News

Rare angioedema types marked by time to diagnosis, attack differences

There are notable differences between hereditary angioedema with normal C1-inhibitor function (HAE nC1-INH) and idiopathic angioedema of unknown etiology (AE-UNK), even though they share many features, a real-world Canadian study indicates. Some of the differences include the time to reach a diagnosis, attack triggers and characteristics, and treatment…

Company plans to seek Canada OK of HAE treatment donidalorsen

Theratechnologies said it will ask Health Canada next year to approve donidalorsen, an experimental injection therapy designed to reduce the risk of swelling attacks in people with hereditary angioedema (HAE). The company agreed to acquire exclusive rights to commercialize the treatment in the country. Donidalorsen, originally developed…

Rare connective tissue diseases more common in HAE: Study

Rare connective tissue diseases are more common among people with hereditary angioedema (HAE) than in the general population, according to the results of a new Italian study the researchers say is the first to seek to better understand disease prevalence among HAE patients. Among the diseases investigated — which…

Orladeyo now available to treat HAE patients in Ireland

Orladeyo (berotralstat), an oral therapy designed to reduce or prevent hereditary angioedema (HAE) attacks, is now available in Ireland. The Irish Health Services Executive (HSE) recommended Orladeyo as a prophylactic, or preventive, treatment for HAE in adults and adolescents, ages 12 and older. Biocryst Pharmaceuticals, the company marketing…

DRI Healthcare Trust secures royalty rights for sebetralstat

DRI Healthcare Trust, an investment firm, has acquired rights to royalty interest in sebetralstat, an on-demand treatment for hereditary angioedema (HAE) that’s being considered for approval in several countries, including the U.S. For Kalvista Pharmaceuticals, the therapy’s developer, the deal could be worth more than $175 million,…

Preclinical studies of HAE gene-editing therapy promising

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…