Regulatory approval

Published by the Food and Drug Administration. Accelerated

The U.S. Food and Drug Administration (FDA) granted accelerated approval to dordaviprone adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. The underlying trials, including ONC018 (NCT03134131), assessed H3 K27M status through either IHC or sequencing for patient eligibility. The H3 K27M nomenclature refers to the H3-3A:c.83A>T p.K28M (K27M) variant.

This is written in the approval document as:

MODEYSO is a protease activator indicated for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.

Citation

Chimerix, Inc. Modeyso (dordaviprone) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219876s000lbl.pdf. Revised August 2025. Accessed October 2, 2025.

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Organizations	Biomarkers	Cancer types	Therapies
FDA (2)	H3-3A p.K28M	Diffuse Midline Glioma	Dordaviprone

Contributions

Date	Contributor	Description
2025-10-02	Van Allen lab	On 2025-08-06, the US FDA granted accelerated approval to Modeyso (dordaviprone) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.
2025-08-06	Food and Drug Administration	Indication received accelerated approval.