Steve Bryson, PhD, science writer —

Changes in the levels of certain metabolites — molecules that take part in metabolism — in the blood may serve as accurate biomarkers to diagnose hereditary angioedema (HAE), a study suggests. Moreover, ratios of metabolite levels also were able to differentiate between people with HAE and those with…

KalVista Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration (FDA) requesting the approval of sebetralstat, an oral on-demand treatment for people, 12 and older, with hereditary angioedema (HAE). The agency now has 60 days to determine whether the application, submitted in the form of…

Treatment with oral Orladeyo (berotralstat) led to significantly fewer disease-related or all-cause hospitalizations and emergency room visits by people with hereditary angioedema (HAE) in the U.S., according to a real-world claims analysis. “The data presented here represent the first documentation that prescribing Orladeyo significantly reduces healthcare resource utilization,”…

A 46-year-old woman with hard to treat idiopathic angioedema was successfully treated with the asthma and allergies medication omalizumab, which is sold as Xolair, according to a recent case study. “In the context of isolated [angioedema], even after failure of up-dosed antihistaminic therapy, a trial with omalizumab is…

Long-term daily treatment with Orladeyo (berotralstat) effectively reduced swelling attacks and improved patient-reported outcomes in Japanese adults with hereditary angioedema (HAE), according to data from a Phase 3 clinical trial. Findings were detailed in the study, “Berotralstat for long-term prophylaxis of hereditary angioedema in Japan: Parts 2…

Intellia Therapeutics’ experimental gene-editing therapy NTLA-2002 reduced the number of monthly swelling attacks by 95% among 10 people with hereditary angioedema (HAE) treated in the Phase 1 portion of a Phase 1/2 clinical trial, according to newly published data. The single-dose treatment also led to sustained, dose-dependent reductions in…

The Phase 3 KONFIDENT trial, which is testing oral sebetralstat as an on-demand treatment for hereditary angioedema (HAE), has achieved its targeted number of on-treatment swelling attacks. The trial can wrap up now that three attacks per patient were treated. Topline data is expected in early 2024. If…

The European Commission has expanded the approval of Takhzyro (lanadelumab) to include children with hereditary angioedema (HAE) as young as 2 years of age. This makes Takhzyro the first routine preventive, or prophylactic, treatment for HAE attacks to be approved for patients younger than 6 in the European…

Most older adults with hereditary angioedema (HAE) have more frequent and severe swelling attacks, and more chronic diseases that combine to have a negative impact on their quality of life, results from a focus group study show. Other age-related difficulties included financial barriers to accessing medication. Despite these challenges,…

The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to Intellia Therapeutics’ NTLA-2002, an experimental gene-editing therapy to prevent swelling attacks in people with hereditary angioedema (HAE). A PRIME designation supports the development of experimental therapies that address unmet medical needs. Eligibility for this status is…