FDA accepts Ionis NDA for donidalorsen

The US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, under development by Ionis Pharmaceuticals (Nasdaq: IONS).

Ionis noted that the agency has set an action date of August 21, 2025, under the Prescription Drug User Fee Act (PDUFA). The FDA application was based on positive results with monthly and bi-monthly dosing in the pivotal Phase III OASIS-HAE and OASISplus (open label extension (OLE) and switch) studies, as well as the ongoing Phase II OLE study.

"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 III OASIS-HAE and OASISplus studies, as well as new three-year results from our Phase II OLE study, we believe that donidalorsen has the potential to advance the prophylactic treatment paradigm for people living with HAE," said Brett Monia, chief executive of Ionis.