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Targeted Therapies

Here’s how the last 70 years of FDA approvals for pediatric brain cancers compare to just the past 16 months (2024–2025):

Historical Context: 1950s–Early 2020s

  • For decades, there were no FDA-approved drugs specifically targeting pediatric brain cancers.

  • Standard care consisted almost entirely of:

    • Surgery (when possible)

    • Radiation therapy

    • Chemotherapy (often with agents originally developed for adult cancers—temozolomide, carboplatin, vincristine, etc.).

  • These were “borrowed” treatments rather than drugs designed for children or for their specific tumor biology.

 Until recently, pediatric brain cancer drug development was extremely limited, partly because:

  • Brain tumors are rare (pediatric cancers make up only ~1% of all cancers).

  • Ethical and practical challenges in running pediatric clinical trials.

  • Historically, pediatric cancers did not attract the same R&D funding as adult cancers.

Turning Point: Mid-2010s–2020s

A few approvals touched on pediatric central nervous system tumors, but not directly on pediatric brain cancers:

  • 2010s–2020s: Drugs like selumetinib (2020) and mirdametinib (2025) were approved for NF1-related plexiform neurofibromas, which are tumors of the nervous system but not brain cancers per se.

Still—no systemic therapy for pediatric gliomas or diffuse midline glioma (DMG) until very recently.

The Breakthrough Era: 2024–2025

In just over a year, three new targeted therapies received FDA approval specifically for pediatric brain tumors:

  1. Tovorafenib (Ojemda, 2024) – relapsed/refractory low-grade glioma with BRAF alterations.

  2. Vorasidenib (Voranigo, 2024) – grade 2 IDH-mutant gliomas (adults and ≥12 pediatric).

  3. Modeyso (2025) – diffuse midline glioma (DMG), the first ever systemic therapy for this devastating childhood tumor.

Comparison in Simple Terms

  • 1950s–2023 (≈70 years):
    → 0 FDA-approved drugs specifically for pediatric brain cancers.
    → Children were treated with adult chemotherapy, surgery, and radiation.

  • 2024–2025 (≈16 months):
    → 3 FDA approvals (tovorafenib, vorasidenib, Modeyso).
    → First true wave of targeted therapies designed with pediatric brain tumor biology in mind.

In summary:
For nearly 70 years, there were no pediatric brain cancer–specific drugs on the market. But in the past year and a half, we’ve seen a historic surge with three first-ever approvals—a dramatic shift that signals the start of a new era in treatment.