NZCR are delighted to have played an important role in helping Jazz Pharmaceuticals achieve the U.S. FDA’s accelerated approval of Modeyso™ (dordaviprone), the first and only targeted systemic therapy for recurrent H3 K27M‑mutant diffuse midline glioma (DMG), an ultra‑rare and devastating brain tumour affecting children and young adults.
A Landmark Approval
Modeyso’s approval marks a seismic shift in the treatment landscape of DMG. The decision was based on a pooled analysis of 50 patients from five open-label Phase II clinical studies, which demonstrated a 22% overall response rate (ORR) through independent, blinded central review using RANO 2.0 criteria Among those who responded, the median duration of response exceeded 10 months, with 73% sustaining benefit for at least six months and 27% for 12 months or more.
At NZCR Christchurch, we are especially proud to have been a key contributor: 15 phase II patients and 100 healthy volunteers in Phase 1 enrolled locally, under the leadership of Drs Chris Wynne and Nick Cross, who served as NZCR’s Principal Investigators for the trials, known internally as CHIPS, PRINGLE and CRISPS. Our involvement not only advanced the trial’s success but also highlights the calibre of New Zealand clinical research on the global stage.
Why This Matters
Diffuse midline glioma with the H3 K27M mutation has long been characterised by rapid progression and a bleak prognosis. Until now, there was no effective systemic treatment available. Launching later this month in the U.S., Modeyso offers meaningful relief for patients and families grappling with this disease.
Looking Ahead
This approval demonstrates the benefits of scientifically rigorous, collaborative multi-national research to achieve medication breakthroughs in tight timeframes. Following a third phase trial currently underway, Jazz Pharmaceuticals and their partners are preparing for a broader launch, with commitments to engage healthcare professionals and advocacy groups to secure rapid patient access.
Celebrating Collective Achievement
For NZCR, the success of the healthy volunteer and patient trials in New Zealand underscores our ongoing commitment to advancing patient-centred research for rare and aggressive conditions. Having contributed robust patient data and local expertise, Chief Scientific Officer Dr Wynne, Chief Medical Officer Nick Cross and the NZCR team reaffirm that New Zealand isan active, valued collaborator in global clinical innovation. This milestone offers a powerful message: international collaboration and scientific rigour lead to tangible breakthroughs delivered at pace.
Want to know more about Modeyso? Read the Jazz Pharmaceuticals release here:
Want to learn more about NZCR’s expertise across a wide range of clinical specialisations?
Contact Marcus Burrows at: marcus.burrows@nzcr.co.nz
