FDA approves pair of therapies for rare pediatric cancers: Novartis’ Lutathera and Day One’s Ojemda

The FDA has signed off on two novel therapies targeting rare pediatric cancers. Novartis’ Lutathera targets aggressive gastroenteropancreatic neuroendocrine tumors (GEP-NETs) in children 12 and up, while Day One Biopharma’s Ojemda (tovorafenib) tackles treatment-resistant BRAF-mutated relapsed or refractory pediatric low-grade glioma (pLGG) with a BRAF fusion or rearrangement, or BRAF V600 mutation

These approvals offer new treatment possibilities for young patients with rare pediatric cancers who previously had limited treatment options. The FDA’s decisions highlight the growing focus on developing targeted therapies for these underserved malignancies.

Lutathera targets rare gastrointestinal cancers in children

Lutathera, a radioligand therapy (RLT) from Novartis, has won an indication for the treatment of pediatric patients 12 years and older with somatostatin receptor-positive (SSTR+) GEP-NETs. GEP-NETs are a group of rare, often slow-growing cancers that originate in the neuroendocrine cells of the gastrointestinal tract and pancreas. These tumors can be particularly challenging to treat, especially in advanced stages.

The therapy won FDA approval for gastroenteropancreatic neuroendocrine tumors in 2018. 

The NETTER-P trial, which served as the basis for Lutathera’s most-recent approval, demonstrated its consistent safety profile and comparable drug exposure between pediatric and adult patients. 

Ojemda: A personalized approach to childhood brain tumors

FDA’s approval of Ojemda covers children as young as six months with hard-to-treat pLGG. This targeted drug attacks a key mutation found in up to 75% of these brain tumors, the most common in children. The mutation plays an important role in the growth and progression of the cancer, making it a promising target for targeted therapies like Ojemda.

Results from the FIREFLY-1 trial supported Ojemda’s approval. The trial showed a promising response rate in children with this specific mutation, demonstrating the power of personalized medicine for rare childhood cancers. 

Ojemda stands out as the only systemic pLGG treatment with a convenient once-weekly dose, available as a tablet or liquid, that can be taken with or without food.

To support patients, Day One Biopharmaceuticals has launched an EveryDay Support From Day One program program to help them navigate insurance, access financial assistance, and get information on the therapy. 

Experts like Dr. Sabine Mueller and Courtney Davies from the Pediatric Brain Tumor Foundation praised the approvals, highlighting the importance of new treatment options for children with pLGG and their families.

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Filed Under: clinical trials, Oncology, Rare disease
Tagged With: ediatric Cancers, FDA approvals, Orphan Drugs, R&D investment, Rare Disease Treatment, Targeted Therapies
 

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