Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.
Ionis will have 11 presentations at the conference, which is set to take place from February 28 to March 3.
The data from the pivotal Phase III OASIS and OASISplus studies and three-year data from a Phase II open-label extension study show “significant and clinically meaningful improvements in quality-of-life measures.”
Specifically, Ionis said donidalorsen was demonstrated to result in sustained reductions in HAE attack rates while providing enhanced disease control.
HAE is a rare genetic disorder characterized by unpredictable, recurrent episodes of severe swelling that can affect various parts of the body, including the hand, feet and airways. For many patients, managing these potentially life-threatening attacks remains a challenge, often leading to emergency interventions and a significant impact on quality of life.
It is estimated that HAE affects more than 20,000 people in the US and Europe. Prophylactic treatment approaches are often used in the US to prevent and reduce the severity of HAE attacks.
As an RNA ligand-conjugated antisense (LICA) prophylactic, donidalorsen aims to bring long-term control over the frequency and severity of HAE attacks.
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“In new analyses from our OASISplus prospective switch cohort, donidalorsen continued to demonstrate the ability to reduce the HAE attack rate burden and improve quality of life in patients previously on other prophylactic treatments, with the simplicity of monthly or every two-month self-administration via an autoinjector,” said Ionis’s senior vice president and head of clinical development Kenneth Newman, MD, in a statement from the company.
Dr. Newman also said that as a first-in-class RNA-targeted medicine, Ionis believes donidalorsen could potentially advance the prophylactic treatment paradigm for HAE.
Ionis’s New Drug Application (NDA) for donidalorsen to HAE attacks in adult and pediatric patients 12 years of age is currently under FDA review with a Prescription Drug User Fee Act (PDUFA) date of August 21, 2025.
If approved, donidalorsen would be the company’s second independent commercial product after the December 2024 approval of Tryngolza (olezarsen) as the first treatment for familial chylomicronemia syndrome (FCS).
Related: KalVista Shares New Data on Sebetralstat for Hereditary Angioedema (HAE)
The company’s SOD1-ALS treatment Qalsody (tofersen) and spinal muscular atrophy (SMA) drug Spinraza (nusinersen) were jointly developed with and marked by Biogen, while polyneuropathy caused by hereditary ATTR amyloidosis (hATTR-PN) treatment Wainua (eplontersen) is co-commercialized with AstraZeneca.
In 2024, Qalsody brought in $32 million, and Wainua generated $85 million in global sales, with $4 million and $20 million in royalties for Ionis, respectively.
Despite limited markets for the rare disease drugs, analysts predict that Wainua could reach $700 million in peak sales in just the ATTRv-PN indication.
This month, KalVista also shared positive new trial data for its oral, on-demand long-term prophylaxis (LTP) HAE treatment sebetralstat at the Western Society of Allergy, Asthma & Immunology (WSAAI) 2025 Annual Meeting.
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