News

Half the patients with hereditary angioedema (HAE) treated with Takhzyro (lanadelumab) for at least a year prolonged their dosing interval and used significantly less on-demand treatments, a real-world study found. Study findings were detailed in a clinical letter titled, “Real World treatment patterns of hereditary angioedema with…

A single infusion of the investigational gene therapy NTLA-2002 was well tolerated at two different doses and reduced the number of swelling attacks in people with hereditary angioedema (HAE) by more than 90%, according to interim results from the Phase 1 portion of a Phase 1/2 trial. The…

Atorvastatin, a cholesterol-lowering medication, has been linked to angioedema for the first time in a recent case report, highlighting the need for comprehensive evaluation of all medications taken by patients, regardless of their medical history. “Statins cannot be disregarded as the primary or contributing cause of angioedema,” researchers…

The European Patent Office has granted patent protection to Verseon covering the company’s experimental plasma kallikrein inhibitors (PKIs). Two of these investigational medications, called VE-4666 and VE-4062, are currently in preclinical development as potential treatments of hereditary angioedema (HAE). “We’re very pleased with the European Patent Office’s…

NTLA-2002, an experimental gene-editing therapy designed to prevent swelling attacks in people with hereditary angioedema (HAE), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). This designation is given to investigational treatments that have the potential to improve care for rare diseases, defined as…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on trials testing the investigational therapy PHA121 (PHA-022121) in people with hereditary angioedema (HAE). The hold is “based on [a] review of nonclinical data,” Pharvaris, the therapy’s developer, said in a press release. According to…

KalVista Pharmaceuticals has launched a two-year open-label extension (OLE) study, dubbed KONFIDENT-S, to test the long-term safety of oral sebetralstat (formerly known as KVD900) as an on-demand treatment for swelling attacks caused by hereditary angioedema (HAE). The study follows the ongoing Phase 3 KONFIDENT clinical trial, involving adults and…

Garadacimab, an investigational preventive treatment for hereditary angioedema (HAE), reduced the number of attacks for up to six months in patients 12 and older, according to top-line data from a Phase 3 trial. According to CSL Behring, the therapy’s developer, the study, called VANGUARD (NCT04656418), not only…

Regulatory authorities in Saudi Arabia have approved daily Orladeyo (berotralstat) to prevent hereditary angioedema (HAE) attacks in adult and adolescent patients, ages 12 and older. “There is a significant need for new treatment options for HAE in Saudi Arabia,” Charlie Gayer, chief commercial officer of BioCryst Pharmaceuticals,…

Off-label prescription of certain medications indicated for hereditary angioedema (HAE) with C1 deficiency to people with other types of angioedema is common, a team of French researchers found. Fewer than 20% of prescriptions for these medications, namely Berinert, Cinryze, Ruconest (conestat alfa), and Firazyr…