- In June 2024, BioCryst Pharmaceuticals, Inc. presented new evidence indicating that patients with hereditary angioedema (HAE) who have normal C1-inhibitor levels and function (HAE-nC1-INH) experienced a reduction in monthly attack rates after initiating treatment with oral, once-daily ORLADEYO (berotralstat). The analysis included six patients with HAE-nC1-INH. After six months of treatment with berotralstat, five patients showed a 75 to 100 percent reduction in their HAE attack rate, while one patient, who was also taking tranexamic acid, experienced a 29 percent reduction in attack frequency
- In May 2024, KalVista Pharmaceuticals, Inc. outlined its strategic plans for fiscal year 2025, starting on May 1. These plans include the development of sebetralstat, the company’s investigational oral plasma kallikrein inhibitor for on-demand treatment of hereditary angioedema (HAE). The strategy involves submitting multiple regulatory filings for sebetralstat and preparing for its swift commercialization upon approval
- In January 2024, Ionis announced positive topline results from the Phase 3 OASIS-HAE study of the investigational drug donidalorsen in patients with hereditary angioedema. The trial successfully met its primary endpoint, demonstrating a significant reduction in the rate of angioedema attacks in patients treated with donidalorsen (80 mg) via subcutaneous injection every 4 weeks (Q4W) (p<0.001) or every 8 weeks (Q8W) (p=0.004), compared to placebo. Additionally, donidalorsen achieved statistical significance on all secondary endpoints in the Q4W group and key secondary endpoints in the Q8W group
- In February 2023, Takeda announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for the expanded use of TAKHZYRO (lanadelumab-flyo) for prophylaxis to prevent hereditary angioedema (HAE) attacks in pediatric patients aged 2 to <12 years. Prior to this approval, the only available routine prophylaxis treatments for children aged 6 to <12 required dosing every three to four days, while no approved prophylaxis options existed for children aged 2 to <6. With this approval, TAKHZYRO becomes the first prophylactic treatment for this younger age group
- In December 2022, Takeda introduced Cinryze in India for the treatment of hereditary angioedema. Cinryze is approved for routine prevention (prophylaxis) of angioedema, as well as for the treatment of acute angioedema attacks and pre-procedure prevention. The launch aims to address acute HAE attacks and reduce the frequency of future episodes through prophylactic therapy, ultimately improving the quality of life for individuals with HAE
