Therapeutic Response Statement (Sensitivity)
This therapeutic response statement supports the relationship that H3-3A p.K27M status confers therapeutic sensitivity to Dordaviprone in patients with Diffuse Midline Glioma.
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. H3 K27M 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 statement is based on a regulatory approval from the Food and Drug Administration:
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.