FDA Grants Accelerated Approval For Targeted Drug for Rare Brain Tumor

The FDA has granted accelerated approval for Jazz Pharmaceuticals’ Modeyso (dordaviprone) to treat adult and pediatric patients 1 year of age and older. It is indicated for patients with a diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.

Diffuse midline glioma with an H3 K27M mutation is an ultra-rare and aggressive brain tumor that affects an estimated 2,000 people in the United States each year, many of whom are children and young adults.

“This approval represents a long-awaited treatment option for families affected by H3 K27M-mutant diffuse midline glioma,” David F. Arons, president and CEO of the National Brain Tumor Society, said in a news release . “This is a fast-moving, devastating disease that turns families' lives upside