Jazz Pharmaceuticals Achieves Landmark FDA Approval for Brain Cancer Therapy

In a significant advancement for pediatric oncology, Jazz Pharmaceuticals has secured accelerated FDA approval for its novel treatment targeting a rare and aggressive form of brain cancer. The regulatory green light for Modeyso (dordaviprone) was granted in August 2025 for patients aged one year and older diagnosed with diffuse midline glioma harboring an H3K27M mutation. This authorization represents the first systemic therapy specifically developed for this challenging condition.

Further validating the treatment’s importance, the FDA confirmed on August 30 that Modeyso qualified for a Rare Pediatric Disease Priority Review Voucher, underscoring its potential to address a critical unmet medical need.

This milestone follows the successful completion of Jazz Pharmaceuticals’ acquisition of Chimerix, the therapy’s original developer. Read more...