Patients with transthyretin amyloid cardiomyopathy (ATTR-CM) experience a lower rate of gastrointestinal complications following the use of vutrisiran (marketed as Amvuttra), according to a press release recently published by Alnylam Pharmaceuticals.
The findings came from their phase 3 study assessing the use of vutrisiran in the treatment of patients with ATTR-CM and those with transthyretin amyloid polyneuropathy (ATTR-PN), two conditions in which symptoms are driven by the abnormal accumulation of misfolded transthyretin in the body. Vutrisiran is designed to silence the transthyretin gene so that no transthyretin protein can be produced.
What is transthyretin?
Transthyretin (TTR) is a transport protein produced in the liver. Its role is to carry vitamin A (retinol) and thyroxine, a thyroid hormone, around the body. In ATTR-CM, it is faulty or unstable, causing it to misfold and become deposited in the heart, nerves and other internal organs.
This phase 3 study showed that the use of vutrisiran was associated with a lower rate of adverse gastrointestinal events across several patient groups. Those receiving vutrisiran alone had a 37% lower rate of gastrointestinal events; those who took tafamidis at baseline had a had a 49% lower rate; and the overall patient population had a 42% lower rate.
ATTR-CM is a systemic disease that can affect various parts of the body; it is common for patients to experience symptoms that involve more than just the heart. When the gastrointestinal system is involved, patients may have pain, discomfort, nausea, vomiting and unexplained bowel movement habits. If these symptoms cause significant discomfort, patients can find their quality of life being reduced.
Read more about ATTR-CM testing and diagnosis
“We understand that gastrointestinal symptoms place a significant burden on patients, thus I’m encouraged to observe a reduction in these events in as early as three months after initiation of treatment,” said Dr. John Vest, senior vice president and TTR global clinical lead at Alnylam.
Further stages of the study are planned for researchers to delve more deeply into assessing the potential role of vutrisiran in offering clinically significant protection across both the cardiovascular and gastrointestinal systems.
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