The U.S. Food and Drug Administration (FDA) has approved a groundbreaking treatment for hereditary angioedema (HAE), a rare genetic disorder that causes sudden, painful swelling in various body parts.
The drug, garadacimab (brand name Andembry), is the first monthly preventive therapy for HAE in adults and children aged 12 and older. Developed by CSL Behring, it offers a new option for managing a condition that affects roughly 1 in 50,000 people and can be life-threatening if swelling blocks the airway.
How HAE Works
HAE occurs when a genetic defect disrupts the C1 inhibitor protein, which normally controls inflammation. Without it, patients experience severe swelling in the face, throat, abdomen, or limbs. Abdominal attacks can cause extreme pain, nausea, and vomiting, while throat swelling can lead to suffocation without emergency care.
Clinical Trial Results
The FDA approval was based on a six-month trial involving HAE patients. Participants received either:
- A 400mg starting dose of Andembry, followed by 200mg monthly injections, or
- A placebo.
Results showed:
- 62% of patients had no attacks during treatment.
- Attacks dropped by over 99% for many patients, with an average 89% reduction compared to placebo.
- Emergency treatments decreased by 99%, and moderate-to-severe attacks fell by 90%.
An ongoing study also confirmed long-term safety and sustained effectiveness.
How Andembry Works Differently
Andembry is the first drug to target Factor XIIa, a protein that triggers swelling in HAE patients.
“This is a major advancement,” said Dr. Tim Craig, a Penn State University professor involved in the research. “Instead of treating swelling after it starts, we can now block it at the source.”
Dosing and Side Effects
The treatment is administered via a simple under-the-skin autoinjector, allowing patients or caregivers to use it at home. Common side effects include:
- Sore throat, runny nose
- Abdominal pain
- Mild injection site reactions (redness, itching, bruising)
CSL Behring expects Andembry to be available by late June 2025, offering new hope for HAE patients.
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