Ipsen S.A. — Ojemda EMA EPAR public assessment report (EMEA/H/C/006140, 2026)
Ipsen S.A. · 2026 · EMA EPAR public assessment report · company profile →
Report summary
The EMA granted a Conditional Marketing Authorisation for Ipsen's Ojemda (tovorafenib) for pediatric low-grade glioma (LGG) with BRAF fusions/rearrangements or V600 mutation, for patients aged 6 months and older who have progressed after prior systemic therapies. This decision, despite initial concerns about overlap with existing therapies and long-term safety, was based on promising efficacy and unmet medical need. Ojemda, available as tablets and oral suspension, is a RAF kinase inhibitor. Non-clinical studies detailed its mechanism, pharmacokinetics, and potential toxicities, including reproductive and phototoxicity risks. Clinical data from the FIREFLY-1 study in pediatric LGG patients showed an overall response rate of 52.6-53.9% and a median duration of response of 16.8-18.0 months. Safety analysis revealed a high incidence of adverse events, including Grade ≥3 TEAEs (81.8%), growth retardation, and specific concerns like hepatic and cardiac safety. Further data from the FIREFLY-2 study is deferred until 2030 to confirm long-term safety and efficacy.
Key findings — cited to the page
| TEAEs Related to Study Drug with Severity Grade 3 or Higher for > 350 - 420 mg/m2 dose | 55.6 % | FIREFLY-1 · p.107 |
| TEAEs Related to Study Drug with Severity Grade 3 or Higher for > 420 mg/m2 dose | 74.6 % | FIREFLY-1 · p.107 |
| TEAEs with Severity Grade 3 or Higher for ≤ 350 mg/m2 dose | 73.3 % | FIREFLY-1 · p.107 |
| TEAEs Related to Study Drug with Severity Grade 3 or Higher for ≤ 350 mg/m2 dose | 66.7 % | FIREFLY-1 · p.107 |
| Rhinorrhoea (Any Grade) | 13.9 % | FIREFLY-1 · p.108 |
| Grade ≥ 3 TEAEs in FIREFLY-1 (Arm 1 + Arm 2) | 81.8 % | FIREFLY-1 · p.106 |
| Vomiting (Any Grade) | 56.2 % | FIREFLY-1 · p.107 |
| Fatal (Grade 5) TEAEs in FIREFLY-1 (Arm 1 + Arm 2) | 2.9 % | FIREFLY-1 · p.106 |
| TEAEs with Severity Grade 3 or Higher for > 350 - 420 mg/m2 dose | 74.6 % | FIREFLY-1 · p.107 |
| TEAEs with Severity Grade 3 or Higher for > 420 mg/m2 dose | 91.5 % | FIREFLY-1 · p.107 |
| Headache (Any Grade) | 52.6 % | FIREFLY-1 · p.107 |
| Hair colour changes (Any Grade) | 77.4 % | FIREFLY-1 · p.107 |
Source: Ipsen S.A. ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Oncology library →