News

Swelling attacks affecting the airway usually occur in hereditary angioedema patients between the ages of 20 and 30 — and are more frequent among smokers, a study shows. However, the frequency of attacks was higher in people with acquired angioedema, a type of the chronic disorder in which…

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…

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…

Switching from under-the-skin injections to oral pills of Orladeyo (berotralstat), a preventive treatment, can be done without the need to taper prior therapy or go through complex regimens, according to a case study of a patient and her mother, both of whom have hereditary angioedema (HAE). The report,…

Over a third of hereditary angioedema patients continue to experience an unpredictable rate of annual attacks, including those on long-term preventive therapy, a retrospective analysis shows. These findings highlight the variability of the disease and support the need for constant vigilance by patients and their doctors. The study, “…

Effexor XR (venlafaxine), a medication normally used to treat depression and anxiety, might have triggered acute allergic angioedema in an 81-year-old woman, a case study reports. Her symptoms, including tongue swelling, nausea, vomiting, and headaches, eased after she went off Effexor XR and was treated with diphenhydramine, an anti-allergy…

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…