AstraZeneca PLC (LSE:AZN) and Ionis Pharmaceuticals' (NASDAQ:IONS) drug Wainzua (eplontersen) has been recommended for approval in the European Union for treating hereditary transthyretin-mediated amyloidosis, a rare disease that damages nerves and leads to disability.
The green light is based on a successful phase III trial, showing the drug improved patients' nerve function and quality of life compared to a placebo.
Wainzua would be the first treatment in the EU for this condition that can be self-administered monthly using an auto-injector.
If approved, it will offer patients a new option to manage this serious disease, which can be fatal within a decade without treatment.
The treatment was previously approved in the United States under the name Wainua and is now seeking approval in other countries.
AstraZeneca and Ionis are also studying its potential use for other related conditions.