Steve Bryson, PhD, science writer —

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…

A 48-year-old man experienced complete remission from hereditary angioedema (HAE) after undergoing a bone marrow transplant for blood cancer, a case study reports. The transplanted stem cells might have given rise to new cells that acted as a healthy source of C1-inhibitor (C1-INH), the protein that is missing or…

A genetic analysis revealed hereditary angioedema (HAE) was hidden among people with chronic, recurring swelling, but without an apparent family history and normal C1-inhibitor (C1-INH) levels, a study reported. Searching for mutations in patients with chronic, recurring angioedema with an inherited component may detect asymptomatic family members at risk…

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…

Adult and adolescent patients, ages 12 and older, with hereditary angioedema (HAE)  in Israel can now receive once-daily Orladeyo (berotralstat) to prevent HAE-related swelling attacks. The decision to approve the medication by the Israeli Ministry of Health follows similar decisions in Canada and Switzerland, as well as…