News

BioCryst Pharmaceuticals has received funds worth $350 million that will be used in part to support the global launch of Orladeyo (berotralstat), an oral therapy to prevent hereditary angioedema (HAE) swelling attacks. The money came through various transactions with Royalty Pharma, a global buyer of pharmaceutical royalties, and…

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…

Ionis Pharmaceuticals has launched a registrational Phase 3 clinical trial to evaluate the safety and effectiveness of its investigational therapy donidalorsen, formerly known as IONIS-PKK-LRx, in people with hereditary angioedema (HAE) types 1 and 2. While the trial, called OASIS-HAE, may already be recruiting, information on recruitment status…

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…

KalVista Pharmaceuticals is planning to launch a Phase 3 trial of KVD900, an investigational oral and on-demand treatment for adults with hereditary angioedema (HAE), with dosing expected to begin in early 2022. “Our development team is finalizing the Phase 3 trial protocol and preparing for study initiation,…

Orladeyo (berotralstat) reduced the frequency of swelling attacks in people with hereditary angioedema (HAE) and improved their quality of life over the course of one year, according to the latest findings from the long-term APeX-S trial. The approved oral medication also was able to sustainably lower HAE attack…

Finger-prick blood samples taken at home could help to provide a simple and convenient way to predict acute hereditary angioedema (HAE) attacks, preliminary results from the Hereditary Angioedema Kininogen Assay (HAEKA) study in Germany suggest. Samples collected during attacks over the study’s course are expected to lead to the discovery…

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…

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…