News

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

Medications that lower cholesterol levels, collectively known as statins, can in rare instances trigger angioedema episodes that are self-resolving if the patients stops using the statin, a case study reports. The study describes a side effect of Crestor (rosuvastatin), but its researchers caution that other, chemically-related statins could also…

Although the symptoms of hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE) present in childhood, most patients are diagnosed in adulthood, highlighting a need for increased awareness of C1-INH-HAE among pediatricians, according to a new study. The research, “Pediatricians diagnosed few patients with childhood-presented hereditary angioedema: Icatibant Outcome Survey…

Patients in Germany treat acute hereditary angioedema attacks with Firazyr (icatibant) earlier and have fewer severe attacks than do patients in 11 other countries where Firazyr is also approved, a retrospective study shows. The disparity is thought to arise from a easier access to Firazyr in Germany and a public healthcare…

The oral treatment candidate BCX7353 eases hereditary angioedema (HAE) attacks, reduces the occurrence of symptoms, and helps decrease the use of rescue medications, according to BioCryst Pharmaceuticals’ early Phase 2 trial results. The data also showed that the investigational therapy was safe and well-tolerated. BCX7353 is an oral…

Delayed angioedema can be triggered when a person eats red meat following a tick bite, according to a case report from Jersey Shore University Medical Center in the U.S. The study, “Diagnosing Meat Allergy After Tick Bite Without Delay,” was published in the Journal of the American…

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…