Experimental Treatments

AKCEA-TTR-LRx

AKCEA-TTR-LRx (ION-682884) is an RNA-targeted therapy being developed for the treatment of adults with all forms of transthyretin amyloidosis, including FAP and wild-type. It is currently in Phase 3 trials.

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Dolobid

Dolobid (diflunisal) is a nonsteroidal anti-inflammatory medication that works by reducing hormones that cause inflammation and pain. It has been shown to inhibit the progression of neuropathy and preserve quality of life in people with FAP. It is currently used off-label for the treatment of FAP.

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Doxycycline and Taurodesoxycholic Acid

The combination of doxycycline and taurodesoxycholic acid (TUDCA) is being investigated as a potential treatment for FAP. Doxycycline is an antibiotic, and TUDCA is a bile acid. They are both amyloid matrix solvents that work by disrupting or dissolving the deposited amyloid fibrils. It was last tested for FAP patients in a Phase 2 trial.

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SOM0226

SOM0226, also known as tolcapone and CRX-1008, is a repositioned compound being evaluated for the treatment of FAP. A repositioned compound is one that already is approved to treat a different disease and thought to be of use in others. SOM0226 works by imitating the process by which thyroxine binds to TTR in the bloodstream. It is currently being studied in Phase 2 trials.

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Vutrisiran

Vutrisiran (previously ALN-TTRsc02) is an experimental treatment for FAP and other types of ATTR amyloidosis. Vutrisiran targets faulty mRNA, the intermediate messenger molecule between the TTR gene and transthyretin protein. Positive results from a Phase 3 trial were announced in April 2021.

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Vyndaqel

Vyndaqel (tafamidis) is under investigation as a treatment for FAP in the U.S. It received approval from the U.S. Food and Drug Administration for cardiomyopathy in adults with wild-type or hereditary TTR cardiac amyloidosis and is approved for the treatment of FAP in Europe. It is being tested in a long-term Phase 3 study in FAP patients.

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