FDA approves Ionis/AstraZeneca WAINUA (eplontersen) for hATTR polyneuropathy

IONIS PHARMACEUTICALS INC

Approval based on Phase 3 NEURO-TTRansform 35-week interim; co-primary endpoints of serum TTR and mNIS+7 and Norfolk QoL-DN showed sustained benefit.
WAINUA is the only self-administered auto-injector treatment for ATTRv-PN; available in U.S. in January 2024.
Global regulatory reviews underway; AstraZeneca holds commercialization rights outside U.S., recently expanded to Latin America.
Eplontersen also in Phase 3 CARDIO-TTRansform for ATTR-CM, fully enrolled with >1,400 patients.

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