News

Mutations in the CPN1 gene leading to a deficiency in the activity of the carboxypeptidase N (CPN) enzyme were found in four families with hereditary angioedema (HAE),  according to a new study from Europe. The team of researchers identified three genetic variants in the CPN1 gene that were associated…

Orladeyo (berotralstat), an oral therapy for preventing swelling attacks in adults and adolescents with hereditary angioedema (HAE), is now available in Italy. The Italian Medicines Agency (Agenzia Italiana del Farmaco, AIFA), has finalized reimbursement and is recommending Orladeyo as a routine prophylactic (preventive) treatment for people with HAE,…

Note: This story was updated March 12, 2024, to clarify the data pertaining to the proportion of sebetralstat-treated swelling attacks seen in HAE patients taking part in the KONFIDENT trial. A single dose of oral sebetralstat — not the two allowed in trial — was sufficient to ease or…

People with hereditary angioedema (HAE) who were new to Orladeyo (berotralstat) treatment saw rapid reductions in the number of swelling attacks they experienced soon after starting the oral therapy, according to real-world data from the U.S. The benefits of Orladeyo lasted as long as 18 months, or about…

Birth control pills and other changes in hormone levels are a common trigger for swelling attacks among women with rare forms of hereditary angioedema (HAE), according to a new study of patients in Portugal. “The main identified triggers were oral estrogen-containing contraceptives (in 24 of 24 women; 100%),” the…

Sebetralstat, an oral small molecule being developed by KalVista Pharmaceuticals as an on-demand treatment for hereditary angioedema (HAE), has been issued an innovation passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). An innovation passport is the first step into the U.K.’s Innovative Licensing and Access…

Preventive treatment with Takhzyro (lanadelumab) reduced swelling attack frequency and improved quality of life in Japanese patients with hereditary angioedema (HAE), a small real-world study shows. The study, “Effect of lanadelumab on attack frequency and QoL in Japanese patients with hereditary angioedema: Report of five…

On-demand treatment with the experimental oral medication sebetralstat was effective for resolving swelling attacks in people with hereditary angioedema (HAE) — meeting all endpoints, or goals, in the Phase 3 KONFIDENT trial — top-line data show. Based on the positive findings, sebetralstat’s developer KalVista Pharmaceuticals is planning…

A 60-year-old man with hereditary angioedema (HAE) caused by an unknown mutation developed visual loss due to swelling of the optic nerve in both eyes, according to a case reported in Italy. His symptoms eased after starting treatment with Berinert (human C1 esterase inhibitor), an intravenous (into-the-vein) C1…

Three genetic regions were found to be associated with the risk of developing angioedema as a side effect of angiotensin-converting enzyme (ACE) inhibitors, according to a genome-wide association study (GWAS) of European and African-American people. These include a new genetic locus that hadn’t been…