Marisa Wexler, MS, senior science writer —

Preventive treatment with oral berotralstat (BCX7353) significantly lowers the frequency of swelling attacks in people with hereditary angioedema (HAE), newly published results from the APeX-2 clinical trial demonstrate. The study, “Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: a randomized, double-blind, placebo-controlled phase 3…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

The first documented case of a hereditary angioedema (HAE) attack in an unborn child — whose symptoms resolved after treating the mother with the recombinant human C1-inhibitor (rhC1-INH) therapy, Ruconest — has been reported. The case report, “Hereditary Angioedema Attack in Utero and Treatment of the Mother…

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…