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Ojemda approved in the European Union as the first targeted therapy in relapsed or refractory pediatric low-grade glioma regardless of BRAF alteration

April 22, 2026 – Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical – BRAF alteration, European Commission, Ipsen, conditional marketing authorisation, r/r paediatric low-grade glioma

New treatment option for rare, life-altering pediatric brain tumors
Less than 10% of new medicine approvals over the past five years have focused on pediatric diseases; Ojemda (tovorafenib) represents a rare achievement that reinforces the urgent need to close the innovation and investment gaps in pediatric therapeutics
Approval is based on pivotal Phase II FIREFLY-1 data demonstrating meaningful and durable tumor responses.

22 April 2026 — Paris, France — Ipsen today announced that the European Commission (EC) has granted conditional marketing authorization for Ojemda (tovorafenib) as monotherapy for the treatment of patients 6 months of age and older with pediatric low-grade-glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation, who have progressed after one or more prior systemic therapies. This EC decision applies across all 27 EU Member States, as well as Iceland, Liechtenstein, and Norway.

More than 800 children are diagnosed with BRAF altered pediatric low-grade glioma (pLGG) each year in the EU. This brain tumor, while classified as low-grade (slow progression) carries a profound lifelong burden, frequently leading to significant physical and neurological impairments including loss of vision, speech difficulties and motor dysfunction, which can significantly impact a child’s education, independence and long-term quality of life.

Until now, many children living with pLGG have had to go through invasive surgeries, multiple lines of chemotherapy, and radiotherapy, often resulting in health complications.

“For children diagnosed with low-grade glioma, the journey is often long and challenging with limited available treatment options,” said Sandra Silvestri, M.D., PhD, Executive Vice President and Chief Medical Officer, Ipsen. “Today’s approval is a meaningful step forward for these children, and their families, while reinforcing our commitment to addressing high unmet need. Now, our focus is on ensuring that eligible children across Europe can access this therapy as quickly as possible.”

The EC approval is based on data from the pivotal Phase II FIREFLY-1 studyi which evaluated tovorafenib in 137 children and young adults with relapsed or refractory BRAF-altered pLGG who had received at least one prior systemic therapy. The study demonstrated:

Clinically meaningful tumor response: An overall response rate of 71% per the Response Assessment in Neuro-Oncology criteria for High-Grade Gliomas (RANO-HGG) criteria and 53% per Response Assessment in Pediatric Neuro-Oncology for Low-Grade Glioma (RAPNO-LGG) criteria, with a clinical benefit rate of 77% per RANO-HGG criteria and 58% per RAPNO-LGG criteria
Rapid and durable responses: Based on RAPNO-LGG criteria, among responders, the median time to response was 5.4 months with a median duration of response of 18.0 months
Manageable safety profile: Tovorafenib was generally well-tolerated, with predominantly Grade 1 or 2 treatment-related adverse events (TRAEs) and a low discontinuation rate (9.5% patients discontinued treatment due to events considered by the investigator to be related to tovorafenib). The most common TRAEs were hair color changes, blood creatine phosphokinase increased, fatigue, anemia, vomiting, hypophosphataemia, headache, rash maculo-papular, pyrexia, growth retardation, dry skin
Convenient Dosing: Once-weekly oral administration, with or without food, in liquid or tablet formulation, minimizing disruption to daily family routine.

“Families affected by low-grade glioma often endure years of uncertainty, difficult treatment decisions, and the fear of long-term consequences,” said Professor François Doz, Professor of Pediatrics at Paris Descartes University, Deputy Director of Clinical Research, Innovation and Teaching in the SIREDO Oncology Centre of the Curie Institute (Care, Innovation and research in Cancer of the child, adolescent and young adult) and Director of Teaching of the Hospital Ensemble of the Institut Curie. “The approval of a targeted therapy like tovorafenib represents a major step forward, offering families not only a new treatment option, but a renewed optimism.”

The EU Health Technology Assessment (HTA) Regulation, which began phasing in from January 2025, introduced a new Joint Clinical Assessment (JCA) process to streamline and harmonize the comparative clinical evidence review across EU Member States. Ojemda is the first medicine to undergo a JCA evaluation.

About Ipsen
We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com.