AstraZeneca, Ionis get US FDA nod for Wainua in "important milestone"

(Alliance News) - AstraZeneca PLC and Ionis Pharmaceuticals Inc on Friday celebrated the news that their drug eplontersen was approved by the US Food & Drug Administration.

Cambridge, England-based AstraZeneca said eplontersen, brand name Wainua, is now authorised in the US to treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.

The news marks Wainua's first-ever regulatory approval, and it is the only approved ATTRv-PN treatment that can be self-administered via an auto-injector.

The pharmaceutical company said the approval was based on positive interim results from the NEURO-TTRansform Phase 3 trial.

At 35 weeks, AstraZeneca said the trial showed that patients treated with Wainua demonstrated consistent and sustained benefit on the primary endpoints of serum transthyretin concentration and neuropathy impairment. Patients' quality of life also improved.

ATTRv-PN, AstraZeneca explained, causes peripheral nerve damage with motor disability within five years of diagnosis, and is usually fatal within a decade without treatment.

"The FDA approval of Wainua marks an important milestone for people living with [ATTRv-PN], who will now have an effective, well-tolerated treatment...to combat this devastating disease," commented Ionis Chief Executive Officer Brett Monia.

"It is also a pivotal moment for Ionis as Wainua will be the first in a steady cadence of potential commercial launches for the company."

AstraZeneca said it and Ionis will commercialise Wainua to treat ATTRv-PN in the US, as part of a global development and commercialisation deal recently expanded to include exclusive rights for AstraZeneca in Latin America and all other countries outside the US.

AstraZeneca expects Wainua to become available in the US in January 2024.

AstraZeneca shares were down 0.8% at 10,392.00 pence each in London early on Friday.

By Emma Curzon, Alliance News reporter

Comments and questions to newsroom@alliancenews.com

Copyright 2023 Alliance News Ltd. All Rights Reserved.

Astrazeneca