- BioCryst Pharmaceuticals' ORLADEYO® (berotralstat) shows significant reduction of hereditary angioedema (HAE) attacks across all age groups.
- The APeX-P trial highlights a decrease in angioedema symptoms in children, from 11% to 4% over 12 weeks.
- ORLADEYO provides consistent safety and efficacy, making it a promising oral prophylactic treatment for HAE patients.
BioCryst Pharmaceuticals (BCRX, Financial) has unveiled compelling new data demonstrating the efficacy of its drug ORLADEYO® (berotralstat) in reducing attacks of hereditary angioedema (HAE) across various age groups. The findings, presented at the 14th C1-inhibitor Deficiency & Angioedema Workshop, underscore the drug's potential as an effective, once-daily oral prophylactic treatment for HAE patients.
The APeX-P trial focused on the impact of ORLADEYO on young children aged 2-11. Over the course of 12 weeks, the percentage of days with angioedema symptoms declined from 11% to 4%. Remarkably, at least 50% of the children involved in the study experienced no HAE attacks each month, highlighting the substantial control ORLADEYO offers.
The Italian expanded access program further supports these findings, showing improvements in disease control with Angioedema Control Test scores rising from 11.5 to 16.0 after six months. Quality of life scores also improved significantly. Meanwhile, the Berolife study demonstrated a decrease in median monthly attack rates in adolescents, dropping from 1.8 to 0.55 attacks per month over 18 months of treatment.
Common adverse events reported were nasopharyngitis, upper respiratory tract infections, and headaches, reinforcing ORLADEYO's consistent safety profile across all studies. The oral administration of ORLADEYO represents a shift from traditional injectable treatments, potentially increasing adherence and improving daily life for HAE patients.
