FDA Greenlights Groundbreaking RNA-Targeted Treatment for Rare Hereditary Disease
The U.S. Food and Drug Administration (FDA) granted approval to Dawnzera (donidalorsen) on August 21, 2025. This marks a significant milestone as the first and only RNA-targeted prophylactic treatment for Hereditary Angioedema (HAE). Developed by Ionis Pharmaceuticals, Dawnzera represents a significant leap forward in the treatment of this rare genetic disorder.
Dawnzera is a prekallikrein-directed antisense oligonucleotide. It is indicated for prophylaxis to prevent attacks of hereditary angioedema in adult and pediatric patients 12 years of age and older. HAE is a rare genetic condition that causes recurrent episodes of severe swelling in various parts of the body, including the face, throat, and extremities.
This approval comes on the heels of other recent FDA drug approvals in August 2025. These include:
- Tonmya for fibromyalgia treatment
- Papzimeos, an immunotherapy for recurrent respiratory papillomatosis
The FDA has been particularly active this month in approving innovative treatments for rare and underserved medical conditions. The RNA-targeted approach represents a new frontier in precision medicine. It offers patients with hereditary angioedema a preventive treatment option that works at the genetic level to reduce the frequency and severity of attacks.
This approval underscores the FDA’s commitment to fast-tracking access to breakthrough therapies for rare diseases.
Source: https://www.drugs.com/newdrugs.html