Ionis' DAWNZERA Gains CHMP Backing For Approval In Europe For Hereditary Angioedema

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(RTTNews) - Ionis Pharmaceuticals Inc. (IONS) announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency has adopted a positive opinion recommending approval of DAWNZERA for the routine prevention of recurrent attacks of hereditary angioedema in adults and adolescents aged 12 years and older.

Hereditary angioedema (HAE) is a rare, potentially life-threatening genetic condition characterized by recurrent episodes of severe swelling in various parts of the body, including the hands, feet, stomach, face, and throat. The condition is estimated to affect about one in 50,000 people worldwide, underscoring the need for effective prevention therapies.

DAWNZERA works by inhibiting plasma prekallikrein (PKK), a protein that plays an important role in activating inflammatory mediators associated with acute attacks of HAE.

The CHMP recommendation is based on robust Phase 3 data from the OASIS-HAE and OASISplus trials, which demonstrated significant reductions in attack frequency.

The pivotal OASIS-HAE and OASISplus trials enrolled adults and adolescents aged 12 years and older with recurrent HAE attacks. These studies evaluated DAWNZERA's efficacy and safety, including its use via self-administration with an autoinjector.

Results showed that patients experienced a significant reduction in attack frequency across multiple measures of disease activity, with benefits sustained over time.

Safety data from the trial indicated that DAWNZERA was generally well tolerated.

DAWNZERA was approved in the U.S. in August 2025 for prophylaxis to prevent attacks of HAE in both adult and paediatric patients aged 12 years and above, becoming the first and only RNA-targeted prophylactic treatment for hereditary angioedema.

With FDA approval already secured and CHMP's positive opinion now referred to the European Commission for a final decision expected in the first quarter of 2026, DAWNZERA is positioned to become a transformative therapy for patients living with HAE.

IONS has traded in the range of $23.95 to $76.78 over the past year. The stock closed Friday's (November 14, 2025) trading at $71.55, up 2.45%.