The Cure Starts Now Commends Approval of Modeyso™ for H3 K27M-Mutated DMG

Jazz Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Modeyso™ (dordaviprone / ONC201) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma (DMG) harboring 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.

H3 K27M mutated DMG is an aggressive brain tumor that affects an estimated 2,000 people in the U.S. each year, many of whom are children and young adults. It is often researched within a category of cancer referred to DIPG (Diffuse Intrinsic Pontine Glioma)/DMG which more narrowly affects approximately 350 pediatric patients per year within the U.S.  While DMG can expand to a broader group of patients and occur in other forms, most efforts and funding for research are initiated through private funding like The Cure Starts Now and the DIPG/DMG Collaborative.  

Since 2018, The Cure Starts Now Foundation has helped advance efforts to create both research and access to this unique therapy, starting first with its partnership to develop one of the first industry-patient partnerships for Expanded Access in association with the Musella Foundation, a partner in the DIPG/DMG Collaborative.  Further investment has come in partnership with the DIPG/DMG Collaborative towards another 7 specific research aims for this therapy.

“We are proud to partner in the development of yet another option for families fighting DIPG and DMG cancers,” said Keith Desserich, Chairman and Co-Founder of The Cure Starts Now Foundation. “As another tool in our arsenal, we see tremendous opportunity for improved treatments, ultimately leading us to a cure for these cancers.”

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