Jazz Pharmaceuticals plc (JAZZ) Wins FDA Nod for Modeyso in Rare Pediatric Brain Tumors

In this article, we will be taking a look at the 12 Cheap Healthcare Stocks to Buy Now. Jazz Pharmaceuticals plc stands ninth among them.

Jazz Pharmaceuticals plc (NASDAQ:JAZZ), a global biopharmaceutical leader, develops and commercializes therapies for complex conditions in oncology, neuroscience, and sleep medicine. Its portfolio includes treatments for narcolepsy, epilepsy, and rare cancers, reflecting a focus on addressing unmet medical needs.

The company recently reached a major milestone with the FDA’s accelerated approval of Modeyso (dordaviprone) on August 6, 2025, for treating H3 K27M-mutant diffuse midline glioma, a rare and aggressive brain cancer affecting around 2,000 patients annually in the U.S., primarily children and young adults. Modeyso is the first and only therapy approved for patients aged one year and older with progressive disease following prior treatment. In clinical trials, the drug demonstrated a 22% overall response rate among 50 patients, with a median response duration of 10.3 months; 73% of responders maintained their response for over six months. Modeyso is administered as a weekly oral capsule and will become commercially available soon, with continued approval contingent on the ongoing Phase 3 ACTION confirmatory trial.

Jazz Pharmaceuticals plc (JAZZ) Wins FDA Nod for Modeyso in Rare Pediatric Brain Tumors

In addition to Modeyso, Jazz Pharmaceuticals plc (NASDAQ:JAZZ) is diversifying its pipeline and expanding its strategic focus. The business recently licensed SAN2355, a preclinical epilepsy drug, strengthening its neuroscience portfolio. In oncology, it is advancing zanidatamab in Phase 3 for advanced gastroesophageal cancers and pursuing regulatory approval for Zepzelca as a first-line treatment for extensive-stage small cell lung cancer.

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