Approved Treatments

Amvuttra (vutrisiran) is an under-the-skin, or subcutaneous, injectable therapy approved for adults with FAP. The therapy, administered once every three months, works to reduce TTR protein production, thereby preventing the formation of toxic clumps, easing symptoms of neuropathy, or nerve damage, and slowing disease progression.

Onpattro (patisiran), Amvutrra’s predecessor, is an approved treatment for adults with FAP. Administered via an into-the-vein, or intravenous, infusion every three weeks, the medication is designed to lower TTR levels, lessen symptoms, and slow their progression.

Wainua (eplontersen), Tegsedi’s successor, is a once-monthly subcutaneous, injectable therapy approved in the U.S. for adults with FAP. Designed to lower TTR levels, 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.