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Wainua eplontersen APPROVED
Drug Profile
ModalityAntisense oligonucleotide
RouteSC
Therapy AreaRare Disease
Launch2023-12-21
US LOE2035-12-22
Peak Sales Est$2000M
Formulations[{"id":"wainua-sc","doses":"45mg","route":"SC","device":"Pre-filled syringe","setting":"PATIENT_SELF
Companies
IONS (ORIGINATOR)0%
AZN (ORIGINATOR)100%
Mechanism: TTR silencer
Expert: GalNAc-conjugated antisense oligonucleotide that silences hepatic transthyretin mRNA via RNase H-mediated degradation, reducing both wild-type and variant TTR.
Everyday: Stops the liver from making a misfolded protein that damages nerves and the heart.
Targets: ["TTR"]
Revenue History
PeriodRevenue ($M)
2024$45M
Q1 2025$28M
Programs (2)
IndicationStageKey StudyRegional Status
hATTR-PNAPPROVEDNEURO-TTRansform[{"stage":"APPROVED","region":"US","approval_date":"2023-12-21"}]
hATTR-CMPHASE3CARDIO-TTRansform[]
Upcoming Catalysts (1)
Wainua - ATTR-CM - Ph3 - Topline (CARDIO-TTRansform) H2 2026
Notes
TTR-targeted ASO for hATTR-PN. Ionis partnership.
Data from Supabase · Updated 2026-03-24