Biocom California congratulates Ionis Pharmaceuticals on the U.S. Food and Drug Administration (FDA) approval of DAWNZERA™ (donidalorsen), the first and only RNA-targeted therapy approved for the prevention of hereditary angioedema (HAE) attacks. This milestone represents a transformational advance for patients and a powerful demonstration of California’s leadership in life science innovation.

HAE is a rare and potentially life-threatening genetic disease that causes recurrent swelling attacks across different parts of the body, impacting daily life and, at times, putting patients at risk of airway obstruction. Affecting an estimated 7,000 people in the U.S., HAE has long required better preventive options.

With this approval, DAWNZERA provides the longest dosing option available for HAE patients, with administration every 4 or 8 weeks by autoinjector, strong and durable efficacy, including an 81% reduction in monthly attack rates compared to placebo in Phase 3 studies, and a 94% mean reduction after one year in long-term follow-up and patient-preferred outcomes, with 84% of patients in a switch study favoring DAWNZERA over prior prophylactic therapies, citing better disease control, faster administration, and less injection site discomfort.

This approval marks Ionis’ second independent commercial launch in just nine months, following the approval of TRYNGOLZA® for familial chylomicronemia syndrome (FCS). With two additional potential launches next year, Ionis continues to deliver on its vision of turning pioneering RNA science into life-changing medicines.

Beyond the science, Ionis is also supporting access and care through Ionis Every Step™, a comprehensive suite of patient services designed to provide education, insurance navigation, affordability resources, and digital support tools for people prescribed DAWNZERA.

Biocom California applauds Ionis’ relentless pursuit of innovation, commitment to rare disease communities, and continued contributions to California’s global leadership in biotechnology. DAWNZERA’s approval exemplifies the impact of science translated into real-world solutions, and the collaboration among researchers, patients, and advocates that makes it possible.

Read Ionis’ full press release here.