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DAWNZERA™ (donidalorsen) receives positive opinion from CHMP, recommended for approval in EU for hereditary angioedema (HAE)
Recommendation based on breadth of clinical evidence demonstrating significant HAE attack rate reduction with DAWNZERA European Commission decision expected
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nRecommendation based on breadth of clinical evidence demonstrating significant HAE attack rate reduction with DAWNZERA\n\n\n\nEuropean Commission decision expected in Q1 2026\n\n\n\n CARLSBAD, Calif.--(BUSINESS WIRE)--\nIonis Pharmaceuticals, Inc. (Nasdaq: IONS) and Otsuka Pharmaceutical Co., Ltd. (Otsuka) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of DAWNZERA™ (donidalorsen) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. The positive opinion is now referred to the European Commission (EC) for an approval decision.\n\n\n“We believe the positive opinion from the CHMP reflects the robust clinical evidence supporting DAWNZERA and its potential to deliver a meaningful benefit to people living with HAE in the EU,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “This advancement is made possible by the shared dedication of the teams at Ionis and Otsuka to bring DAWNZERA to as many people living with HAE as possible.”\n\n\nThe CHMP opinion was based on positive results from the Phase 3 OASIS-HAE and OASISplus studies, in which DAWNZERA demonstrated positive results across multiple measures of disease including significant and sustained reduction in mean monthly HAE attack rate, including when self-administered via autoinjector.\n\n\nHAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect about 1 in 50,000 people worldwide.\n\n\nDAWNZERA was approved by the U.S. Food and Drug Administration in August 2025 for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older. Otsuka holds exclusive rights to bring donidalorsen to patients across Europe and Asia Pacific.\n\n\n“We are encouraged by the CHMP’s positive opinion, a key milestone in advancing access to potentially life-changing treatments for the HAE community,” said Andy Hodge, president and CEO, Otsuka Pharmaceutical Europe Ltd. “We look forward to the forthcoming decision from the European Commission and remain committed to addressing the unmet needs of th...