A Potential First-in-Class Oral Therapy for Hereditary Angioedema
We assign a 69.2% overall probability that this new therapy will gain the FDA’s nod for on-demand HAE attacks.
Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic condition marked by sudden bouts of severe swelling. In February 2024, a fast-rising biotech developer delivered late-stage clinical data showing its oral therapy met both primary and secondary endpoints in treating on-demand attacks of HAE. If approved, it would stand out as the first oral treatment specifically indicated for on-demand use, rather than just prevention. Notably, the phase III trial data showed a significant reduction in the time to symptom relief at both the 300 mg and 600 mg doses, pushing anticipation for an upcoming regulatory decision even higher.
We assign a 69.2% overall probability that this new therapy will gain the FDA’s nod for on-demand HAE attacks.
MACE Scores are based on:
Market Demand
Adoption by payers and key opinion leaders (KOLs)
Clinical progress
Experience and capital.