The number of cases of hereditary angioedema with normal C1 inhibitor (HAE-nl-C1INH) within the U.S. was estimated to be between 1,230 and 1,331 from May 2019 and April 2020, according to a survey conducted among U.S. physicians. Patients waited a mean of six years to receive a diagnosis, which…
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A 70-year-old woman diagnosed with non-episodic angioedema associated with eosinophilia (NEAE) — high levels of immune eosinophil cells — was successfully treated with reslizumab, a medication normally used to treat asthma associated with eosinophilia, according to a case report. Her symptoms had started after receiving a…
The U.S. Food and Drug Administration (FDA) has extended the approval of Takhzyro (lanadelumab) to include children with hereditary angioedema (HAE) starting at age 2. Takhzyro, available to patients ages 12 and older in the U.S. since 2018, is now the only approved treatment to prevent HAE swelling…
Astria Therapeutics has initiated a Phase 1b/2 trial called ALPHA-STAR to test STAR-0215, the company’s investigational therapy to prevent swelling attacks in people with hereditary angioedema (HAE). ALPHA-STAR (NCT05695248) intends to enroll 18 adults with HAE types 1 or 2 who have had at least four swelling…
Despite long-term preventive treatment, a 38-year-old woman with hereditary angioedema (HAE) with normal C1-inhibitor levels had severe episodes of disease worsening, as described in a recent case report. The life-threatening sequelae and decreased quality of life illustrate the need for more research with this rare form of HAE, according…
BioCryst Pharmaceuticals has tapped Swixx BioPharma to commercialize the hereditary angioedema (HAE) treatment Orladeyo (berotralstat) in central and Eastern Europe. Agreement terms call for Swixx, a Switzerland-based biopharmaceutical company, to handle commercialization of the oral therapy in 15 markets. “We continue to build partnerships with companies…
Enrollment has begun in APeX-P, a Phase 3 trial designed to evaluate the effects of Orladeyo (berotralstat) in up to 20 children, ages 2–11, with hereditary angioedema (HAE). The open-label study, sponsored by BioCryst Pharmaceuticals, will provide data on Orladeyo’s safety, pharmacological properties, and ability to prevent…
The first group of participants has been dosed in a Phase 1 clinical trial evaluating the safety and pharmacological properties of ADX-324, an experimental treatment for hereditary angioedema (HAE) that is being developed by Adarx Pharmaceuticals. “Dosing of our first participants in this trial is a major milestone…
Despite being treated for gastrointestinal symptoms of a hereditary angioedema (HAE) attack, an 80-year-old woman with the disease had to undergo surgery to pinpoint their exact cause. Her abdominal pain, vomiting, and nausea eased only mildly with a C1-esterase inhibitor (C1-INH) concentrate, a type of medication usually recommended to…
The prevalence of hereditary angioedema (HAE) in Asia-Pacific countries is lower than that reported for the disease in Western countries, according to data from an international survey. However, access to on-demand and preventive therapies to treat HAE is limited in these nations — a phenomenon researchers say is linked…
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