Steve Bryson, PhD, science writer —

Intellia Therapeutics has launched a Phase 3 clinical trial to evaluate the efficacy and safety of NTLA-2002, a single-dose gene-editing therapy for people with hereditary angioedema (HAE). The new trial, dubbed HAELO (NCT06634420), was given the green light by the U.S. Food and Drug Administration (FDA) based…

Orladeyo (berotralstat), an approved oral treatment to prevent attacks in hereditary angioedema (HAE) patients 12 years and older, is one step closer to being eligible for reimbursement under public drug plans in Canada, according to its developer, Biocryst Pharmaceuticals. Biocryst has successfully completed negotiations with the pan-Canadian…

People with non-hereditary angioedema living with a substance use disorder (SUD) have a significantly elevated risk of breathing-related hospitalizations and complications, according to a U.S. nationwide study involving more than 140,000 people. The increased risk applied to all subtypes of SUD, including excessive use of alcohol, tobacco, cannabis, cocaine,…

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…