DAWNZERA donidalorsen APPROVED
Drug Profile
ModalityAntisense oligonucleotide
RouteSC
Therapy AreaRare Disease
Launch2025-08-01
Peak Sales Est$500M
Formulations[{"id":"dawnzera-sc","route":"SC","device":"Pre-filled syringe","setting":"PATIENT_SELF","frequency"
Companies
IONS (ORIGINATOR)100%
4578 (LICENSEE)0%
Mechanism: Antisense Oligonucleotide (Prekallikrein)
Expert: Antisense oligonucleotide (ASO) targeting hepatic prekallikrein (KLKB1) mRNA. Reduces plasma prekallikrein levels, thereby decreasing kallikrein-mediated bradykinin generation — the primary driver of angioedema attacks in HAE.
Everyday: A designer strand of genetic code that silences the gene making prekallikrein — a protein that triggers the swelling cascade in hereditary angioedema. Without prekallikrein, the dangerous swelling attacks cannot start.
Targets: []
Programs (1)
IndicationStageKey StudyRegional Status
HAE prophylaxisAPPROVEDOASIS-HAE[{"stage":"APPROVED","region":"US","approval_date":"2025-08-01"},{"stage":"APPRO
Notes
Ionis-discovered and owned. Ionis is NDA holder and commercializes independently in US. Otsuka licensed EU + APAC rights (Dec 2023 / Jun 2024). Theratechnologies licensed Canada rights (Dec 2024). Ionis retains manufacturing and global development control.
Data from Supabase · Updated 2026-03-24