Top 10 angioedema news stories of 2024
News on prevention, treatment of swelling attacks of most interest to readers
Throughout 2024, Angioedema News brought our readers all the latest news and updates on clinical research and scientific advances related to the rare disease.
Here are this year’s top 10 most-read articles on angioedema — many related to the prevention and treatment of swelling, the hallmark of the disease — each with a brief description. We’re committed to remaining a dependable resource for the angioedema community throughout 2025.
No. 10 – Otsuka strikes deal to commercialize donidalorsen in Europe
Under a deal with Ionis Pharmaceuticals, Otsuka Pharmaceutical gained exclusive rights to commercialize the experimental therapy donidalorsen in Europe. Otsuka is also responsible for filing applications to European regulatory authorities to seek approval of the therapy candidate — a potential prophylactic, or preventive, treatment to reduce the rate of swelling attacks in people with hereditary angioedema (HAE). The company also obtained exclusive rights to market donidalorsen in the Asia-Pacific region later in the year.
No. 9 – Monthly HAE attacks drop by 95% with single dose of NTLA-2002
Intellia Therapeutics’ gene-editing therapy candidate NTLA-2002 reduced monthly swelling attacks by 95% among 10 people with HAE, according to data from the Phase 1 part of a Phase 1/2 clinical trial. Such treatment also sustainably reduced the levels of kallikrein, the enzyme that’s overactive in HAE. In October, the company launched a Phase 3 study dubbed HAELO to evaluate the therapy’s efficacy in a larger number of patients. Those data may provide evidence to support a future application seeking NTLA-2002’s approval.
No. 8 – Takeda partners with Pentavere to drive new HAE treatments
Pentavere Research Group partnered with Takeda Canada to create one of the largest datasets of patients with an ultra-rare form of HAE, in which the levels of a protein called C1 inhibitor are normal. The partners now aim to address data gaps on the prevalence and clinical outcomes of this rare form of HAE, for which no approved therapies exist. Toronto-based Pentavere, an artificial intelligence digital health data company, analyzed data from Takeda’s observational PROSPECT study to glean insights to inform therapeutic strategies and improve patient outcomes.
No. 7 – Takhzyro shown to safely prevent HAE attacks in adolescents
Treatment with Takeda‘s Takhzyro (lanadelumab) reduced monthly swelling attacks by a mean of 94.7% in adolescents with HAE, according to data from a Phase 3 extension study. Among 21 HAE patients, ages 12 to 17, who participated in the HELP open-label extension study, nearly all days were free of swelling attacks, with eight patients experiencing no attack during treatment. Patients also reported a clinically meaningful improvement in quality of life and high treatment satisfaction.
No. 6 – Pharvaris testing deucrictibant as on-demand therapy in Phase 3 study
In March, Pharvaris began testing an immediate-release capsule formulation of deucrictibant in a Phase 3 study, called RAPIDe-3, as an on-demand treatment for HAE swelling attacks. During the study, all participants self-administer deucrictibant or a placebo to treat two swelling attacks in a random order. The primary goal of the trial is to evaluate deucrictibant’s ability to shorten the time to onset of symptom relief.
No. 5 – FDA lifts hold on deucrictibant as preventative HAE treatment
The U.S. Food and Drug Administration (FDA) lifted its clinical hold, in January, on deucrictibant as a preventive treatment for HAE attacks. The agency had placed the hold on all clinical trials testing the therapy after a review of preclinical data; the hold was lifted after the completion of a six-month toxicology study in a rodent model. That decision enabled Pharvaris to restart the open-label extension part of the Phase 2 CHAPTER-1 study, which is assessing the safety and efficacy of deucrictibant as an HAE preventive treatment.
No. 4 – Ionis put together its application seeking FDA approval of donidalorsen
Back in January, Ionis announced its plans to ask the FDA to approve donidalorsen for people with HAE, based on data from the Phase 3 OASIS-HAE clinical trial, which showed that the therapy significantly reduced the rate of swelling attacks compared with a placebo. Other secondary trial goals of disease control and life quality were also met. The approval application has since been accepted for review by the FDA, and a decision is now expected by Aug. 21, 2025.
No. 3 – Long-term preventive garadacimab safe in HAE patients
Long-term preventive treatment with CSL Behring’s garadacimab safely reduced the frequency of swelling attacks in adults and adolescents with HAE, according to interim data from an ongoing Phase 3 extension study. Most adverse events were mild or moderate, and those associated with treatment were mainly injection site reactions. The extension study followed the placebo-controlled Phase 3 VANGUARD trial, which showed garadacimab reduced the number of monthly swelling attacks by more than 85% in HAE patients, ages 12 and older. The candidate therapy is now under review for approval in the U.S. and the European Union.
No. 2 – Preventative Haegarda reduces HAE attacks in real-world study
Long-term preventive treatment with Haegarda reduced the rate and severity of swelling attacks, and the use of rescue medications, among 36 adults with HAE who had previously been on on-demand therapy for a year. Based on patient interviews, the treatment lowered the mean annualized attack frequency by 80.2%. Altogether, 20 patients saw their attack rates drop to one or fewer per year, with 12 having no attacks. Mean attack severity also fell significantly after treatment, as did the use of rescue medications. The therapy also led to improvements in several aspects of quality of life.
No. 1 – HAE attacks in adults drop with long-term daily Orladeyo
BioCryst Pharmaceuticals’ Orladeyo (berotralstat), when given as a long-term daily treatment, reduced swelling attacks and improved patient-reported outcomes in adults in Japan with HAE. Those were the results detailed in published data from Parts 2 and 3 of a multipart Phase 3 clinical trial called APeX-J, which tested the therapy in HAE patients ages 12 and older. Among patients on 150 mg of Orladeyo who completed about two years of treatment, the mean monthly attack rate dropped by 84.8%. Throughout the study, the median attack rate was one or fewer attacks each month, which fell to zero by the study’s end. Symptom-free days remained stable at around 90%, reaching a maximum of 339 days, or about 11 months. In Part 3, participants receiving doses of either 110 or 150 mg of the therapy showed a similar proportion of symptom-free days.
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At Angioedema News, we hope our stories and reporting throughout 2024 positively impacted the lives of those with angioedema. We look forward to serving the community further in 2025, seeking to provide ongoing support and information.
A very happy new year to all our readers!
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