EMA Panel Backs Conditional EU Approval for Ojemda in Pediatric Low-Grade Glioma

Overview

• CHMP recommended a conditional marketing authorisation for tovorafenib for patients aged six months and older whose paediatric low‑grade glioma has progressed after at least one systemic therapy.
• The indication covers tumours with BRAF fusions or rearrangements or BRAF V600 mutations, to be used as monotherapy under specialist supervision.
• In a single‑arm phase 2 trial of 77 patients, Ojemda achieved a 52.6% overall response rate with a median response duration of 18.0 months, with no complete responses reported.
• The medicine is a once‑weekly oral BRAF inhibitor and would be available as a 25 mg/ml powder for suspension and 100 mg tablets if authorised.
• Ipsen must provide results from an ongoing randomised phase 3 study, while orphan designation review, joint clinical assessment and national pricing and reimbursement decisions will follow any EU authorisation.