News

The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing approval to BioCryst Pharmaceuticals’ Orladeyo (berotralstat), as the first oral and daily therapy to prevent acute attacks of hereditary angioedema (HAE) in individuals ages 12 and older. “HAE UK welcomes the decision from the MHRA…

Firazyr (icatibant), when used off-label to treat acute non-hereditary angioedema, can elicit different treatment responses, and its use — and best use — needs to be better understood by doctors treating attacks in this patient group, a real-world study from Australia reports. Biomarkers that could more easily distinguish…

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…