Jazz Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has granted accelerated approval for Modeyso (dordaviprone). It is for the treatment of patients aged one year and older who have been diagnosed with diffuse midline glioma harbouring an H3 K27M mutation, with progressive disease following prior therapy.
Modeyso is the first and only treatment option approved by the FDA for this ultra-rare and aggressive brain tumour that affects an estimated 2,000 people in the US each year, many of whom are children and young adults. The disease is characterised by rapid progression, and historically has had no effective systemic treatment options.
Modeyso is administered as an oral capsule once weekly and, to address this urgent unmet patient need, Modeyso is expected to be commercially available in the coming weeks.
"This is a major turning point in neuro-oncology," said Patrick Wen, MD, Director for Center for Neuro-Oncology, Dana-Farber Cancer Institute and Professor of Neurology, Harvard Medical School. "This therapy was developed with the underlying biology of the tumour in mind and introduces a new treatment option for a population with historically limited choices."
David F Arons, President and Chief Executive Officer of US-based charity the National Brain Tumor Society, added: "This approval represents a long-awaited treatment option for families affected by H3 K27M-mutant diffuse midline glioma. Families finally have a treatment option, and a reason to believe in more time together to make memories that might not have otherwise been possible."
As positive as this news is, it is only approved in the US, so Modeyso will not yet be generally available to UK patients. Any access in the UK is via a managed access program (MAP) for DIPG patients only in the absence of any available open clinical trials. UK patients with H3K27M+ DMG have access to the Phase 3 clinical trial ACTION study, which contains a placebo arm.
You can read the full Modeyso press release from Jazz Pharmaceuticals here.
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