News

Patients with hereditary angioedema caused by deficiencies in the complement C1-inhibitor protein experience symptomatic relief and remission with the use of on-demand treatments, according to an Italian study. Firazyr (icatibant), an agonist of the bradykinin receptor, led to reduced attack durations compared to plasma derived C1-inhibitor treatments. But the treatment also…

A new gene therapy candidate, ADVM-053, developed by Adverum Biotechnologies, was granted orphan drug status by the U.S. Food and Drug Administration for the treatment of hereditary angioedema (HAE). The FDA’s orphan drug designation program grants orphan status to investigational therapies being developed for the diagnosis or treatment of…

The U.S. Food and Drug Administration has approved Shire‘s Takhzyro (lanadelumab-flyo), a first-of-its-kind antibody injection to prevent attacks of hereditary angioedema (HAE) in patients age 12 and older. The approval follows a priority review granted by FDA to Takhzyro, which helps accelerate the clinical program…

Two signaling pathways — NOTCH2 and NF-kB — are likely involved in both autoimmunity and driving splenic marginal zone lymphoma in patients with acquired angioedema, new research shows. The study, “Splenic marginal zone lymphomas in acquired C1-inhibitor deficiency: clinical and molecular characterization,” was published in the journal…

The U.S. Food and Drug Administration (FDA) has granted fast track status to BCX7353, an investigational therapy being developed by BioCryst Pharmaceuticals, for the prevention of angioedema attacks in patients with hereditary angioedema. That status will support the development of BCX7353, speed its review and, depending on how it performs…

Daily treatment with BCX7353, an investigative oral inhibitor of plasma kallikrein, reduces the rate of attacks in hereditary angioedema (HAE) patients by more than 70%, a Phase 2 study shows. Also, more patients receiving the prophylactic, or preventive, treatment remained without an attack during the study’s four-week duration. The study,…