In June 2022, the FDA approved Amvuttra, a prescription medicine designed to treat polyneuropathy (nerve damage) due to hereditary transthyretin amyloidosis (hATTR amyloidosis). This approval was huge. Typically, managing hATTR amyloidosis can be difficult, with many patients undergoing an organ transplant. While other drugs are available, these treatment options required patients to undergo intravenously administered treatment every three weeks.
Requiring treatment every three months, Amvuttra offers a more straightforward and less invasive therapeutic option. According to an article published by News Medical, UC San Diego Health is now the first place in the United States offering Amvuttra to patients. In fact, UC San Diego Health started offering Amvuttra in July, just one month after the therapy’s approval!
Studies showed that Amvuttra improved both nerve function and quality of life. UC San Diego Health is excited to be able to offer this to patients and to create a more impactful treatment experience. Patients such as Nancy Kim look forward to the freedom this will bring to their lives. Nancy, who has hATTR amyloidosis and who has had six family members pass away due to amyloidosis complications, has already seen her symptoms lessen since beginning treatment.
About Hereditary ATTR (hATTR) Amyloidosis
Amyloidosis is a group of diseases in which abnormal proteins (amyloids) accumulate and form deposits in organs. As the amyloids build up, the organs stiffen, causing a loss of function. Amyloidosis may be primary (AL), secondary (AA), dialysis-related (DRA), senile systemic (SSA / wild-type), organ-specific, or familial (ATTR & non-ATTR).
TTR gene mutations cause hATTR amyloidosis. These mutations cause a protein called transthyretin (TTR) to misfold. As a result, TTR begins forming deposits in nerves and organs. In many cases, hATTR amyloidosis affects the heart, nervous system, kidneys, and digestive system. Symptoms vary based on affected organ, and can (but do not always) include:
- Nausea and vomiting
- Unintended weight loss
- Peripheral and autonomic neuropathy
- Shortness of breath
- Worsening fatigue
- Sensorimotor impairment
- Chest pain
- Atrial fibrillation
- Swelling of the legs, abdomen, and arms
- Diarrhea or constipation
- Appetite loss
- Bladder control difficulties