Ionis Pharmaceuticals(US:IONS) RTTNews·2025-11-17 06:44Core Viewpoint - Ionis Pharmaceuticals Inc. has received a positive opinion from the CHMP recommending the approval of DAWNZERA for the prevention of recurrent attacks of hereditary angioedema in adults and adolescents aged 12 years and older [1][6]. Group 1: Hereditary Angioedema (HAE) - HAE is a rare genetic condition affecting approximately one in 50,000 people globally, characterized by severe swelling in various body parts [2]. - The condition highlights the need for effective prevention therapies due to its potentially life-threatening nature [2]. Group 2: DAWNZERA Mechanism and Trials - DAWNZERA functions by inhibiting plasma prekallikrein (PKK), a protein involved in activating inflammatory mediators linked to HAE attacks [3]. - The CHMP's recommendation is based on Phase 3 data from the OASIS-HAE and OASISplus trials, which showed significant reductions in attack frequency [3][4]. - The pivotal trials included adults and adolescents aged 12 years and older, demonstrating DAWNZERA's efficacy and safety, including self-administration via an autoinjector [4]. Group 3: Safety and Approval Status - Safety data indicated that DAWNZERA was generally well tolerated during trials [5]. - DAWNZERA was previously approved in the U.S. in August 2025, becoming the first RNA-targeted prophylactic treatment for HAE [5]. - The positive opinion from CHMP is now referred to the European Commission, with a final decision expected in the first quarter of 2026, positioning DAWNZERA as a transformative therapy for HAE patients [6]. Group 4: Stock Performance - Ionis Pharmaceuticals' stock has traded between $23.95 and $76.78 over the past year, closing at $71.55 on November 14, 2025, reflecting a 2.45% increase [6].