Jazz Pharmaceuticals plc has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Modeyso™ (dordaviprone). This marks the first and only treatment for recurrent H3 K27M-mutant diffuse midline glioma, a rare and aggressive brain tumor affecting both children and young adults. The approval is based on the overall response rate in patients with progressive disease following prior therapy. Continued approval may depend on the verification of clinical benefits in the Phase 3 ACTION confirmatory trial. This development represents a significant milestone for patients and families impacted by this devastating disease, providing a long-awaited treatment option. Jazz Pharmaceuticals acknowledged the collective efforts of patients, advocates, clinicians, researchers, and partner institutions in achieving this breakthrough. An investor webcast will be hosted by the company on August 27, 2025, to discuss the approval further.
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