Author Archives: Marisa Wexler MS

People with hereditary angioedema (HAE) commonly have histories of cancer, which raises the possibility that it might predispose a person to develop cancer. These findings were reported in the study “Malignancy and immune disorders in patients with hereditary angioedema,” published in the journal Allergy, Asthma…

Pharming Group and Sanofi have renewed an agreement for the manufacturing of Ruconest (conestat alfa), Pharming’s approved treatment for swelling attacks in people with hereditary angioedema (HAE). The two companies have been working together for more than a decade, and now have agreed to extend the collaboration…

The first participant has been dosed in a Phase 1/2 clinical trial that is testing Intellia Therapeutics’ gene-editing therapy, NTLA-2002, in people with hereditary angioedema (HAE). “With the progress of our first-in-human clinical study evaluating NTLA-2002 for people living with HAE, we look forward to beginning clinical testing as…

Starting on Dec. 1, people with hereditary angioedema (HAE) in Australia will have access to Takhzyro (lanadelumab), an under-the-skin injection to prevent HAE swelling attacks, through Australia’s Pharmaceutical Benefits Scheme (PBS). “Without this PBS subsidy, Australian patients are paying around $241,000 per year for this treatment,” Greg Hunt, Australia’s…

Pharvaris is actively recruiting people with hereditary angioedema (HAE) types 1 or 2 for two clinical trials testing its investigational oral therapy PHVS416 as either an on-demand or prophylactic (preventive) treatment for swelling attacks. Results from both studies are expected next year. The Phase 2 trial RAPIDe-1…

Takhzyro (lanadelumab), an approved under-the-skin injection therapy to prevent swelling attacks in people with hereditary angioedema (HAE), will soon be available at a lower cost to patients in Poland and, likely, those in Australia. Poland’s Minister of Health approved access to and reimbursement for Takhzyro for about 50…

CAMP4 Therapeutics’ platform that targets a class of RNA known as regulatory RNAs, or regRNAs, can improve the activity of the gene that is defective in most cases of hereditary angioedema (HAE). That is according to new preclinical data shared by scientists from CAMP4 at the 17th Annual…

Orladeyo (berotralstat), a daily oral therapy to prevent swelling attacks in people with hereditary angioedema (HAE), is now available in England, Wales, and Northern Ireland for eligible patients ages 12 and older. That authorization came as the U.K.’s National Institute for Health and Care Excellence (NICE) recommended Orladeyo —…

The U.S. Food and Drug Administration (FDA) has lifted the hold it had placed on KalVista Pharmaceuticals’ Phase 2 KOMPLETE trial of KVD824 as a treatment to prevent swelling attacks in people with hereditary angioedema (HAE). KalVista said it is working closely with study investigators and clinical sites so…

The prices of three approved preventive treatments — Cinryze, Haegarda, and Takhzyro (lanadelumab) — for hereditary angioedema (HAE) must be reduced by more than half for these medications to be cost-effective. Those are the conclusions of a new report published by the Institute for Clinical and Economic Review,…