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Ionis Pharmaceuticals Announces FDA Approval of DAWNZERA™ for Hereditary Angioedema Treatment

Ionis Pharmaceuticals, Inc. (IONS) | Aug. 21, 2025

By Victor Perez

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Ionis Pharmaceuticals received FDA approval for DAWNZERA™ (donidalorsen) as the first RNA-targeted prophylactic treatment for hereditary angioedema (HAE).

DAWNZERA demonstrated significant reduction in HAE attack rates and offers extended dosing intervals, providing a promising treatment option for patients.

The approval is based on positive Phase 3 trial results showing substantial efficacy and safety of DAWNZERA in managing HAE symptoms.

Groundbreaking Approval

First and only RNA-targeted medicine approved for HAE, offering long dosing intervals and strong efficacy.

Clinical Efficacy

DAWNZERA significantly reduced HAE attack rates by 81% compared to placebo and demonstrated long-term disease control.

Patient Preference

84% of patients preferred DAWNZERA over previous treatments due to better disease control, convenience, and fewer side effects.

  • DAWNZERA's approval marks a significant advancement in HAE treatment options, providing patients with a promising prophylactic option.
  • The Phase 3 study results demonstrate the effectiveness of DAWNZERA in reducing HAE attack rates and improving patient quality of life.

Ionis Pharmaceuticals celebrates the FDA approval of DAWNZERA as a pivotal moment in advancing treatment options for HAE patients, reflecting the company's commitment to innovation and patient care.