Ionis announces FDA acceptance of New Drug Application for donidalorsen for prophylactic treatment of HAE
IONS 11.04.2024
Drug:UNKNOWN Donidalorsen
Diseases:Hereditary angioedema
Date of Upcoming Event:2024-11-01
Name of Upcoming Event:2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting

About Gravity Analytica
- Donidalorsen will be a first-in-class RNA-targeted medicine for hereditary angioedema, assuming approval
- Donidalorsen PDUFA date set for
August 21, 2025 - Donidalorsen has the potential to be Ionis' second independent commercial launch
HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. Donidalorsen is designed to reduce the production of prekallikrein (PKK), interrupting the pathway that leads to HAE attacks.
"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," said
The FDA previously granted donidalorsen Orphan Drug Designation in 2023. Otsuka, which has exclusive rights to commercialize donidalorsen in Europe and
Ionis recentlypresentednew results from the Phase 3 and Phase 2 OLE studies at the 2024
Across all three studies, donidalorsen was well-tolerated, with no serious treatment-emergent adverse events (TEAEs) related to donidalorsen. Most adverse events (AEs) were mild or moderate in severity, and injection site reactions were the most common AE.
The donidalorsen ACAAI e-poster presentations can be found on Ionis'website.
About Hereditary Angioedema (HAE)HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect more than 20,000 patients in the
About DonidalorsenDonidalorsen is an investigational RNA-targeted medicine designed to target prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with acute attacks of hereditary angioedema (HAE). By reducing the production of
Donidalorsen is an investigational medicine that has not been approved for the treatment of any disease by regulatory authorities.
About Ionis Pharmaceuticals, Inc.For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visitIonis.comand follow us onX (Twitter)andLinkedIn.
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to
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Ionis Pharmaceuticals Media Contact:Hayley Soffer –media@ionis.com– 760-603-4679
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