BioCryst announces positive results from APeX-P trial for Orladeyo (berotralstat) in pediatric patients with hereditary angioedema aged 2 to <12 years
BioCryst Pharmaceuticals announced positive results from an interim analysis of the ongoing APeX-P clinical trial evaluating an oral granule formulation of once-daily Orladeyo (berotralstat) in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.
Dr Helen Thackray, Chief Research and Development Officer of BioCryst, said: “We are pleased to share results from APeX-P, the largest trial to date evaluating a prophylactic therapy for HAE in patients 2 to <12 years old, which will be presented later this week in a late-breaking abstract at the AAAAI / WAO Joint Congress. Importantly, the data show the oral granule formulation of Orladeyo to be safe and well tolerated in the trial, with early and sustained reductions in monthly attack rates. We remain on track to submit our New Drug Application to the FDA this year, and we look forward to addressing a significant unmet need for children with HAE and their families.”
HAE Attack Rates in Pediatric Patients 2 to <12 Years of Age with Prophylactic Berotralstat: Results from Interim Analysis of APeX-P; Poster #L55
- Orladeyo was safe and well tolerated in APeX-P, with no new safety signals identified. Adverse events (AEs) were similar across all ages and weights.
- Orladeyo resulted in early and sustained reductions in monthly attack rates. The median (range) and mean (±SEM) monthly attack rates in the standard-of-care period were 0.96 (0–5.0) and 1.5 (±0.2) attacks/month, respectively. After one month of taking Orladeyo, median and mean monthly attack rates dropped to 0 (0-4.0) and 0.5 (±0.2), and the median monthly attack rate remained at 0 through month 12 (month 12 range: 0-1.7); the mean monthly attack rate at month 12 was 0.3 (±0.1).
- Eighty-three percent of participants experienced symptom onset before six years of age and 90 percent were diagnosed with HAE in the same timeframe.
(Source: BioCryst)