News

An assay that measures C1 inhibitor (C1-INH) activity on a dried blood spot after a blood sample is blotted onto a piece of filter paper appears to work as well as a standard, color-based assay to diagnose hereditary angioedema (HAE) and is easier to handle, a study found. Dried…

The first patient has been dosed in a Phase 3 clinical trial testing NTLA-2002, Intellia Therapeutics’ gene-editing treatment candidate for hereditary angioedema (HAE). The trial, called HAELO (NCT06634420), is expected to enroll an estimated 60 adults with HAE types 1 or 2; it’s recruiting participants at five…

Kalvista Pharmaceuticals has submitted an application asking Japan’s Ministry of Health, Labour and Welfare (MHLW) to approve its oral therapy sebetralstat as an on-demand treatment to manage swelling attacks in people with hereditary angioedema (HAE), ages 12 and older. “The submission of our [new drug application] for sebetralstat…

Astria Therapeutics is on track to start a Phase 3 clinical trial of navenibart, its therapy to prevent swelling attacks in people with hereditary angioedema (HAE), by March. The trial, dubbed ALPHA-ORBIT, will test flexible dosing schedules, with navenibart being given every three months and every six months.

CSL’s garadacimab, under the brand name Andembry, has been approved in the U.K. and Australia for the routine prevention of swelling attacks in adults and adolescents with hereditary angioedema (HAE), ages 12 and older. In Australia, the therapy is intended for HAE patients with deficiency or dysfunction of…

Pharvaris plans a trial of deucrictibant to treat acquired angioedema this year, following promising data showing the experimental oral small molecule helped to stop or prevent swelling attacks in three patients. The company has been testing deucrictibant in a pair of Phase 3 trials, CHAPTER-3 (NCT06669754)…

Intellia Therapeutics is planning to submit an application requesting the approval of NTLA-2002, its gene-editing therapy for hereditary angioedema (HAE), to U.S. regulators in late 2026. The company recently announced it would be discontinuing programs for other diseases, as well as reducing its workforce, to focus efforts…

Takhzyro (lanadelumab), an injectable medication used to prevent hereditary angioedema (HAE) attacks, is highly effective at reducing the frequency of these episodes and increasing the proportion of patients who remain attack-free, a study into its real-world use reports. Researchers evaluated Takhzyro’s use in the clinic among HAE patients…

Delaying on-demand treatment of hereditary angioedema (HAE) swelling attacks increases their severity and duration, and its symptoms are also more likely to spread to other parts of the body, according to a study that also found about 79% of patients reported treatment-related anxiety, which was associated with delaying…

Despite being on prophylatic, or preventive, treatment, people with hereditary angioedema (HAE) report similar struggles in managing and recovering from acute attacks to those using on-demand therapy alone, a new survey study in the U.S. has revealed. The researchers noted that anxiety levels among patients when anticipating the use…