Inotersen: An Exciting Prospect for hATTR Amyloidosis Treatment


A new hereditary ATTR (hATTR) amyloidosis treatment, inotersen, an investigational antisense drug, may not be far from a US Food and Drug Administration (FDA) approval. Positive phase 3 data released this past March displayed an improvement in quality of life by 50% in patients with the disease, and with no effective treatment available, a lot hangs on the line.
 
In an effort to learn more about the prospective treatment, Rare Disease Report® (RDR®) spoke with Morie A. Gertz, MD, oncologist, internist, hematologist, and chair of Internal Medicine, Minnesota, at Mayo Clinic, to discuss how the impact the treatment could have on the hereditary hATTR patient population and how close it may be to receiving an approval from the FDA.
 
On July 11, 2018, inotersen was approved in the European Union for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
 
Inotersen: An Exciting Prospect For HATTR Amyloidosis Treatment