Jazz Pharmaceuticals plc (JAZZ) Advances Rare Cancer Portfolio with First CNS Glioma Therapy Approval

In this article, we will be taking a look at the 10 Best Pharma Stocks to Buy According to Billionaires. Jazz Pharmaceuticals plc is one of them.

Jazz Pharmaceuticals plc (NASDAQ:JAZZ) continues to advance its mission to transform patient lives through innovative therapies for conditions with limited treatment options. The company’s marketed products lead in sleep medicine (Xywav, Xyrem), epilepsy (Epidiolex), and oncology (Zepzelca), while strategic acquisitions, including GW Pharmaceuticals and Chimerix, have strengthened its pipeline with promising investigational drugs. The stock stands tenth on our list among the best pharmaceutical stocks. 

On August 6, 2025, Jazz Pharmaceuticals plc (NASDAQ:JAZZ) received FDA accelerated approval for Modeyso (dordaviprone), a DRD2 antagonist for patients with relapsed H3 K27M-mutated diffuse midline glioma. Modeyso is the first approved therapy for this ultra-rare CNS cancer, demonstrating a 22% overall response rate in a pooled analysis of 50 patients across five trials. While the label carries warnings for hypersensitivity and QTc prolongation, these risks are considered acceptable given the aggressive nature of the disease. Continued approval will depend on results from the confirmatory Phase III Action trial, with interim data expected by year-end 2025.

The approval underscores JAZZ’s growing focus on rare oncology indications. The business is also advancing Zepzelca for small-cell lung cancer, planning a supplemental New Drug Application for frontline use based on positive Phase III results, and zanidatamab, a HER2-targeted bispecific antibody in pivotal trials for gastroesophageal adenocarcinoma, with results expected in Q2 2025.

Looking ahead, Jazz Pharmaceuticals plc (NASDAQ:JAZZ)is poised for near-term growth with the commercial launch of Modeyso, ongoing pivotal trial readouts, and regulatory submissions for expanded indications.

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