French biopharmaceutical company Ipsen (Euronext Paris:IPN) (ADR:IPSEY) announced on Wednesday that the European Commission has granted conditional marketing authorisation for Ojemda (tovorafenib) as a monotherapy for patients aged six months and older with relapsed or refractory paediatric low-grade glioma involving BRAF alterations.

The approval applies across all 27 EU Member States, as well as Iceland, Liechtenstein, and Norway, positioning the therapy as a new treatment option for a rare and life-altering paediatric brain tumour.

More than 800 children in the EU are diagnosed annually with BRAF-altered paediatric low-grade glioma, a condition associated with long-term physical and neurological impairments. Historically, treatment has relied on invasive surgeries, chemotherapy, and radiotherapy, often resulting in significant health complications and diminished quality of life.

The decision is supported by data from the Phase II FIREFLY-1 trial, which evaluated tovorafenib in 137 patients with relapsed or refractory disease. The study demonstrated overall response rates of 71% under RANO-HGG criteria and 53% under RAPNO-LGG criteria, with durable responses showing a median duration of 18 months and a median time to response of 5.4 months.

Tovorafenib exhibited a manageable safety profile, with predominantly low-grade adverse events and a 9.5% discontinuation rate due to treatment-related effects. Its once-weekly oral dosing, available in liquid or tablet form, offers a convenient administration schedule designed to reduce disruption to patients and families.

The approval also marks the first medicine to undergo evaluation under the EU's Joint Clinical Assessment process introduced in 2025, highlighting a shift toward harmonised clinical evidence review.

Ipsen secured ex-US rights to tovorafenib from Day One Biopharmaceuticals Inc in 2024.