Steve Bryson, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking the approval of donidalorsen as a preventive treatment for swelling attacks in adults and children, ages 12 and older, with hereditary angioedema (HAE). According to Ionis Pharmaceuticals, the company developing the therapy, the…

A new mutation in the SERPING1 gene was identified as the cause of hereditary angioedema (HAE) in several members of a Han Chinese family, a study reports. Blood tests revealed varied levels of C1-INH, the protein encoded by the SERPING1 gene and used to help diagnose the condition, in affected…

The European Commission has granted orphan medicinal product designation (OMPD) to navenibart, an experimental long-acting therapy for hereditary angioedema (HAE). The designation is aimed at providing incentives for the development of therapies that are meant to diagnose, prevent, or treat rare diseases, or those affecting fewer than 10,000 people…

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…