Ipsen has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending conditional marketing authorisation for Ojemda (tovorafenib) as a monotherapy for paediatric low-Grade Glioma (pLGG).

The recommendation covers patients aged six months and older with pLGG harbouring a BRAF fusion or rearrangement or a BRAF V600 mutation whose disease has progressed after at least one prior systemic therapy.

Each year, more than 800 new cases of BRAF-altered pLGG are diagnosed in the European Union. The rare brain tumour can lead to multiple disabilities, including vision impairment, speech difficulties and neurological complications, significantly affecting the quality of life of affected children and their families.

Treatment for these patients is often prolonged and complex, frequently involving invasive surgery along with multiple rounds of chemotherapy or radiotherapy. These treatments can result in long-term health complications and developmental challenges.

Christelle Huguet, Executive Vice President and Head of Research and Development at Ipsen, said children diagnosed with paediatric low-grade glioma often face a difficult treatment journey with limited therapeutic options and no clearly defined standard of care. She noted that the positive opinion for tovorafenib marks a significant step toward delivering a targeted treatment that addresses tumour growth driven by abnormal BRAF genes.

If approved by the European Commission, Ojemda (tovorafenib) could become the first targeted therapy in the European Union specifically for children with relapsed or refractory BRAF-altered paediatric low-grade glioma, regardless of the type of BRAF alteration.

The CHMP’s recommendation is based on data from the pivotal Phase II FIREFLY-1 clinical trial, which evaluated the therapy in 137 children and young adults with relapsed or refractory BRAF-altered pLGG who had previously received systemic treatment.

The study demonstrated clinically meaningful and durable tumour responses, with an overall response rate of 71 percent according to Response Assessment in Neuro-Oncology High-Grade Glioma (RANO-HGG) criteria and 53 percent based on Response Assessment in Paediatric Neuro-Oncology Low-Grade Glioma (RAPNO-LGG) criteria. The clinical benefit rate reached 77 percent and 58 percent respectively under the two assessment frameworks.

Among responding patients, the median time to response was 5.4 months and the median duration of response was 18 months, indicating sustained therapeutic effects.

The therapy also demonstrated a manageable safety profile. Most treatment-related adverse events were mild to moderate (Grade 1 or 2), with a relatively low discontinuation rate of 9.5 percent. Common side effects included hair colour changes, increased creatine phosphokinase levels, fatigue and anaemia.

Another potential advantage is its convenient once-weekly oral dosing schedule, available in both tablet and liquid formulations, which may reduce disruption to daily family routines.

Professor Olaf Witt, Director of Translational Paediatric Oncology at Hopp Children's Cancer Center Heidelberg, noted that treatment options for relapsed or refractory paediatric low-grade glioma have remained limited for decades. He added that targeted therapies such as tovorafenib could significantly improve disease management, particularly for tumours driven by the BRAF-fusion oncogene.

Following the CHMP’s positive opinion, the European Commission will now review the recommendation, with a final decision on marketing authorisation expected in the coming months.

Tovorafenib is a Type II RAF kinase inhibitor designed to target signalling pathways responsible for abnormal cell growth and division in tumours. By blocking these pathways, the therapy can slow or stop tumour growth and potentially shrink cancerous cells.

The drug has already received accelerated approval from the U.S. Food and Drug Administration in the United States for patients aged six months and older with relapsed or refractory BRAF-altered paediatric low-grade glioma. Continued approval in the US will depend on confirmatory clinical trial results.

Further research is ongoing, including the Phase III FIREFLY-2/LOGGIC trial evaluating tovorafenib as a frontline treatment for patients under 25 years of age with BRAF-altered pLGG.