Lindsey Shapiro, PhD, science writer —

An investigational gene-editing therapy for hereditary angioedema (HAE) called P-KLKB1-101 showed it could safely work as intended in preclinical studies, according to recent data. The treatment, developed by Poseida Therapeutics, successfully edited the KLKB1 gene and lowered levels of the kallikrein enzyme across experiments in lab-grown human liver…

Initiating treatment with sebetralstat within a few minutes of the start of a hereditary angioedema (HAE) attack was associated with it resolving significantly faster than when treatment was delayed by more than two hours, according to new findings from the KONFIDENT Phase 3 trial. Sebetralstat also led to…

Treatment with deucrictibant continued to successfully prevent and treat swelling attacks in people with hereditary angioedema (HAE), according to new data from the open-label extension portions of two clinical trials. The findings, presented at the Bradykinin Symposium held in Berlin Sept. 5-6, support the use of deucrictibant…

The U.S. Food and Drug Administration (FDA) has agreed to review Kalvista Pharmaceuticals’ application seeking the approval of sebetralstat as an on-demand treatment for swelling attacks in people 12 and older with hereditary angioedema. Regulators expect to make their decision on the new drug application (NDA) the…

Hereditary angioedema (HAE) patients in South Australia are generally satisfied with their prophylactic (preventive) treatment regimens, a study found. Danazol appeared to be a popular choice for patients with milder disease, while newer therapies like C1 esterase inhibitor (C1-INH) products and Takhzyro (lanadelumab) were preferred for patients…

Treatment with donidalorsen to prevent swelling attacks led to fewer of them and gains in quality of life for adults and teens with hereditary angioedema (HAE), according to final data from a Phase 3 trial and interim findings from its open-label extension study. The patients who switched from other prophylactic…

KalVista Pharmaceuticals plans to submit an application to the U.S. Food and Drug Administration (FDA) in June seeking approval of its oral therapy sebetralstat for the on-demand treatment of hereditary angioedema (HAE), the company announced this week while unveiling its strategic plan for fiscal year 2025. Similar applications…

The investigational therapy donidalorsen was well tolerated and significantly reduced the rate of swelling attacks in hereditary angioedema (HAE) patients, meeting the main goal of the Phase 3 OASIS-HAE trial. Other secondary trial goals were also met, according to Ionis Pharmaceuticals, which is developing the treatment. The company…

Preventive treatment with Orladeyo (berotralstat) was associated with improved treatment satisfaction among hereditary angioedema (HAE) patients who had switched from injectable therapies, according to a recent clinical trial analysis. BioCryst Pharmaceutical’s oral treatment also was well tolerated and associated with low HAE attack rates, as has been…

The Institut national d’excellence en santé et services sociaux (INESSS) has recommended that BioCryst Pharmaceuticals’ Orladeyo (berotralstat) be reimbursed by public health plans for eligible adults and adolescents 12 and older with hereditary angioedema (HAE) in the Canadian province of Québec. A similar recommendation was made earlier…