Approved Treatments

Amvuttra (vutrisiran) is an approved treatment for FAP and other types of ATTR amyloidosis. Amvuttra targets faulty mRNA, the intermediate messenger molecule between the TTR gene and transthyretin protein. It eases the symptoms of neuropathy, or those resulting from the nerve damage that marks the disease.

Onpattro (patisiran) is an approved treatment for FAP that uses a technology researchers call RNA interference, or RNAi. Onpattro consists of lipid nanoparticles that contain siRNA. The nanoparticles deliver the siRNA to liver cells, where they significantly lower the expression of the faulty TTR gene.

Tegsedi (inotersen) is approved to treat adults with FAP. It is a once-weekly injection administered under the skin, or subcutaneously. Tegsedi is designed to interfere with the production of TTR and prevent the buildup of amyloid deposits.

Wainua (eplontersen) is an under-the-skin, or subcutaneous, injectable therapy approved in the U.S. for adults with FAP. Designed to prevent nerve damage and thereby slow or halt disease progression, it is the first and only approved FAP therapy that can be self-administered by patients through an autoinjector.

Non-pharmacological strategies that can be used in FAP patients include liver transplant, which can slow disease progression, and physiotherapy and occupational therapy, which can improve patients’ mobility, strength, and ability to perform daily activities.