BioCryst Pharmaceuticals announced the enrollment of the first patient in the pivotal APeX-P trial evaluating oral, once-daily ORLADEYO in pediatric hereditary angioedema, HAE, patients who are 2 to less than 12 years of age. "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. Pediatric patients are a particularly important group where the challenges posed by disease and treatment can be significant to these children and their families, especially given the uncertainty they face as they are newly diagnosed during childhood. It is imperative that we strive to help normalize patients’ lives, as early experiences can have a lasting impact on how HAE is perceived – and managed – for their entire lifetimes. We are excited by the opportunity to introduce this new pediatric formulation of ORLADEYO that could significantly reduce the treatment burden for children and families impacted by HAE," said Dr. Ryan Arnold, chief medical officer of BioCryst. Following the completion of APeX-P, BioCryst plans to submit a supplemental New Drug Application for the potential expanded use of ORLADEYO for prophylaxis to prevent attacks in pediatric HAE patients.
Published first on TheFly
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