DUBLIN, Aug. 20, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) will host an investor webcast on Wednesday, August 27, 2025, at 4:30 p.m. EDT / 9:30 p.m. IST to provide an overview of clinical data, patient need and commercialization strategy for Modeyso™ (dordaviprone). Modeyso was approved under accelerated approval by the U.S. Food and Drug Administration (FDA) on August 6, 2025, for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring ...

Core Insights - Jazz Pharmaceuticals has received FDA accelerated approval for Modeyso™ (dordaviprone) as the first treatment for H3 K27M-mutant diffuse midline glioma, primarily affecting children and young adults [1][2] - Modeyso is expected to be commercially available in the coming weeks, addressing an urgent unmet need for patients with this aggressive brain tumor [1][2] - The approval is based on a clinical analysis of 50 patients, showing an overall response rate of 22% and a median duration of response of 10.3 months [2][7] Company Overview - Jazz Pharmaceuticals is a global biopharma company focused on developing innovative medicines for serious diseases with limited treatment options [21] - The company aims to transform the lives of patients and their families through a patient-focused and science-driven approach [21] Product Details - Modeyso is an orally administered small molecule given once weekly, targeting the specific genetic mutation associated with the tumor [7] - The ongoing Phase 3 ACTION trial will further evaluate the clinical benefit of Modeyso in newly diagnosed patients [7] Clinical Data - The FDA's decision was based on an integrated efficacy analysis from five open-label clinical studies, with serious adverse reactions reported in 33% of patients [2][3] - Among responders, 73% maintained their response for at least six months, and 27% for at least 12 months [2] Market Impact - The approval of Modeyso represents a significant advancement in neuro-oncology, providing a new treatment option for a population with historically limited choices [2][6] - The estimated annual incidence of H3 K27M-mutant diffuse midline glioma in the U.S. is around 2,000 cases [1][6]