Patricia Inácio, PhD, science writer —

The U.S. Food and Drug Administration (FDA) agreed to review BioCryst Pharmaceuticals‘ application requesting approval of oral berotralstat as a once-daily treatment to prevent swelling attacks in people with hereditary angioedema (HAE). ByoCryst submitted the new drug application (NDA) to the FDA in December 2019, and the…

Haegarda, a C1-INH replacement medication by CSL Behring, is now available in the Canadian province of Quebec for the preventive treatment of swelling attacks in adults and adolescents with hereditary angioedema (HAE), the company announced. “The availability of a new treatment option to help health care professionals…

BioCryst Pharmaceuticals has submitted an application to the Japanese regulatory authority asking that oral berotralstat (BCX7353) be approved for the prevention of swelling attacks in people with hereditary angioedema (HAE). The new drug application (NDA), submitted to the Pharmaceuticals and Medical Devices Agency (PMDA) in…

The European Medicines Agency (EMA) has approved a type II variation for a new facility to produce Ruconest (conestat alfa), Pharming’s lead therapy for acute attacks in hereditary angioedema (HAE). The new facility is expected to be fully operational by 2021 and to increase the…

Results of the Phase 2 clinical trial testing KalVista Pharmaceuticals‘ oral investigational therapy KVD900 as an on-demand treatment for hereditary angioedema (HAE) attacks will be announced by August. The company also announced it has selected KVD824 as an oral preventive therapy for HAE. A Phase…

Treatment with Takhzyro (lanadelumab) effectively reduces the number of attacks in patients with hereditary angioedema even in early phases of treatment, new analysis of data from the Phase 3 HELP study shows. Most recent trial results were shared in a poster, “Lanadelumab demonstrates rapid and sustained prevention 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…

Delays in the diagnosis of hereditary angioedema causes family caregivers to feel fear, anxiety, and uncertainty, according to a study focused on better understanding what these caregivers go through during the diagnosis process. Giving attention and credibility to family caregivers could not only improve understanding and awareness of the disease but…

KVD900, a potential new approach for treating hereditary angioedema, prevents the breakdown of kininogen and subsequent buildup of bradykinin — the underlying cause of the disease, KalVista Pharmaceuticals announced. The company confirmed this mechanism of action using a new test that measures the amounts of high molecular weight kininogen in the plasma. The…

Comparing the genetic profile of patients with hereditary angioedema during remission and in acute attacks, researchers identified two genes that seem to participate in edema formation. The genes – uPAR and ADM – are involved in vessel dilation and inflammation, and may be potential therapeutic targets for the disease. The…