José Lopes, PhD, managing science editor —

Use of genetic testing enabled the detection of hereditary angioedema (HAE) in a previously misdiagnosed woman, found to be carrying a gene variant linked with HAE of unknown genetic cause. The findings support the consideration of genetic testing to diagnose unexplained angioedema, with an end goal of speeding treatment…

The unique mode each specific compound binds to and blocks the enzyme plasma kallikrein could lead to the development of improved therapies for patients with hereditary angioedema (HAE), according to a new study. The research, “Structures of full-length plasma kallikrein bound to highly specific inhibitors describe…

Daily treatment with BCX7353, an investigative oral inhibitor of plasma kallikrein by BioCryst Pharmaceuticals, was well-tolerated and lowered attack rates in hereditary angioedema (HAE) patients included in a Phase 3 trial. Both 110 mg and 150 mg doses provided a significant benefit by decreasing HAE attacks — the APeX-2…

Using under-the-skin delivery of a C1-inhibitor (C1-INH) formulation in patients with hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) decreased the number and severity of HAE attacks, was well-tolerated, and was associated with high patient satisfaction in a Phase 3 trial. The research, “Fixed-Dose Subcutaneous C1-Inhibitor Liquid…

An abnormally short form of the clotting protein factor XII (FXII), caused by specific mutations in its gene, may lead to swelling episodes in hereditary angioedema (HAE) patients with normal C1-inhibitor activity, according to a study. The study, “A Mechanism for Hereditary Angioedema with Normal C1-Inhibitor:…

Delays between symptom onset and diagnosis of hereditary angioedema (HAE) types 1 and 2 have been decreasing, according to a real-world study that also revealed a younger age at diagnosis in this patient population over time. The study, “Improvement in diagnostic delays over time in patients…

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), favors granting marketing authorization to lanadelumab for routine prevention of hereditary angioedema (HAE) attacks in patients ages 12 and older. Shire’s lanadelumab is a monoclonal antibody that specifically binds to…

The oral treatment candidate BCX7353 eases hereditary angioedema (HAE) attacks, reduces the occurrence of symptoms, and helps decrease the use of rescue medications, according to BioCryst Pharmaceuticals’ early Phase 2 trial results. The data also showed that the investigational therapy was safe and well-tolerated. BCX7353 is an oral…

Treatment of hereditary angioedema (HAE) with C1-esterase inhibitors (C1-INH) provides efficient relief of acute attacks across different locations of the body, according to a new study. The research was presented at the 2018 American Academy of Allergy Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress, which…

Patients with hereditary angioedema (HAE) show development of atherosclerosis in peripheral blood vessels during symptom-free periods, a new study reports. The research, “Impaired Endothelial Function in Hereditary Angioedema During the Symptom-Free Period,” was published in the journal Frontiers in Psychology. HAE is caused mainly by…