News

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…

A class of medications used to treat diabetes is associated with the occurrence of angioedema, particularly among older adults, a study has found. The use of these medications, called dipeptidyl peptidase-4 inhibitors (DPP-4Is), was linked to the condition in some groups even when patients were not also using a…

Swelling in the hands and feet, as well as elevated blood levels of eosinophils — a type of immune cell — led clinicians to diagnose a Japanese woman with non-episodic angioedema associated with eosinophilia (NEAE), as described in a case report. Distinguishing NEAE from similar conditions is important, the…

Astria Therapeutics has launched a Phase 1a trial in healthy adults to test STAR-0215, an investigational antibody-based therapy designed to prevent swelling attacks in people with hereditary angioedema (HAE), with dosing once every three months or longer. The launch came soon after the U.S. Food and Drug Administration…

Protease-activated receptor 1 (PAR1), a protein involved in the regulation of blood vessel permeability or leakage, may contribute to hereditary angioedema (HAE) attacks. That is the preliminary conclusion of a study reporting evidence suggesting that PAR1 was highly activated in the throat tissue of a woman with HAE type…