The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting a conditional marketing authorization for a new medicine to treat pediatric patients with low-grade glioma, a noncancerous brain tumor.

Ojemda (tovorafenib, Ipsen Pharma) can be used in patients aged 6 months or older whose tumor has certain changes in the BRAF gene, and in those whose disease has progressed after one or more therapies.

Pediatric low-grade gliomas are the most common brain tumors in children. Despite the excellent prognosis, many survivors experience chronic conditions affecting vision, movement, learning, and overall development as a result of their disease and/or treatment.

Current treatment options include surgery and chemotherapy, but chemotherapy’s benefits are often modest and can cause substantial side effects. For those whose disease worsens after these treatment modalities, there are no further options. 

Ojemda is a once-weekly oral targeted therapy. It works by blocking RAF proteins involved in tumor growth, slowing down or stopping the signals that cause tumor cells with certain BRAF alterations to grow and multiply.

EMA’s recommendation is based on results from an uncontrolled, open-label, phase 2 clinical study involving 77 patients with pediatric low-grade glioma whose tumor had changes in the BRAF gene and whose disease had worsened despite previous treatment. Forty patients (52.6%) achieved a response at some point during treatment with once-weekly Ojemda, with the response lasting 18 months on average.

No patients achieved a complete response (disappearance of the tumor and no new lesions), 29 achieved a partial response (at least 50% decrease in tumor size and no new lesions), and 11 achieved a minor response (25%-49% decrease in tumor size and no new lesions).

The most common side effects reported included hair color changes, increased blood creatine phosphokinase, tiredness, anemia, vomiting, low blood levels of phosphates, headache, rash, fever, growth retardation, and dry skin.

To confirm the efficacy and the safety of Ojemda, Ipsen Pharma must submit the results of an ongoing randomized controlled, phase 3 study.

The CHMP’s opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorization. 

Sophie Cousins, MIPH, is a global health journalist who has reported from more than 20 countries.