Tag: Attr amyloidosis

With more than 7,000 rare diseases known worldwide, affecting an estimated 300 million people, Pfizer's focus on what is Pfizer rare disease represents a significant commitment to an area of high unmet medical need. For over thirty years, the company has worked to provide critical treatment options for patients with rare conditions, advancing research across areas like rare hematology, neurology, and cardiology.

Over the last decade, there has been a significant expansion in the number of FDA-approved drugs for amyloidosis, with distinct treatments now available for different types of the disease. The answer to **What drugs are FDA approved for amyloidosis?** depends on whether a patient has transthyretin (ATTR) or light chain (AL) amyloidosis, as the treatments for each type differ substantially.

According to clinical trial data, AMVUTTRA begins decreasing transthyretin (TTR) protein levels just weeks after the first dose. However, it may take several months to see clinical improvements in symptoms, as the treatment gradually works to slow disease progression and can even reverse some symptoms over the long term.

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?

An estimated 4,000 people develop primary amyloidosis in the U.S. each year, a rare disease that has seen a recent surge in treatment options [1.10.3]. So, what is the new drug for amyloidosis? The answer depends on the type, with several groundbreaking therapies approved in late 2023 and 2024 [1.3.1, 1.4.1].

First approved in 2022 for hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy, **vutrisiran** (brand name Amvuttra) is a gene-silencing medication that offers a new approach to treatment. It works by targeting the root genetic cause of the disease, preventing the production of the toxic protein that causes nerve and heart damage.