Ireland-based biopharma company Jazz Pharmaceuticals plc (Nasdaq:JAZZ) announced on Wednesday that the US Food and Drug Administration (FDA) has granted accelerated approval for Modeyso (dordaviprone) for the treatment of adult and paediatric patients aged 1 year and older with diffuse midline glioma harbouring an H3 K27M mutation with progressive disease following prior therapy.

Continued approval for this indication may be contingent upon verification and description of clinical benefit in the Phase 3 ACTION confirmatory trial.

According to Jazz Pharmaceuticals, 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. To address this urgent unmet patient need, Modeyso is expected to be commercially available in the coming weeks.