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…
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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…
Bradykinin — the molecule involved in most angioedema attacks — also plays a part in a nonallergic type caused by angiotensin-converting enzyme (ACE) inhibitors, including migraine and blood pressure medications, a study found. But Firazyr (icatibant), an inhibitor of…
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,…
Emotional Difficulties May Trigger Stress and Angioedema Flares in Children with HAE, Study Suggests
Children with hereditary angioedema frequently have difficulties recognizing their own emotions (alexithymia), which could be a key source of psychological stress that triggers acute attacks of swelling, an Italian study found. Guidelines recommend that management of these patients should include psychological intervention to ease stress…
Most physicians in Canada say they follow the guidelines for diagnosing and treating hereditary angioedema (HAE), and to have adopted treatments with the C1 esterase inhibitors Berinert, Cinryze, and Haegarda, as well as Firazyr…
Specific Inhibitors Efficiently Treat Acute HAE Attacks in Various Body Locations, Study Reports
Treatment of hereditary angioedema (HAE) with C1-esterase inhibitors (C1-INH) provides efficient relief of acute attacks across different locations of the body, according to a new study. The research was presented at the 2018 American Academy of Allergy Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress, which…
Researchers have developed a new way to distinguish bradykinin-mediated angioedema from histamine-mediated angioedema. This biomarker tool may improve the use of targeted therapies in angioedema patients. The study, “Threshold‐Stimulated Kallikrein Activity Distinguishes Bradykinin‐ From Histamine‐Mediated Angioedema,” was published in Clinical & Experimental Allergy. Angioedema encompasses…
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