When Jazz Pharmaceuticals' incoming CEO Renee Gala takes the reins at the drugmaker next week, she'll have a brand-new launch to manage.
Wednesday, the FDA approved Jazz's Modeyso (dordaviprone) for patients ages 1 and older with H3 K27M-mutant diffuse midline glioma who have progressive disease following prior therapy. The drug, picked up in the company's $935 million buyout of Chimerix earlier this year, is the first systemic therapy for those with the aggressive brain cancer.
The FDA granted the medicine accelerated approval based on results seen across 50 patients in five open-label, nonrandomized U.S. trials, the agency said in an Aug. 6 release.
Among the evaluated patients, who were at least 90 days post radiation therapy, the overall response rate was 22% and the median duration of response was 10.3 months, according to the FDA.
The drug's label carries warnings for hypersensitivity, QTc interval prolongation and embryo-fetal toxicity, the FDA noted.
Jazz struck a $935 million deal for Chimerix back in March and closed the deal the following month.
In a December 2024 corporate presentation, Chimerix estimated there are about 2,000 patients who could potentially be eligible for dordaviprone in the U.S. Citing the potential for "ultra-orphan" pricing, the biotech said there could be a blockbuster market opportunity for its therapy. Before selling itself to Jazz, Chimerix had kicked off a phase 3 trial of its cancer med.
Meanwhile, Jazz has been working through a CEO change in 2025. Jazz's longtime CEO Bruce Cozadd announced his intention to retire in December, and the company revealed Gala as his successor in July. Gala, who's been with Jazz for five years, is set to start her new role on Monday.