AZN :FDA Oks Wainua For Treatment Of Polyneuropathy Of Hereditary Transthyretin-Mediated Amyloidosis

(RTTNews) - The U.S. Food and Drug Administration has approved Ionis Pharmaceuticals Inc. (IONS) and AstraZeneca's (AZN.L, AZN) Wainua or eplontersen for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. Wainua is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector, the companies said in a statement.

IONS closed Thursday's regular trading at $49.11 up $0.78 or 1.61%. In the after-hours trading, the stock further gained $3.44 or 7.00%.

The approval was based on the positive 35-week interim analysis from the Phase 3 NEURO-TTRansform study which showed 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, and key secondary endpoint of quality of life on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy.

ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. WAINUA is a ligand-conjugated antisense oligonucleotide medicine designed to reduce the production of TTR protein at its source.

Wainua will be available in the U.S. in January 2024.

As part of a global development and commercialization agreement, AstraZeneca and Ionis will commercialize Wainua for the treatment of ATTRv-PN in the U.S. and are seeking regulatory approval in Europe and other parts of the world. The agreement was recently expanded to include exclusive rights for AstraZeneca to commercialize Wainua in Latin America in addition to all other countries outside the U.S. Wainua was granted Orphan Drug Designation in the U.S. and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR).

Eplontersen is currently being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three-to-five years from disease onset. The CARDIO-TTRansform Phase 3 study is fully enrolled with more than 1,400 patients.

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