US biotechnology company Ionis Pharmaceuticals Inc (Nasdaq: IONS) announced on Monday that its New Drug Application (NDA) for donidalorsen has been accepted for review by the US Food and Drug Administration (FDA).

Donidalorsen is an investigational RNA-targeted medicine for prophylaxis intended to prevent attacks of hereditary angioedema (HAE) in adult and paediatric patients 12 years of age and older.

The FDA has scheduled an action date of 21 August 2025 based on the Prescription Drug User Fee Act (PDUFA). The application was based on positive results with monthly and bi-monthly dosing in the pivotal Phase 3 OASIS-HAE and OASISplus (open label extension and switch) studies, and the ongoing Phase 2 OLE study.

Brett Monia, PhD, Ionis CEO said: "Despite currently available treatments, many people living with HAE continue to experience painful and potentially life-threatening breakthrough attacks. Based on the totality of clinical evidence from the Phase 3 OASIS-HAE and OASISplus studies, as well as new three-year results from our Phase 2 OLE study, we believe that donidalorsen has the potential to advance the prophylactic treatment paradigm for people living with HAE. With the FDA acceptance of our donidalorsen NDA, we are poised for our second independent launch next year, assuming approval, which will allow us to continue to deliver on our goal to bring a steady cadence of medicines to patients with serious diseases."