Jazz Pharmaceuticals experienced increased retail chatter on Wednesday after the U.S. Food and Drug Administration (FDA) granted accelerated approval to Modeyso (dordaviprone) for the treatment of a specific form of brain tumor.

The drug is approved to treat adult and pediatric patients aged one year and older with diffuse midline glioma carrying an H3 K27M mutation, following progression after prior therapy. Continued approval may depend on confirmatory results from the Phase 3 ACTION trial.

Modeyso is the first FDA-approved therapy for this rare and aggressive brain tumor, which affects an estimated 2,000 people in the U.S. annually, many of them children and young adults, according to the company.

The oral, once-weekly capsule is expected to be commercially available in the coming weeks. The FDA based its decision on data from 50 patients enrolled across five open-label studies. About 1 in 5 patients responded to treatment, with responses lasting a median of just over 10 months. 

Among those who responded, nearly three-quarters saw benefits for at least six months, and more than a quarter maintained their response for at least a year.

Patrick Wen of Dana-Farber Cancer Institute called the approval a “major turning point in neuro-oncology,” noting that Modeyso introduces a new treatment option for a population with limited choices. 

About a third of glioma patients in the trial experienced serious side effects on Modeyso, including hydrocephalus, seizures, vomiting, and muscle weakness. Fatigue, nausea, and headaches were the most common complaints.

Separately, Truist Securities reduced its price target on Jazz Pharmaceuticals to $200 from $230, citing slower momentum in Epidiolex and cancer therapies such as Rylaze and Zepzelca. 

In the second quarter, Rylaze revenue dropped 7% to $100.7 million, and Zepzelca sales slipped 8% to $74.5 million. Epidiolex posted modest 2% growth to $251.7 million. 

Truist noted that the company’s total revenue was propped up by strong momentum in the oxybate franchise, where the lead drug, Xywav, rose 13% to $415.3 million. However, it flagged potential headwinds from two upcoming non-oxybate narcolepsy treatments nearing FDA review.

On Stocktwits, retail sentiment for Jazz Pharmaceuticals was ‘extremely bullish’ amid ‘high’ message volume.

Jazz Pharmaceuticals’ stock has declined 14.4% so far in 2025.

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