Donidalorsen FDA Approval Status
Last updated by Judith Stewart, BPharm on Nov 6, 2024.
FDA Approved: No
Generic name: donidalorsen
Company: Ionis Pharmaceuticals, Inc.
Treatment for: Hereditary Angioedema
Donidalorsen is an investigational RNA-targeted medicine in development for prophylaxis to prevent attacks of hereditary angioedema.
- Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic condition that involves recurrent attacks of angioedema (severe swelling) in various parts of the body.
- Donidalorsen is an investigational RNA-targeted medicine that works by targeting and reducing the production of prekallikrein (PKK). PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE.
- On November 4, 2024, Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) had accepted for review the New Drug Application (NDA) for donidalorsen for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older. The FDA has set an action date of August 21, 2025 under the Prescription Drug User Fee Act (PDUFA).
- The NDA submission was based on positive results with monthly and bi-monthly dosing in the pivotal Phase 3 OASIS-HAE and OASISplus (open label extension (OLE) and switch) studies, as well as the ongoing Phase 2 OLE study.
Development timeline for donidalorsen
Further information
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.