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Jun 09, 2026
ADARx will present onvuzosiran Phase 1/2 data and Phase 3 STOP-HAE design at AAAAI and EAACI 2026. The investigational siRNA for hereditary angioedema has FDA Orphan Drug Designation.
Mar 15, 2026
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Mar 11, 2026
Phase 2 data for BW-20805 showed up to 100% reduction in HAE attacks with dosing intervals up to 6 months, while onvuzosiran (ADX-324) advances to Phase 3 trials with semi-annual dosing targeting prekallikrein.
Feb 28, 2026
The FDA removed the clinical hold on Intellia Therapeutics' MAGNITUDE-2 Phase 3 trial of nexiguran ziclumeran for ATTRv-PN, allowing patient enrollment and dosing to resume with enhanced safety monitoring.
