A New Chapter for Children with pLGG
Sandra Silvestri, M.D., PhD, Executive Vice President and Chief Medical Officer, Ipsen.
For the hundreds of children diagnosed with pediatric low-grade glioma (pLGG) across Europe each year, the journey is rarely what families expect. It is often long, complex, and life-altering. However today, we’re excited that the treatment landscape has progressed.
The European Commission (EC) has granted conditional marketing authorization for the first targeted therapy in Europe for children with relapsed or refractory pLGG, irrespective of the type of BRAF alteration.
As a physician, I have seen how heavily uncertainty can weigh down the families of children with life-altering conditions. With pLGG, it is not just a medical challenge. It can be a lifelong one. For these families, this approval represents something genuinely new: a treatment option developed specifically for the biology of pLGG.
pLGG is a rare and life-altering brain tumor that can cause profound and lasting neurological consequences – loss of vision, speech difficulties, learning challenges – all of which shape a child’s development and quality of life well into adulthood. Treatment frequently involves surgery and repeated chemotherapy, with limited options if the disease returns.
At a time when only a small fraction of new medicines focus on pediatric diseases, this approval marks a rare but important advance in pediatric oncology.
The EC approval reflects the collective effort of researchers, clinicians, trial participants, patient advocates, and Ipsen teams who have worked with a singular focus on this disease. I am deeply grateful to each of them.
But approval is just the beginning. Our focus now turns to ensuring this therapy reaches the children and families across Europe who need it. We stand ready to work collaboratively with stakeholders across member states to support timely access.
Children living with rare cancers deserve more options. To them, and to the families who have never stopped hoping: we will keep going.
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