Tag: Onpattro

According to prescribing information, Onpattro is administered via intravenous infusion once every three weeks by a healthcare professional. This consistent schedule is designed to manage the polyneuropathy symptoms associated with hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Hereditary transthyretin-mediated (hATTR) amyloidosis affects an estimated 50,000 people worldwide [1.5.3, 1.5.4]. This article explores what is the difference between Onpattro and AMVUTTRA, two RNA interference (RNAi) therapies used to treat its symptoms.

In 2018, ONPATTRO became the first FDA-approved medication using RNA interference technology, ushering in a new era of gene-based medicines. The groundbreaking therapy acts as a gene silencer, specifically targeting and reducing the production of a protein responsible for a rare, debilitating genetic disease known as hATTR amyloidosis.

As the first FDA-approved small interfering RNA (siRNA) therapy, Onpattro (patisiran) represents a significant advance in treating hereditary transthyretin-mediated (hATTR) amyloidosis. This groundbreaking medication uses a unique gene-silencing mechanism to address the root cause of the disease, rather than just managing its symptoms.