APeX-P Trial of Orladeyo Now Enrolling Children 2–11 With HAE
Phase 3 study testing approved therapy's safety, efficacy in youngsters
Enrollment has begun in APeX-P, a Phase 3 trial designed to evaluate the effects of Orladeyo (berotralstat) in up to 20 children, ages 2–11, with hereditary angioedema (HAE).
The open-label study, sponsored by BioCryst Pharmaceuticals, will provide data on Orladeyo’s safety, pharmacological properties, and ability to prevent HAE attacks in young children.
The trial is now recruiting youngsters with HAE at sites in Canada, Austria, France, and Spain. Locations in Germany, Israel, Italy, Poland, Romania, and the U.K., as well as an additional site in France, are expected to open soon.
Following APeX-P’s completion, BioCryst plans to ask regulatory authorities to expand the use of Orladeyo as a treatment to prevent HAE attacks in pediatric patients.
“Today’s announcement marks a very important step in our continuing efforts to reduce the burden of therapy for people living with HAE around the world with oral, once-daily Orladeyo,” Ryan Arnold, BioCryst’s chief medical officer, said in a press release.