FDA Greenlights Dawnzera: A Groundbreaking RNA-Targeted Treatment for Hereditary Angioedema
The FDA has recently given its stamp of approval to Dawnzera (donidalorsen). This marks a significant milestone as it is the first-ever RNA-targeted prophylactic treatment for hereditary angioedema. The innovative treatment, developed by Ionis Pharmaceuticals, was officially approved on August 21, 2025, and is suitable for both adult and pediatric patients aged 12 years and older.
Hereditary angioedema is a rare genetic disorder that triggers severe swelling episodes. These episodes can become life-threatening when they affect the airways. Dawnzera functions as a prekallikrein-directed antisense oligonucleotide, tackling the root cause of attacks at the RNA level, rather than merely alleviating symptoms.
“This represents a significant advancement in precision medicine,” medical experts have noted. The approval of Dawnzera coincides with several other major FDA decisions in August 2025. These include the first disease-modifying therapy for bronchiectasis and a novel treatment for fibromyalgia. The pioneering approach of targeting RNA paves the way for new possibilities in treating other rare genetic conditions.