The European Commission has approved Ipsen’s oral therapy Ojemda (tovorafenib) to treat certain children with pediatric low-grade glioma (pLGG).
This approval, which follows a positive recommendation from European regulators last month, covers all member states of the European Union plus Iceland, Liechtenstein, and Norway. It applies to patients ages 6 months and older whose tumors have a BRAF fusion, rearrangement, or a BRAF V600 mutation, and whose cancer has progressed after at least one prior systemic therapy. Ojemda has already been granted accelerated approval for this indication in the U.S.
Ojemda targets BRAF changes in pediatric glioma
“For children diagnosed with low-grade glioma, the journey is often long and challenging with limited available treatment options,” Sandra Silvestri, MD, PhD, Ipsen’s executive vice president and chief medical officer, said in a company press release. “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.”
Ojemda is a once-weekly oral therapy designed to block the activity of mutated BRAF and related proteins. BRAF is a protein that helps regulate cell growth, and in many pLGG tumors, it becomes altered through a specific genetic change (V600) or through genetic fusions and rearrangements that lead to abnormal protein activity. By blocking this activity, Ojemda aims to slow or stop the signaling pathways that drive cancer cell growth.
“Families affected by low-grade glioma often endure years of uncertainty, difficult treatment decisions, and the fear of long-term consequences,” said François Doz, MD, a professor of pediatrics at Paris Descartes University. “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 European approval of Ojemda was based primarily on data from the Phase 2 FIREFLY-1 (NCT04775485) clinical trial. The study tested Ojemda in more than 130 patients with BRAF-altered pLGG that was relapsed or refractory (meaning the tumor had failed to respond to previous treatment or had regrown after initially responding).
Results showed that many patients given Ojemda responded to treatment, meaning their tumors got smaller. Overall response rates ranged from 53% to 71%, depending on the criteria used. The median time to response was less than six months, and the median duration of response was 18 months. The most common side effects reported in FIREFLY-1 included hair color changes, fatigue, vomiting, hypophosphatemia (low phosphate levels), headache, rash, fever, dry skin, and slowed growth.
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