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.
Tag Archive for: Ionis
In the next two weeks, the agency will release regulatory decisions on investigational treatments for a rare hereditary disorder, non-small cell lung cancer and more.
The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 9-0 that tofersen’s effect on neurofilament light chain could be a reasonable predictor of clinical benefit.
The FDA’s Peripheral and Central Nervous System Drugs advisory committee will discuss the merits of Biogen and Ionis’s ALS candidate tofersen on March 22nd.
AstraZeneca has decided to drop the hypercholesterolemia candidate AZD8233 through to Phase III, despite meeting its Phase IIb primary efficacy endpoint, its partner Ionis announced Friday.
