FDA Accepts Ionis Pharmaceuticals' NDA for Donidalorsen as Prophylactic Treatment for HAE

Ionis Pharmaceuticals, Inc. has reached a significant milestone with the U.S. Food and Drug Administration (FDA) accepting for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine. This treatment is designed for the prophylaxis to prevent attacks of hereditary angioedema (HAE) in both adult and pediatric patients aged 12 years and older. The FDA has set an action date of August 21, 2025, under the Prescription Drug User Fee Act (PDUFA).

Hereditary angioedema (HAE) is a rare, potentially life-threatening genetic condition characterized by recurrent episodes of severe swelling in various body parts, including the hands, feet, genitals, stomach, face, and throat. Donidalorsen works by reducing the production of prekallikrein (PKK), thereby interrupting the pathway that leads to HAE attacks.

The NDA submission was supported by positive results from the pivotal Phase 3 OASIS-HAE and OASISplus studies, which included both monthly and bi-monthly dosing regimens, as well as the ongoing Phase 2 open-label extension (OLE) study. These studies demonstrated the potential of donidalorsen to significantly impact the prophylactic treatment landscape for HAE.

Brett Monia, Ph.D., CEO of Ionis, expressed optimism about donidalorsen's potential, stating, "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." He also highlighted the significance of the FDA's acceptance of the NDA, marking a step closer to potentially launching a new treatment option for patients with serious diseases.

This development underscores Ionis Pharmaceuticals' commitment to addressing unmet medical needs and bringing innovative treatments to patients suffering from rare and serious conditions.