Jazz gets its Chimerix approval

Jazz Pharmaceuticals has taken the first step towards justifying its $935m takeover of Chimerix, gaining US accelerated approval for dordaviprone on Wednesday in an ultra-rare brain tumour. Dordaviprone, a DRD2 antagonist now branded Modeyso, has the go ahead in relapsed H3 K27M-mutated diffuse midline glioma, which primarily affects children and young adults. The nod was based on an overall response rate of 22% among 50 patients who met prespecified criteria in a pooled analysis of five uncontrolled trials. Modeyso’s label carries warnings for hypersensitivity and QTc prolongation, likely acceptable risks in an aggressive disease with no other options. Jazz plans to launch the drug in the “coming weeks”; a webcast on 27 August will give more details on the group’s commercial strategy, perhaps including Modeyso’s price. Continued approval will depend on the confirmatory Action trial, although readout timings are unclear. Jazz said in May that the first interim analysis was expected by year-end 2025, although in its second-quarter earnings call this week the company said it was assessing timelines, and would give an update “when appropriate”. Jazz added that enrolment was on track, although it has previously been more pessimistic about recruitment. 

Notable trials of Modeyso in glioma

Note: *expanded access; **ph3 confirmatory trial. Source: OncologyPipeline & product label.