By Mariam Sunny and Puyaan Singh
(Reuters) – The U.S. Food and Drug Administration (FDA) on Thursday approved Ionis Pharmaceuticals and partner AstraZeneca’s drug to treat nerve damage caused by a life-shortening rare disease.
The drug, branded as Wainua, is approved for patients with polyneuropathy, or nerve damage caused by hereditary transthyretin amyloidosis (ATTR-PN), which affects an estimated 40,000 patients globally.
Transthyretin amyloidosis is a progressive condition characterized by the build-up of abnormal protein deposits in the body’s organs and tissues.
The disease causes damage to nerves outside the brain and the spinal cord and can be fatal within a decade if untreated, according to AstraZeneca.
Wainua is to be administered once a month at home via an under-the-skin injection. It uses a newer type of technology compared to an older Ionis drug, and is designed to target the liver, where the protein is produced.
Ionis CEO Brett Monia said the company estimates that only 20% of the patients suffering from ATTR-PN are on treatment.
“This is a market where more than one player can have great success,” he added.
William Blair analyst Myles Minter estimates global peak sales of about $750 million for the treatment of ATTR-PN alone.
The new drug can be self-administered and requires less dosing than previously approved therapies – Alnylam Pharmaceuticals’ therapies Amvuttra and Onpattro, as well as Ionis’ Tegsedi.
“We still expect Alnylam to retain leadership in this market, but with AstraZeneca as a global collaboration partner and a more convenient, self-administered product, we think Ionis has a good position,” Morningstar analyst Karen Andersen wrote in a note prior to the approval.
Ionis and AstraZeneca are also testing it to treat other forms of the disease that can lead to cardiomyopathy, a heart muscle disease.
Analysts expect total peak sales to be between $3.5 billion and $7 billion if the drug gets expanded approval to treat cardiomyopathy.
(Reporting by Puyaan Singh, Mariam Sunny, Bhanvi Satija and Sriparna Roy in Bengaluru; Editing by Tasim Zahid and Krishna Chandra Eluri)
