- AstraZeneca
- 21 October 2024 07:26:25
Source: Sharecast
Wainzua, jointly developed with California biotech firm Ionis, has already been approved in the US for use in the treatment of adults with polyneuropathy – disease of the peripheral nerves – associated with hereditary transthyretin-mediated amyloidosis, commonly referred to as ATTRv-PN.
ATTRv-PN is a debilitating diseases that leads to peripheral nerve damage with motor disability within five years of diagnosis. Without treatment, it is generally fatal within a decade.
If approved by the European Commission, Wainzua, otherwise known as eplontersen, will be the only approved medicine in the EU for the condition that can be self-administered on a monthly basis.
Monday's recommendation from the Committee of Medicinal Products for Human Use came after a phase III trial showed Wainzua demonstrated consistent and sustained benefit improving neuropathy impairment and quality of life when compared with a placebo.
"Due to the progressive nature of polyneuropathy of hereditary transthyretin-mediated amyloidosis, it is critical to have timely diagnosis and new therapies to help people have greater control over this potentially fatal disease," said Ruud Dobber, executive vice president of Astra's BioPharmaceuticals Business unit.
"Today's recommendation brings Wainzua one step closer for patients in Europe, and if approved, will offer a new treatment option that can provide consistent TTR suppression and results in improved quality of life."