Ionis Pharmaceuticals, Inc. recently announced that the United States Food and Drug Administration (FDA) has accepted their New Drug Application (NDA) for donidalorsen, a pioneering RNA-targeted medication designed to prevent hereditary angioedema (HAE) attacks in patients aged 12 and older. The FDA has set August 21, 2025, as the decision date under the Prescription Drug User Fee Act (PDUFA).
The NDA submission is supported by encouraging results from both monthly and bi-monthly dosing regimens in the critical Phase 3 OASIS-HAE and OASISplus studies, which included an open-label extension (OLE) and switch study, along with ongoing findings from the Phase 2 OLE study.
Hereditary angioedema (HAE) is a rare genetic disorder that can pose life-threatening risks through recurrent severe swelling (angioedema) affecting different body regions such as the hands, feet, genitals, stomach, face, and throat. Donidalorsen works by diminishing the production of prekallikrein (PKK), thereby disrupting the pathway that triggers HAE attacks.
"Currently available treatments don't fully prevent painful and potentially life-threatening breakthrough HAE attacks for all patients," explained Brett Monia, Ph.D., CEO of Ionis. "Based on comprehensive clinical evidence from the Phase 3 OASIS-HAE and OASISplus studies, as well as new three-year data from our Phase 2 OLE study, we are optimistic that donidalorsen could transform preventative treatment options for HAE sufferers. With the FDA's acceptance of our NDA for donidalorsen, we are on track for our second independent product launch next year, contingent on approval, reinforcing our commitment to consistently deliver effective treatments for those with serious illnesses."
