Tag: Rnai therapy

In September 2023, the FDA approved Rivfloza® (nedosiran), marking a significant milestone for patients with primary hyperoxaluria type 1 (PH1), a rare genetic disorder characterized by excessive oxalate production. As an innovative RNA interference (RNAi) therapy, Rivfloza works to reduce the burden of oxalate by targeting the source of its overproduction in the liver.

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.

According to the results of the Phase 3 HELIOS-A clinical trial, 48% of patients treated with AMVUTTRA experienced a reversal of neuropathy impairment from their baseline after 18 months, a significant improvement compared to the placebo group. For many suffering from this progressive and debilitating condition, the key question remains: Does AMVUTTRA really work?

The annual list price for ONPATTRO (patisiran) is approximately $450,000, but the net price after discounts can be closer to $345,000 [1.2.2, 1.2.3]. This article explores the details of **how much does ONPATTRO cost per year**, including factors that influence its price and available financial support.

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.

In 2022, the FDA approved Amvuttra (vutrisiran) as a major therapeutic advance for specific types of amyloidosis. Often mistakenly searched for as 'ambutra,' this medication represents a powerful form of gene-silencing therapy that targets the root cause of hereditary transthyretin-mediated (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.