US biotechnology company BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) on Friday presented new data demonstrating the effectiveness of ORLADEYO (berotralstat) in reducing hereditary angioedema (HAE) symptoms across paediatric, adolescent, and adult populations.

Interim results from the ongoing APeX-P trial in children aged 2 to 11 showed a drop in symptomatic days from 11% to 4% over 12 weeks, with sustained improvement up to 48 weeks. At least half of the children experienced monthly attack-free periods while on treatment.

In Italy's expanded access program, berotralstat use correlated with improved Angioedema Control Test and quality-of-life scores through six months. Patients experienced fewer HAE attacks, and all eligible participants transitioned to commercial ORLADEYO following the program's closure.

Data from the French Berolife study further confirmed long-term benefits in adolescents, with median monthly attack rates decreasing from 2.25 to 0.55 over six months, sustained through 18 months.

Across studies, berotralstat was well-tolerated, with safety profile consistent with that of previous trials. Reported side effects included nasopharyngitis, upper respiratory infections, and headaches.

BioCryst emphasised ORLADEYO's once-daily oral dosing and consistent efficacy, positioning it as a reliable prophylactic treatment for HAE at all life stages.