Ionis announces FDA acceptance of New Drug Application for donidalorsen for prophylactic treatment of HAE
Ionis Pharmaceuticals (IONS) announced FDA acceptance of its New Drug Application for donidalorsen, a first-in-class RNA-targeted medicine for hereditary angioedema (HAE) prevention. The FDA set a PDUFA date of August 21, 2025. The application is supported by positive results from Phase 3 OASIS-HAE and OASISplus studies, showing a 96% mean reduction in HAE attack rates maintained up to three years. The drug was well-tolerated with no serious treatment-related adverse events. If approved, donidalorsen will be Ionis' second independent commercial launch. Otsuka holds exclusive commercialization rights in Europe and Asia Pacific.
Ionis Pharmaceuticals (IONS) ha annunciato l'accettazione da parte della FDA della sua Nuova Richiesta di Medicinali per donidalorsen, un medicinale innovativo mirato all'RNA per la prevenzione dell'angioedema ereditario (HAE). La FDA ha fissato una data PDUFA per il 21 agosto 2025. La domanda è supportata da risultati positivi degli studi di Fase 3 OASIS-HAE e OASISplus, che mostrano una riduzione media del 96% nei tassi di attacchi da HAE mantenuta fino a tre anni. Il farmaco è stato ben tollerato senza eventi avversi gravi correlati al trattamento. Se approvato, donidalorsen sarà il secondo lancio commerciale indipendente di Ionis. Otsuka detiene i diritti esclusivi di commercializzazione in Europa e nella regione Asia-Pacifico.
Ionis Pharmaceuticals (IONS) anunció la aceptación por parte de la FDA de su Solicitud de Nuevo Medicamento para donidalorsen, un medicamento dirigido al ARN de primera clase para la prevención del angioedema hereditario (HAE). La FDA estableció una fecha PDUFA para el 21 de agosto de 2025. La solicitud cuenta con el respaldo de resultados positivos de los estudios de Fase 3 OASIS-HAE y OASISplus, que muestran una reducción media del 96% en las tasas de ataques de HAE mantenida hasta por tres años. El medicamento fue bien tolerado sin eventos adversos graves relacionados con el tratamiento. Si se aprueba, donidalorsen será el segundo lanzamiento comercial independiente de Ionis. Otsuka tiene los derechos exclusivos de comercialización en Europa y Asia-Pacífico.
아이오니스 제약(Ions)은 FDA가 유전성 혈관부종(HAE) 예방을 위한 최초의 RNA 표적 의약품인 도니달로르센의 신약 신청서를 승인했다고 발표했습니다. FDA는 2025년 8월 21일을 PDUFA 날짜로 설정했습니다. 이 신청서는 HAE 발작률이 3년까지 유지되는 평균 96% 감소를 보여주는 3상 OASIS-HAE 및 OASISplus 연구의 긍정적인 결과에 기반하고 있습니다. 이 약물은 치료와 관련된 심각한 부작용 없이 잘 견뎌졌습니다. 승인된다면 도니달로르센은 아이오니스의 두 번째 독립 상업 출시가 될 것입니다. 오츠카는 유럽 및 아시아 태평양에서 독점적인 상업화 권리를 보유하고 있습니다.
Ionis Pharmaceuticals (IONS) a annoncé l'acceptation par la FDA de sa demande de nouveau médicament pour le donidalorsen, un médicament innovant ciblant l'ARN pour la prévention de l'angioedème héréditaire (HAE). La FDA a fixé une date PDUFA au 21 août 2025. La demande est soutenue par des résultats positifs des études de phase 3 OASIS-HAE et OASISplus, montrant une réduction moyenne de 96 % des taux d'attaques d'HAE maintenue pendant trois ans. Le médicament a été bien toléré sans événements indésirables graves liés au traitement. S'il est approuvé, le donidalorsen sera le deuxième lancement commercial indépendant d'Ionis. Otsuka détient les droits exclusifs de commercialisation en Europe et en Asie-Pacifique.
Ionis Pharmaceuticals (IONS) hat die Annahme des Antrags auf Zulassung eines neuen Arzneimittels für Donidalorsen durch die FDA bekannt gegeben, einem ersten RNA-zielgerichteten Medikament zur Prävention von hereditärem Angioödem (HAE). Die FDA hat ein PDUFA-Datum für den 21. August 2025 festgelegt. Der Antrag wird durch positive Ergebnisse der Phase-3-Studien OASIS-HAE und OASISplus unterstützt, die eine durchschnittliche Reduzierung der HAE-Attackraten um 96 % zeigen, die bis zu drei Jahre aufrechterhalten wird. Das Medikament wurde gut vertragen, ohne schwerwiegende behandlungsbedingte Nebenwirkungen. Bei Genehmigung wird Donidalorsen die zweite unabhängige Markteinführung von Ionis sein. Otsuka hält die exklusiven Vermarktungsrechte in Europa und im asiatisch-pazifischen Raum.
- FDA acceptance of NDA for donidalorsen with PDUFA date set
- Significant clinical efficacy with 96% reduction in HAE attack rates
- Positive safety profile with no serious treatment-related adverse events
- Potential for second independent commercial launch
- Geographic expansion through Otsuka partnership for Europe and Asia Pacific
- Long regulatory timeline with PDUFA date set for August 2025
Insights
The FDA acceptance of donidalorsen's NDA marks a significant milestone for Ionis in the hereditary angioedema (HAE) treatment space. The clinical data is particularly compelling, showing a remarkable
The safety profile appears robust with no serious treatment-related adverse events, which is important for chronic prophylactic treatment. Monthly and bi-monthly dosing options provide flexibility that could enhance patient compliance. The Orphan Drug Designation and potential approval by August 2025 could position Ionis favorably in the
This regulatory milestone strengthens Ionis' commercial expansion strategy. As their second potential independent commercial launch, donidalorsen represents a significant revenue opportunity in the rare disease market. The partnership with Otsuka for European and Asia Pacific markets provides additional revenue streams through potential milestone payments and royalties.
The HAE market is currently dominated by established players, but donidalorsen's efficacy data and convenient dosing schedule could drive market share gains. The potential approval timing in mid-2025 gives Ionis adequate time to build commercial infrastructure and launch preparations, while maintaining strong cash runway.
- 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 Brett Monia, Ph.D., chief executive officer of Ionis. "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."
The FDA previously granted donidalorsen Orphan Drug Designation in 2023. Otsuka, which has exclusive rights to commercialize donidalorsen in Europe and
Ionis recently presented new results from the Phase 3 and Phase 2 OLE studies at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, which demonstrate that donidalorsen delivered significant and sustained reductions in HAE attacks, with overall sustained mean reduction in HAE attack rates of
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 Donidalorsen
Donidalorsen 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 PKK, donidalorsen could be an effective prophylactic approach to preventing HAE attacks, if approved.
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, visit Ionis.com and follow us on X (Twitter) and LinkedIn.
Ionis Forward-looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of our commercial medicines, donidalorsen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2023, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals Investor Contact:
D. Wade Walke, Ph.D. – IR@ionis.com – 760-603-2331
Ionis Pharmaceuticals Media Contact:
Hayley Soffer – media@ionis.com – 760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.
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