Daily treatment with Orladeyo (berotralstat) oral granules led to early and sustained reductions in swelling attack rates in young children with hereditary angioedema (HAE), according to one-year interim data from an ongoing Phase 3 trial. The data, together with additional analyses on disease burden and patient experience, were presented…
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Most people with hereditary angioedema (HAE) who received a single, 50 mg dose of lonvoguran ziclumeran, also called lonvo-z and formerly known as NTLA-2002, in a clinical trial have remained free from swelling attacks and no longer require long-term preventive therapy up to three years of follow-up. That’s according…
Note: This story was updated Dec. 15, 2025, to clarify the FDA approval covers a new oral pellet formulation of Orladeyo. The U.S. Food and Drug Administration (FDA) expanded the approval of Orladeyo (berotralstat), making it the only oral treatment approved in the U.S. as a prophylactic (preventive) therapy…
Suddenly stopping long-term preventive treatment with danazol — a hormone therapy being phased out in hereditary angioedema (HAE) — caused temporary hepatitis, or an inflammation of the liver, for five adults with HAE in the Netherlands, a new study found. According to the researchers, these findings suggest that…
On-demand treatment with deucrictibant can help resolve swelling attacks in people with all major forms of hereditary angioedema (HAE), according to top-line data from a Phase 3 clinical trial. Pharvaris, deucrictibant’s developer, plans to use the Phase 3 findings to support a U.S. Food and Drug Administration (FDA)…
Short-term preventive treatment with on-demand Berinert (human C1 esterase inhibitor) immediately before delivery helps women with hereditary angioedema (HAE) avoid swelling attacks during childbirth, according to case series and literature review from Japan. About half of pregnant women with HAE experienced more acute attacks during pregnancy, particularly in…
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…
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…
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…
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…
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