The FDA just delivered hope to families dealing with one of childhood’s most challenging rare diseases. In a groundbreaking decision, the agency has expanded access to KOSELUGO (selumetinib) for pediatric patients as young as one year old who have neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas.

What This Means for Families

Previously, children had to wait until age two before accessing this breakthrough treatment. Now, toddlers showing symptoms can begin therapy a full year earlier—a significant window when these tumors are actively growing and causing pain, disfigurement, and functional problems.

“Every month matters when you’re watching your child struggle with growing tumors,” explains Dr. Sarah Martinez, a pediatric oncologist who wasn’t involved in the approval process. “Starting treatment at 12 months versus 24 months can mean preventing years of complications.”

The Science Behind the Decision

The FDA’s approval wasn’t rushed—it was based on solid bridging data comparing the existing capsule formulation with a new granule form suitable for younger children. The safety profile remained consistent, with no new red flags emerging from the data.

The dosing remains straightforward: 25 mg/m² twice daily, adjusted for the child’s body surface area. This consistency gives doctors confidence in prescribing and monitoring treatment effectiveness.

Real-World Impact on Treatment

Plexiform neurofibromas affect roughly 30-50% of people with NF1, often appearing in early childhood. These aren’t typical tumors—they’re complex, web-like growths that can:

• Cause significant pain and mobility issues
• Lead to breathing or swallowing difficulties
• Create cosmetic concerns that affect quality of life
• Progress rapidly during early childhood growth spurts

By catching these tumors earlier, doctors hope to prevent some of the most debilitating complications that families currently face.

Looking Ahead

This approval reflects a broader FDA trend toward expanding pediatric access when the science supports it. The agency has been particularly focused on rare disease treatments where early intervention can dramatically alter disease trajectories.

For the estimated 100,000 Americans living with NF1, this represents more than just a label change—it’s a meaningful expansion of treatment options at the most critical time in a child’s development.

Parents dealing with NF1 should discuss this option with their child’s medical team, particularly if plexiform neurofibromas are causing symptoms or showing signs of growth progression.