EMA Recommends Ojemda for Paediatric Low-Grade Glioma

EMA News

Published February 27th, 2026

Detected February 27th, 2026

Summary

The European Medicines Agency (EMA) has recommended a conditional marketing authorisation for Ojemda (tovorafenib) to treat paediatric low-grade glioma in patients aged 6 months and older with specific BRAF gene alterations. This recommendation addresses an unmet medical need for a targeted therapy in this patient population.

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What changed

The European Medicines Agency (EMA) has recommended a conditional marketing authorisation for Ojemda (tovorafenib) for the treatment of paediatric low-grade glioma in patients aged 6 months and older who have specific BRAF gene alterations and whose disease has progressed despite prior systemic treatment. This recommendation is based on Phase 2 clinical trial data showing a response rate of 52.6% in 77 patients. Ojemda is a new oral targeted therapy designed to block RAF proteins involved in tumour growth.

This recommendation is a significant step towards early patient access to a new treatment option for a rare paediatric brain tumour. While the conditional authorisation allows for earlier availability, the marketing authorisation holder, Ipsen Pharma, is committed to submitting results from an ongoing Phase 3 study to confirm efficacy and safety. Following the European Commission's decision, individual Member States will determine pricing and reimbursement. Healthcare providers treating paediatric patients with low-grade glioma and drug manufacturers should be aware of this development and its implications for treatment protocols and market access.

Source document (simplified)

New medicine to treat paediatric low-grade glioma

27 February 2026

Ojemda addresses unmet need for patients aged 6 months and older with paediatric brain tumour with BRAF alterations News Human Medicines Paediatrics

EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Ojemda (tovorafenib) to treat patients aged 6 months and older with paediatric low-grade glioma (a type of non-cancerous brain tumour).

Ojemda can be used when the tumour has certain changes in the BRAF gene (such as a BRAF fusion, rearrangement or V600 mutation) in patients whose disease has worsened despite previous treatment with one or more systemic medicines.

Paediatric low-grade glioma is the most common brain tumour in children. Although survival is generally high, many patients experience serious long‑term complications affecting vision, movement, learning and overall development, with a significant impact on their quality of life.

Current treatment options include surgery and chemotherapy. While chemotherapy can be effective for some patients, its benefits are often modest and it may cause substantial side effects. In addition, targeted therapy is available only to patients whose tumours have a BRAF ** V600E mutation. However, for patients whose disease worsens after this treatment, there are no further treatment options. Ojemda is a new once-weekly oral targeted therapy for a broader group of patients with paediatric low‑grade glioma.

The active substance of Ojemda, tovorafenib, works by blocking RAF proteins involved in tumour growth. By targeting these proteins, tovorafenib helps slow down or stop the signals that cause tumour cells with certain BRAF gene changes, including BRAF ** V600 mutations and BRAF fusions, to grow and multiply.

EMA’s recommendation is based on data from an uncontrolled, open-label, phase 2 clinical study involving 77 patients with paediatric low-grade glioma, whose tumour had changes in the BRAF gene and whose disease had worsened despite previous treatment with one or more systemic medicines. Forty patients (52.6%) achieved a response at some point during treatment with once-weekly Ojemda, and the response lasted 18 months on average. No patients achieved a complete response (disappearance of the tumour and no new lesions), 29 achieved a partial response (at least 50% decrease in tumour size and no new lesions), and 11 achieved a minor response (25–49% decrease in tumour size and no new lesions).

The most common side effects reported with Ojemda include hair colour changes, increased blood creatine phosphokinase (enzyme released into the blood when muscle is damaged), tiredness, anaemia, vomiting, low blood levels of phosphates, headache, rash, fever, growth retardation and dry skin.

Ojemda is recommended for a conditional marketing authorisation, one of the EU regulatory mechanisms to facilitate early access to medicines that fulfil an unmet medical need. This type of approval allows the Agency to recommend a medicine for marketing authorisation with less complete data than normally expected, if the benefit of a medicine’s immediate availability to patients outweighs the risk inherent in the fact that not all the data are yet available.

To confirm the efficacy and the safety of Ojemda, the company has committed to submit the results of an ongoing randomised, controlled, phase 3 study.

The opinion adopted by the CHMP is an intermediary step on Ojemda’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.

Notes:

The applicant for Ojemda is Ipsen Pharma.
Ojemda was designated as an orphan medicinal product for the treatment of glioma on 20 May 2021. Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designation should be maintained.
Ojemda falls under the scope of the EU Health Technology Assessment Regulation. The Member State Coordination Group on Health Technology Assessment (HTA) will endorse the draft joint clinical assessment on this product within 30 days of the granting of the marketing authorisation. The joint clinical assessment is independent of EMA’s review of the marketing authorisation application and is coordinated by the HTA Secretariat within the European Commission.