Pharmaceuticals’ Modeyso
FDA approves Jazz Pharmaceuticals’ Modeyso as first drug for ultra-rare brain cancer
The US Food and Drug Administration (FDA) has granted accelerated approval to Jazz Pharmaceuticals’ Modeyso (dordaviprone) as the first treatment for an ultra-rare and aggressive brain tumour that primarily affects children and young adults.
The drug has been approved to treat adult and paediatric patients aged one year and older with diffuse midline glioma harbouring an H3 K27M mutation who have progressive disease following prior therapy.
The disease, which progresses rapidly and historically has had no effective systemic treatment options, occurs in an estimated 2,000 people in the US every year. Patients usually face an extremely poor prognosis, with median survival being around one year from diagnosis and less than six months after progressing following frontline therapy.
Given as an oral capsule once weekly, Modeyso is a small molecule that selectively binds to the G-protein coupled dopamine receptor D2 and the mitochondrial protease ClpP. Jazz gained access to the drug earlier this year through its $935m acquisition of Chimerix.
The FDA’s approval was supported by an analysis of 50 adult and paediatric patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label clinical studies.
The overall response rate was 22% and the median duration of response was 10.3 months, with 73% of responders maintaining their response for at least six months and 27% for at least 12 months.
Joshua Allen, chief scientific officer, Chimerix, a Jazz Pharmaceuticals company, said: “This approval not only equips clinicians with the first targeted option for this disease but also signals a meaningful shift in what patients and families can expect after diagnosis.”
In line with the FDA’s accelerated approval pathway, continued approval for Modeyso in this indication may be contingent on the verification of clinical benefit in the late-stage ACTION confirmatory trial, which is evaluating the drug in newly diagnosed patients with H3 K27M-mutant diffuse glioma following radiotherapy.
