Hereditary Angioedema (HAE) is a rare but serious genetic disorder that can cause unpredictable, recurrent episodes of severe swelling in various parts of the body, including the hands, feet, face, intestinal tract, and airway. Though rare — with an estimated prevalence of 1 in 50,000 people — HAE can be life-threatening, particularly when it affects the throat and restricts breathing.
Fortunately, biotech and pharmaceutical companies are making major strides in developing targeted therapies for this condition. In this blog, we’ll explore what HAE is, current treatment strategies, and the top companies with promising products in the development pipeline.
What is Hereditary Angioedema?
HAE is most often caused by a deficiency or dysfunction of C1 esterase inhibitor (C1-INH), a protein that helps regulate inflammation and vascular permeability. There are three recognized types:
- Type I HAE: Characterized by low levels of functional C1-INH (about 85% of cases).
- Type II HAE: Normal or elevated levels of non-functional C1-INH.
- HAE with normal C1-INH (nC1-INH): Formerly known as Type III; typically associated with mutations in other genes like FXII.
HAE attacks can be triggered by stress, trauma, infection, hormonal changes, or even seemingly random factors, and can last from several hours to a few days.
Current Treatments Overview
Treatment approaches include:
- Acute attack treatment: On-demand therapies to control swelling during attacks.
- Prophylactic treatment: Long-term options to prevent attacks altogether.
Therapies target different parts of the kallikrein-kinin cascade — primarily focused on bradykinin, the molecule responsible for swelling in HAE.
Top companies making waves in the HAE:
Key Emerging Companies in the HAE Pipeline
Below are some of the major players leading research and development in HAE:
Market Outlook
The global HAE treatment market is expected to grow rapidly, projected to exceed $4 billion by 2028, driven by:
- Increased diagnosis rates
- Patient preference for oral and less invasive therapies
- Ongoing innovation and pipeline diversification
The Future of HAE Treatment
Key trends shaping the future:
Oral convenience (especially for young and active patients)
Fewer injections, less frequent dosing
RNA and gene therapies as potential long-term solutions
Competitive pricing and payer pressure to deliver cost-effective outcomes
As companies race to develop the most convenient, effective, and safe treatments, HAE is becoming a model for how rare disease innovation can improve lives through biotech.
