New pediatric brain tumor drug enters expedited review in KOR

MFDS grants GIFT designation to ‘Ojemda (tovorafenib)’ for pediatric low-grade glioma

‘Ojemda (tovorafenib),’ a long-awaited breakthrough therapy for pediatric brain tumor, has finally entered Korea’s expedited review pathway. The progress comes approximately two years after the drug received approval from the U.S. Food and Drug Administration (FDA).

The Ministry of Food and Drug Safety (MFDS) has officially designated Ipsen Korea’s pediatric low-grade glioma (pLGG) drug Ojemda as a product eligible for Korea’s Global Innovative products on Fast Track (GIFT) program. The designation was granted on June 24. Ojemda is being reviewed for the treatment of

Pediatric low-grade glioma is one of the most common brain tumors in children. Although the term ‘low-grade’ may mistakenly suggest a relatively mild disease, these tumors frequently develop in critical areas such as the optic pathway or the center of the brain, making surgical removal impossible in many cases. They can cause lifelong neurological complications including paralysis and vision loss due to brain damage and may progressively become life-threatening.

Until now, treatment has largely relied on conventional chemotherapy agents such as carboplatin and vincristine. However, tumors often develop resistance and begin growing again after 1-2 years of treatment. Children have also had to endure severe treatment-related adverse events, including hair loss, impaired growth, and neuropathy.

Ojemda is regarded as the first highly effective treatment capable of overcoming these limitations. It is a ‘selective Type II RAF kinase inhibitor’ specifically designed to penetrate the central nervous system and target abnormal signaling within the MAPK (mitogen-activated protein kinase) pathway, which drives tumor cell growth.

Whereas previously available therapies achieved response rates (tumor shrinkage percentage) of only 20-30% in patients with recurrent disease, Ojemda demonstrated response rates of approximately 50-60% and attracted widespread attention from the global medical community. In Korean clinical trials, encouraging outcomes were observed, including cases in which tumors shrank markedly in children who were progressively losing their vision and another in which a pediatric patient with leptomeningeal metastasis (terminal stage), who had been diagnosed with a limited life expectancy, has survived for 3 to 4 years with brain and spinal tumors having almost completely disappeared.

The drug has already gained recognition for its innovativeness from major global regulatory authorities. The FDA granted it Breakthrough Therapy Designation (BTD) before approving it on April 23, 2024, while the European Medicines Agency (EMA) approved the therapy through its Conditional Marketing Authorization (CMA) pathway on April 22, 2026.

In Korea, however, delays in the regulatory process left pediatric patients unable to benefit even from humanitarian compassionate-use programs and to continue receiving highly toxic chemotherapy. Clinical experts have repeatedly urged the MFDS to expedite the review, noting that “large-scale clinical trials are inherently difficult in rare pediatric diseases because of the limited number of patients. Even delays of one or two months caused by administrative procedures could result in children losing their vision—or even their lives.”

With the MFDS’s GIFT designation, Ojemda will be eligible for an expedited review process that can shorten the formal review period by up to 25%. In Korea, the indication is for the treatment of recurrent or refractory pediatric low-grade glioma harboring a BRAF fusion, rearrangement, or BRAF V600 mutation in patients aged six months or older who have previously received systemic therapy.

Although the drug has now taken the first step toward commercialization in Korea by entering the expedited review pathway, pediatric patients must still overcome another major hurdle before they can access the treatment without significant financial burden—National Health Insurance reimbursement.

A medical official said, "Expedited review is tremendously encouraging news for children with rare brain tumors who have no alternative treatment options. However, the reimbursement review process must also reflect the unique characteristics of pediatric diseases and the realities of clinical practice. Flexible reimbursement criteria should allow treatment to be resumed without unnecessary restrictions after temporary interruptions due to adverse events, so that these children are no longer left in a therapeutic blind spot."