News

Japanese researchers have identified a new mutation in the SERPING1 gene linked to hereditary angioedema (HAE) in a 27-year-old woman with a history of recurrent swelling attacks. Her case was described in a report, “Hereditary angioedema with a novel mutation, c.1481G>C, in the SERPING1 gene,” published…

The European Commission has approved BioCryst Pharmaceuticals’ Orladeyo (berotralstat) as the first oral, daily therapy to prevent swelling attacks in adults and adolescents, ages 12 and older, with hereditary angioedema (HAE). An oral preventive therapy should be easier to take than standard under-the-skin and into-the-vein alternatives, and as…

This year, May 16 marks the 10th anniversary of HAE Awareness Day, set aside annually to call global attention to hereditary angioedema (HAE), a chronic disorder characterized by sudden swelling attacks in the skin’s deeper layers. To recognize the work the HAE community has done so far, and to…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The U.S. Food and Drug Administration (FDA) has placed a hold on KalVista Pharmaceuticals’ proposed Phase 2 trial of KVD824 — which the developer had expected to launch by the end of June.  The trial is intended to test…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Japan’s National Health Insurance System (NHI) has approved the addition of Orladeyo (berotralstat), an oral preventive treatment for hereditary angioedema (HAE) attacks, to its drug price list. This means that the NHI will reimburse hospitals and pharmacies prescribing Orladeyo to a certain amount under national health insurance programs.

Newer preventive therapies for hereditary angioedema (HAE) lead to a better quality of life and fewer attacks than on-demand treatments, according to a large patient survey study. The study, “Assessing the cost and quality-of-life impact of on-demand‐only medications for adults with hereditary angioedema,” carried out by…

Takhzyro (lanadelumab) is more effective than Cinryze at preventing swelling attacks in people with hereditary angioedema (HAE), a new study indicates. Analyses suggested that treatment with Takhzyro reduced the rates of HAE attack by 46–73% as compared with Cinryze in an indirect comparison of previous studies. Titled…

The experimental therapy IONIS-PKK-LRx can significantly lower the number of monthly swelling attacks in patients with hereditary angioedema (HAE) type 1 and 2, according to top-line data from an ongoing Phase 2 trial. Newly announced data show the study (NCT04030598), sponsored by therapy developer Ionis Pharmaceuticals,…