Marisa Wexler, MS,  senior science writer—

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

Antisense oligonucleotides (ASOs) being developed by Ionis Pharmaceuticals may reduce the frequency of attacks in people with hereditary angioedema (HAE), a small study suggests. The study, “Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema,” funded by Amsterdam UMC, was published in The New England…

BioCryst Pharmaceuticals is prepared to launch Orladeyo (berotralstat), an oral treatment for hereditary angioedema (HAE), by the end of the year if the medication is approved, the company has announced. “We are currently in an exciting transformation from a company primarily focused on R&D [research and development]…

KalVista Pharmaceuticals anticipates beginning a Phase 2 clinical trial in the second half of this year to test KVD824, an investigational preventive treatment for hereditary angioedema (HAE). In addition, the results of a Phase 2 clinical trial of KVD900, being investigated as an on-demand treatment for…

Cipla has received final approval from the U.S. Food and Drug Administration (FDA) for a generic version of Firazyr (icatibant injection), an approved treatment for acute attacks in adults with hereditary angioedema (HAE). The company announced the FDA approval of its abbreviated new drug application for icatibant…

Ruconest, a recombinant human form of the C1 inhibitor protein, is more cost-effective than other on-demand therapies at treating attacks in people with hereditary angioedema, a new study suggests. The study, “Modeling Cost-Effectiveness of On-Demand Treatment for Hereditary Angioedema Attacks,” was published in the …

New clinical trial data show that BioCryst Pharmaceuticals‘ investigational therapy for hereditary angioedema (HAE), berotralstat, reduces the frequency of HAE attacks and improves patients’ quality of life through 48 weeks of treatment. The data were presented at the European Academy of Allergy and Clinical Immunology Digital Congress. HAE…

The European Commission has approved Pharming‘s Ruconest (conestat alfa) for the treatment of acute swelling episodes in young children with hereditary angioedema (HAE). This approval expands the age range for Ruconest, which previously was approved to treat adults and adolescents with HAE in the European Union…

Mutations in the KCNMA1 gene may predispose certain people to angioedema caused by blood pressure medications, a study suggests. The study, “Genome-wide association study of angioedema induced by angiotensin-converting enzyme inhibitor and angiotensin receptor blocker treatment,” was published in The Pharmacogenomics Journal. Angiotensin-converting enzyme inhibitors…

A new diagnostic test for hereditary angioedema (HAE) can measure the activity of C1-inhibitor (C1-INH) in a drop of dried blood, and may be easy enough to use at point-of-care medical centers. The test was described in Clinica Chimica Acta, in the study “A…