News

Dawnzera safely prevents HAE swelling attacks for up to 4 years

Long-term treatment with Dawnzera (donidalorsen) — approved in the U.S. earlier this year for people with hereditary angioedema (HAE) — effectively reduced swelling attacks in HAE patients in two clinical trial extension studies, according to new data. The newly reported results were based on data from patients who…

EU panel recommends approval of HAE treatment Dawnzera

A European Medicines Agency (EMA) committee recommended that Dawnzera (donidalorsen) be approved to prevent swelling attacks in hereditary angioedema (HAE) patients 12 and older. The positive opinion comes from the EMA’s Committee for Medicinal Products for Human Use (CHMP), which is tasked with reviewing safety and efficacy data…

Oral Ekterly gives relief to kids with HAE, trial data show

Ekterly (sebetralstat), given as a tablet that dissolves on the tongue, resulted in early, effective, and safe management of swelling attacks in children with hereditary angioedema (HAE) aged 2-11, according to interim data from a clinical trial. The ongoing Phase 3 study, KONFIDENT-KID (NCT06467084), which has enrolled…

Uncontrolled HAE attacks mean lower quality of life for patients

While the treatment landscape for people with hereditary angioedema (HAE) has improved significantly in the last decades, symptoms such as uncontrolled swelling attacks — occurring despite the use of on-demand or long-term preventive therapies — have resulted in low health-related quality of life (HRQoL) among patients, particularly women and those with…

HAE therapy ADX-324 hits Phase 3 milestone with first treatment

The first adult participant with hereditary angioedema (HAE) has been dosed in STOP-HAE, a Phase 3 clinical trial evaluating the efficacy of ADARx Pharmaceuticals’ investigational therapy ADX-324. Designed as a long-acting preventive treatment, ADX-324 aims to reduce the frequency of swelling attacks while requiring fewer injections. The…

Access to preventive treatment for HAE attacks limited: Brazil study

Many patients in Brazil lack access to first-line treatments such as Takhzyro (lanadelumab) to prevent swelling attacks in hereditary angioedema (HAE), a study found. “Despite recent advances, access to first-line therapies for long-term prophylaxis of HAE attacks remains limited,” the researchers wrote. These limitations “may likely be encountered in…

Biocryst to acquire Astria, getting experimental navenibart for HAE

Biocryst Pharmaceuticals is preparing to acquire Astria Therapeutics, and with it navenibart, a still-experimental, long-acting prophylactic (preventive) treatment that could improve care for people with hereditary angioedema (HAE) by reducing how often patients need injections. Navenibart is now in Phase 3 clinical testing, after earlier data…

Ekterly eases most HAE attacks within 20 minutes, trial data show

For most people with hereditary angioedema (HAE) given on-demand treatment in clinical trials with the now-approved therapy Ekterly (sebetralstat), swelling attacks stopped getting worse within 20 minutes of the oral medication being administered. That’s according to new data that Kalvista Pharmaceuticals, Ekterly’s developer, shared earlier this month…