Ipsen: Positive EMA Opinion for Ojemda in Pediatric Glioma
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has recommended conditional marketing authorization for Ipsen's Ojemda® (tovorafenib) to treat children with relapsed or refractory low-grade pediatric glioma with BRAF alteration. The decision is based on data from the pivotal FIREFLY-1 study.
Potential First and Only Targeted Therapy in the EU for Pediatric Glioma
If approved, Ojemda is set to become the first and only targeted therapy in the European Union for children with relapsed or refractory low-grade pediatric glioma (pLGG) with any type of BRAF alteration. The medication is indicated as a monotherapy for patients aged 6 months and older who have progressed after one or more previous systemic therapies. Over 800 new cases of pLGG with BRAF alteration are identified each year in the European Union. The current therapeutic journey for these patients typically involves multiple invasive surgeries as well as intensive chemotherapy and radiotherapy over many years, resulting in lifelong health issues and developmental complications.
CHMP's Positive Opinion Based on FIREFLY-1 Study Data
The positive opinion from the CHMP is based on data from the FIREFLY-1 study, which evaluated tovorafenib in 137 children and young adults with relapsed or refractory pLGG. The study demonstrated clinically significant tumor responses, with an overall response rate of 71% according to RANOHGG criteria and 53% according to RAPNOLGG criteria. According to RAPNOLGG criteria, the median time to response was 5.4 months, with a median response duration of 18.0 months. Tovorafenib was generally well tolerated, with primarily grade 1 or 2 adverse effects and a discontinuation rate of 9.5%. The administration regimen is a weekly oral dose, available in liquid or tablet form, with or without food. Following this opinion, the European Commission will review the CHMP's recommendation, with a final decision expected in the coming months.
