Progress for Families Facing Rare Childhood Brain Tumors 

Written by: Christelle Huguet, Executive Vice President, Head of R&D

Today marks an important moment for children and families affected by one of the most challenging rare childhood brain tumors: pediatric low‑grade glioma (pLGG). 

Each year, hundreds of children across Europe are diagnosed with pLGG. For many, the journey involves invasive surgeries, years of intensive chemotherapy and the lifelong impact of potential vision, speech and neurological complications. With no global standard of care, families are often left facing uncertainty, complexity and incredibly difficult choices often over many years. 

That’s why today’s news is so significant. Ipsen has received a positive CHMP opinion for a medicine that, if approved, could become the first and only targeted treatment in Europe for children with relapsed or refractory BRAF altered pLGG. It has the potential to transform how we support children living with this relentless disease and to broaden the treatment options available to doctors as they manage this challenging condition.  

This progress reflects the dedication of so many scientists advancing rare oncology innovation, physicians, trial participants and partners bringing expertise to the clinic, children living with pLGG, their loved ones and patient advocates giving voice to families’ needs, and our teams across Ipsen whose passion and persistence make breakthroughs like this possible.  

I’m humbled and grateful for the commitment that underpins this milestone. 

The European Commission will now review the CHMP recommendation, with a final decision expected in the coming months.  

Moments like this remind us why this work matters. Because children and their families deserve better options. And, because innovation in pediatric oncology can’t wait.