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…
NTLA-2002, an experimental gene-editing therapy for hereditary angioedema (HAE) being developed by Intellia Therapeutics, has been awarded an innovation passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), the company announced. This designation is the first step in the U.K.’s Innovative Licensing and Access…
To continue to improve patient access to top-shelf angioedema care, the global network of Angioedema Centers of Reference and Excellence (ACARE) added six centers last year, two of them in Hong Kong. More centers are expected to join the ACARE network this year — more than a dozen…
Researchers have reported the case of two men who were initially misdiagnosed with a rare type of hereditary angioedema (HAE) known as HAE-nC1INH-UNK when the correct diagnosis was the more common mast cell-mediated angioedema. This “exceedingly rare subtype of HAE … appears to be often diagnosed in patients who…
Rates of prescriptions for Firazyr (icatibant), an on-demand treatment to control swelling episodes in people with hereditary angioedema (HAE), have increased in recent years, especially for women, according to a study from Sweden. While the finding could mean that more patients have poorly-controlled disease, more prescriptions for the…
A single dose of Berinert, a concentrate of C1 esterase inhibitor, when given with standard treatment, may not do more to ease angioedema airway symptoms triggered by a type of medication called an angiotensin-converting enzyme (ACE) inhibitor, a study found. The study, “Efficacy of human C1 esterase…
A single injection of STAR-0215, an experimental treatment to prevent swelling attacks in people with hereditary angioedema (HAE), safely led to rapid and sustained inhibition of the kallikrein protein in healthy adults, according to preliminary data from a Phase 1a trial. “STAR-0215 has shown early proof of concept for…
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