New Hope for Nerve Damage Patients as AstraZeneca/Ionis' Eplontersen Receives FDA Nod for Hereditary Amyloidosis Treatment

Friday, FDA approved AstraZeneca Plc AZN and Ionis Pharmaceuticals Inc's IONS Wainua (eplontersen) for polyneuropathy (nerve damage) of hereditary transthyretin-mediated amyloidosis in adults (hATTR-PN or ATTRv-PN). 

Wainua is the only approved medicine for ATTRv-PN that can be self-administered via an auto-injector.

The approval was based on the positive 35-week interim analysis from the NEURO-TTRansform Phase 3 trial, which showed that patients treated with Wainua demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint of quality of life.

AstraZeneca and Ionis will commercialize Wainua for ATTRv-PN in the U.S. and seek regulatory approval in Europe and other parts of the world as part of a global development and commercialization agreement.

This agreement was recently expanded to include exclusive rights for AstraZeneca to commercialize Wainua in Latin America and all other countries outside the U.S. 

Wainua will be available in the US in January 2024.

ATTR cardiomyopathy and polyneuropathy are progressive systemic diseases caused by aging or genetic mutations (variants), resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium and peripheral nerves, respectively.

Worldwide, there are an estimated 300,000 to 500,000 patients with ATTR-CM and about 40,000 patients with ATTRv-PN.

Citing some analysts, Reuters noted total peak sales to be between $3.5 billion and $7 billion if the drug gets expanded approval to treat cardiomyopathy.

Price Action: IONS shares are up 7% at $52.55 during the premarket session on the last check Friday. AZN shares are up 0.47% at $66.61.