through a restricted safety program due to risks of thrombocytopenia and glomerulonephritis.
Vutrisiran (Amvuttra): Approved in 2022 for hATTR-PN (subcutaneous injection) and in March 2025 for ATTR-CM.
Eplontersen (Wainua): An ASO approved for hATTR-PN, administered as a monthly subcutaneous injection.

FDA-Approved Treatment for Light Chain (AL) Amyloidosis

AL amyloidosis is caused by abnormal plasma cells producing misfolded light chains. Treatment targets these plasma cells.

Combination Therapy with Daratumumab (Darzalex Faspro)

Daratumumab + Hyaluronidase-fihj (Darzalex Faspro): Granted accelerated approval in 2021 as part of a combination regimen (D-VCd) for newly diagnosed adults with AL amyloidosis. This combination showed a higher hematologic complete response rate than VCd alone in the ANDROMEDA trial. Darzalex Faspro is a monoclonal antibody targeting CD38 on plasma cells.

Comparison of Key FDA-Approved Amyloidosis Medications


Drug Name	Primary Indication	Mechanism of Action	Administration	Key Approval Dates	Target Amyloid Type
Daratumumab (Darzalex Faspro)	Newly diagnosed AL amyloidosis	Anti-CD38 monoclonal antibody	Subcutaneous injection (combo)	2021	AL
Tafamidis (Vyndaqel/Vyndamax)	ATTR cardiomyopathy (hATTR & wATTR)	TTR tetramer stabilizer	Oral capsule (daily)	2019	ATTR
Acoramidis (Attruby)	ATTR cardiomyopathy (hATTR & wATTR)	TTR tetramer stabilizer	Oral tablet (twice daily)	Nov 2024	ATTR
Vutrisiran (Amvuttra)	hATTR polyneuropathy & ATTR cardiomyopathy	TTR gene silencer (RNAi)	Subcutaneous injection (quarterly)	2022 (PN), Mar 2025 (CM)	ATTR
Patisiran (Onpattro)	hATTR polyneuropathy	TTR gene silencer (RNAi)	Intravenous infusion (3-weekly)	2018	ATTR
Inotersen (Tegsedi)	hATTR polyneuropathy	TTR gene silencer (ASO)	Subcutaneous injection (weekly)	2018	ATTR

Future Directions in Amyloidosis Treatment

Research continues to explore new therapies, including advanced gene silencing, TTR stabilization, and anti-amyloid antibodies. For AL amyloidosis, emerging treatments like CAR T-cell therapy targeting BCMA show promise for refractory cases.

Conclusion

The FDA has approved various drugs for amyloidosis, tailored to the specific type. For newly diagnosed AL amyloidosis, daratumumab in combination therapy targets abnormal plasma cells. For ATTR amyloidosis, treatments include TTR stabilizers like tafamidis (Vyndaqel/Vyndamax) and acoramidis (Attruby) for cardiomyopathy, and TTR gene silencers such as patisiran (Onpattro), inotersen (Tegsedi), vutrisiran (Amvuttra), and eplontersen (Wainua) for polyneuropathy and cardiomyopathy. Consulting with an amyloidosis specialist is crucial for determining the best treatment plan.

Disclaimer: This information is for general knowledge and should not be taken as medical advice. Consult with a healthcare professional before making any decisions about your treatment or care.

Frequently Asked Questions

The primary FDA-approved medications for ATTR cardiomyopathy (ATTR-CM) are the TTR stabilizers, tafamidis (Vyndaqel/Vyndamax) and acoramidis (Attruby), as well as the gene silencer vutrisiran (Amvuttra).

As of March 2025, vutrisiran (Amvuttra) is one of the most recent FDA approvals for ATTR cardiomyopathy, following the approval of acoramidis (Attruby) in November 2024.

For hereditary ATTR polyneuropathy (hATTR-PN), the FDA has approved several gene-silencing therapies: patisiran (Onpattro), vutrisiran (Amvuttra), eplontersen (Wainua), and inotersen (Tegsedi).

Daratumumab, used for AL amyloidosis, is a monoclonal antibody that targets and destroys the plasma cells producing the abnormal light chains. Tafamidis, used for ATTR amyloidosis, stabilizes the TTR protein to prevent it from misfolding and forming amyloid deposits.

Yes, for ATTR cardiomyopathy, there are oral treatments like tafamidis (Vyndaqel/Vyndamax) and acoramidis (Attruby). For polyneuropathy, there are injectable options, but not specifically oral ones, though diflunisal is sometimes used off-label.

The approval of Darzalex Faspro, in combination therapy, marked the first and only FDA-approved treatment specifically for newly diagnosed patients with AL amyloidosis.

Yes, FDA-approved drugs for wild-type ATTR amyloidosis include the oral TTR stabilizers tafamidis (Vyndaqel/Vyndamax) and acoramidis (Attruby), as well as the injectable gene silencer vutrisiran (Amvuttra), all indicated for cardiomyopathy.

TTR gene silencers are typically administered by injection. Patisiran (Onpattro) is given via intravenous (IV) infusion, while vutrisiran (Amvuttra), inotersen (Tegsedi), and eplontersen (Wainua) are administered as subcutaneous injections.