ATTRv V30M Medications: Pills, Shots, or IV Infusions?

For patients with ATTRv V30M amyloidosis, treatments are available as daily pills, subcutaneous injections (shots under the skin), or intravenous (IV) infusions ^[1][2]. The exact method and frequency of your treatment will depend on which medication you and your doctor choose ^[3].

Currently, there are two main classes of FDA-approved therapies for ATTRv amyloidosis: TTR stabilizers and TTR silencers ^[4]. Knowing how these medications are given—and how much time they require—can help you plan your life, work schedule, and travel around your treatment.

TTR Stabilizers

TTR stabilizers work by binding to the transthyretin (TTR) protein, preventing it from breaking apart and forming the harmful amyloid deposits that cause symptoms.

• Tafamidis (Vyndaqel/Vyndamax): This medication is taken as an oral pill ^[5]. It is swallowed once daily ^[6]. Because it is a pill you take at home, it generally causes the least disruption to daily life compared to treatments that require clinical visits or injections ^[7]. Monitoring mostly involves routine clinical follow-ups.

TTR Silencers

TTR silencers (also known as gene silencers) work by targeting the genetic instructions that create the TTR protein, significantly reducing the amount of TTR your liver produces ^[3]. These medications are administered either through injections or IV infusions.

Subcutaneous Injections (Shots)

Subcutaneous injections are delivered into the fatty tissue just under the skin, usually in the stomach, thigh, or back of the upper arm.

• Eplontersen (Wainua): Approved in late 2023, this medication is given as a once-monthly subcutaneous injection ^[8]. It comes in a single-dose auto-injector pen, making it easier for patients to administer at home ^[8]. Storage note: It is kept in the refrigerator but should sit at room temperature for about 30 minutes before use.
• Inotersen (Tegsedi): This medication is administered as a once-weekly subcutaneous injection using a prefilled syringe ^[1]. Patients are usually trained to self-administer these shots at home ^[7]. Note that this medication requires strict, regular blood and urine testing to monitor your platelet counts and kidney function ^[9].
• Vutrisiran (Amvuttra): This medication is administered once every 3 months ^[10]. Because of the longer time between doses, it must be given by a healthcare professional at a clinic or via a home health nurse ^[2].

Intravenous (IV) Infusions

IV infusions deliver medication directly into a vein.

• Patisiran (Onpattro): This treatment is administered as an intravenous (IV) infusion ^[11]. It is given once every 3 weeks ^[12]. You will need to receive it at an infusion center, a clinic, or via home health services. The infusion itself takes about 80 minutes ^[13]. You must also take premedications (such as antihistamines and corticosteroids) at least 60 minutes before the infusion starts to prevent allergic reactions ^[14]. Because these premedications can cause drowsiness, you may need someone else to drive you home.

Treatment Comparison Table

Choosing the Right Option for Your Lifestyle

Each treatment method requires different levels of commitment. Daily pills and self-injections offer the convenience of staying at home but require you to remember your medication regularly. Infusions and three-month injections require visits with a healthcare professional but leave you free from daily or weekly treatment routines.

When discussing these options with your doctor, you should weigh how the administration route fits into your daily routine, travel plans, and comfort level with needles, medical visits, and required safety monitoring.