News

Single-dose PHA121 Shows Promise in Trial as Treatment for HAE

A single dose of the extended-release formulation of PHA121 — an investigational molecule under development for the prevention and treatment of swelling attacks associated with hereditary angioedema (HAE) — led to sustained, therapeutic levels of the medication in the bloodstream of participants. That’s according to promising data from…

Most HAE Patients Attack-free After Gene-editing Therapy NTLA-2002

Most hereditary angioedema (HAE) patients in a Phase 1/2 clinical trial have gone months without a swelling attack following a single infusion of the experimental gene-editing therapy NTLA-2002. The remaining patients haven’t yet reached the pre-specified 16-week follow-up period for their attack rates to be analyzed, but their comparable…

Orladeyo Reduces Swelling Rates in Patients Who Switched Therapies

People with hereditary angioedema (HAE) who switched to Orladeyo (berotralstat) from other preventive treatments generally experience a reduction in swelling attacks. That is according to real-world data presented by BioCryst Pharmaceuticals, Orladeyo’s developer, during the 2022 Annual Scientific Meeting of the American College of Allergy, Asthma…

High Immune Eosinophil Counts Seen in Rare Case of Angioedema

A Japanese woman was diagnosed with a rare case of non-episodic angioedema associated with eosinophilia (NEAE), with remarkably high levels of immune eosinophil cells in her bloodstream, a recent case report describes. Swelling in her legs and feet, and associated weight gain, were successfully treated with the anti-inflammatory…

ACE Inhibitor Triggers Small Bowel Angioedema in Woman, 34

An ACE inhibitor, a common medication to control blood pressure, triggered small bowel angioedema in a 34-year-old woman, according to a case report. “Given the high prevalence of ACEi [ACE inhibitor] prescriptions, it is an important mimic to consider for other chronic intermittent intestinal diseases,” because stopping their use…

Phase 1 Trial Testing Fast-melting Sebetralstat as HAE Treatment

An orally disintegrating tablet (ODT) formulation of sebetralstat, an investigational on-demand treatment for swelling attacks in hereditary angioedema (HAE) patients, is processed by the body in a similar way to its film-coated tablets, according to data from a Phase 1 clinical trial. Researchers are investigating these ODT tablets…

Pregnancy Not Linked to Worse Disease in Women With HAE: Study

Women with hereditary angioedema (HAE) who have been pregnant multiple times did not experience outcomes of significant disease worsening during pregnancy, a study in Turkey found. A majority did report a greater frequency of HAE attacks during pregnancy, but this was not statistically significant, according to researchers. In patients…

Takhyrzo Found to Be Effective HAE Treatment Across Minority Groups

The efficacy and safety of Takhzyro (lanadelumab) in preventing hereditary angioedema attacks are similar across different racial and ethnic groups, according to an analysis of data from a Phase 3 trial and its open-label extension study. However, with few patients from minorities participating in clinical trials, conclusions are…